Mitochondrial disease patient motivations and barriers to participate in clinical trials
Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to cli...
Saved in:
| Published in | PloS one Vol. 13; no. 5; p. e0197513 |
|---|---|
| Main Authors | , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Public Library of Science
17.05.2018
Public Library of Science (PLoS) |
| Subjects | |
| Online Access | Get full text |
| ISSN | 1932-6203 1932-6203 |
| DOI | 10.1371/journal.pone.0197513 |
Cover
| Abstract | Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD.
A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design.
PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3) participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult), prior research trial experience, or disease severity.
These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions. |
|---|---|
| AbstractList | Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD. A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design. PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions. BACKGROUND:Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD. METHODS:A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design. RESULTS:PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3) participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult), prior research trial experience, or disease severity. CONCLUSIONS:These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions. Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD.BACKGROUNDClinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD.A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design.METHODSA survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design.PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3) participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult), prior research trial experience, or disease severity.RESULTSPMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3) participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult), prior research trial experience, or disease severity.These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions.CONCLUSIONSThese data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions. Background Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD. Methods A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design. Results PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3) participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult), prior research trial experience, or disease severity. Conclusions These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions. Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD. A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design. PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3) participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult), prior research trial experience, or disease severity. These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions. |
| Audience | Academic |
| Author | Xiao, Rui Stoddart, Amy Thompson, John L. P. Burrill, Natalie McCormack, Shana Williams, Lauren Falk, Marni J. Holberts, Amy Zolkipli-Cunningham, Zarazuela Wang, Xiaoyan McCormick, Elizabeth M. |
| AuthorAffiliation | 2 Mitochondrial Medicine Frontier Program, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America Texas Technical University Health Sciences Center, UNITED STATES 5 Division of Human Genetics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America 8 University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States of America 1 Division of Neurology, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America 4 Arcadia University, Glenside, Pennsylvania, United States of America 9 Department of Biostatistics, Mailman School of Public Health, Columbia University Medical Center, New York, New York, United States of America 3 Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America 7 Division of Endocrinology and Diabetes, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvan |
| AuthorAffiliation_xml | – name: 7 Division of Endocrinology and Diabetes, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America – name: 8 University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States of America – name: 5 Division of Human Genetics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America – name: Texas Technical University Health Sciences Center, UNITED STATES – name: 3 Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America – name: 4 Arcadia University, Glenside, Pennsylvania, United States of America – name: 9 Department of Biostatistics, Mailman School of Public Health, Columbia University Medical Center, New York, New York, United States of America – name: 6 Rare Diseases Clinical Research Network, Health Informatics Institute, University of South Florida, Tampa, Florida, United States of America – name: 1 Division of Neurology, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America – name: 2 Mitochondrial Medicine Frontier Program, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America |
| Author_xml | – sequence: 1 givenname: Zarazuela surname: Zolkipli-Cunningham fullname: Zolkipli-Cunningham, Zarazuela – sequence: 2 givenname: Rui surname: Xiao fullname: Xiao, Rui – sequence: 3 givenname: Amy surname: Stoddart fullname: Stoddart, Amy – sequence: 4 givenname: Elizabeth M. surname: McCormick fullname: McCormick, Elizabeth M. – sequence: 5 givenname: Amy surname: Holberts fullname: Holberts, Amy – sequence: 6 givenname: Natalie surname: Burrill fullname: Burrill, Natalie – sequence: 7 givenname: Shana surname: McCormack fullname: McCormack, Shana – sequence: 8 givenname: Lauren surname: Williams fullname: Williams, Lauren – sequence: 9 givenname: Xiaoyan surname: Wang fullname: Wang, Xiaoyan – sequence: 10 givenname: John L. P. surname: Thompson fullname: Thompson, John L. P. – sequence: 11 givenname: Marni J. orcidid: 0000-0002-1723-6728 surname: Falk fullname: Falk, Marni J. |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/29771953$$D View this record in MEDLINE/PubMed |
| BookMark | eNqNk1uL1DAUx4usuBf9BqIFQfRhxlyatvFBWBYvAysL3vAtpOnpTIZMMibpot_edKa7TJdFpA8Jp7_zPzl_zjnNjqyzkGVPMZpjWuE3a9d7K818m8JzhHnFMH2QnWBOyawkiB4d3I-z0xDWCDFal-Wj7JjwqsKc0ZPs52cdnVo523otTd7qADJAvpVRg435xkV9ne7OhlzaNm-k9xp8yKNLjI9a6YRCrm2ujLZaJY04KIXH2cMuHfBkPM-y7x_ef7v4NLu8-ri4OL-cqZKTOGO4wDXBpCCMItmVoGRRdzUiNWWyA0kZVE2FK0Zp2aKKMNY2HEg9dMsb2tGz7Pled2tcEKMpQRBUoKooeM0TsdgTrZNrsfV6I_0f4aQWu4DzS7HrxIDAkhBOKTQt0IIXiGNMW5LewzgvqZRJ691YrW820KrkkZdmIjr9Y_VKLN21YJxVtCyTwKtRwLtfPYQoNjooMEZacP3u3bikGOE6oS_uoPd3N1JLmRrQtnOprhpExTmjHBesYEPZ-T1U-lrYaJUmqNMpPkl4PUlITITfcSn7EMTi65f_Z69-TNmXB-wKpImr4Ey_G7Ep-OzQ6VuLb0Y3AcUeUN6F4KG7RTASw4bc2CWGDRHjhqS0t3fSlI67CU-OaPPv5L_G3BVy |
| CitedBy_id | crossref_primary_10_1007_s13311_023_01445_6 crossref_primary_10_1093_hmg_ddab059 crossref_primary_10_1016_j_mito_2021_08_001 crossref_primary_10_1016_j_ymgme_2024_108348 crossref_primary_10_1002_jcsm_12559 crossref_primary_10_1002_jimd_12319 crossref_primary_10_1016_j_mito_2021_09_008 crossref_primary_10_1126_scisignal_aaw3159 crossref_primary_10_1212_NXG_0000000000000402 crossref_primary_10_1002_ncp_10739 crossref_primary_10_12688_wellcomeopenres_16116_1 crossref_primary_10_3389_ftox_2021_797496 crossref_primary_10_1097_MOP_0000000000000686 crossref_primary_10_1016_j_nmd_2023_12_014 crossref_primary_10_1007_s43441_023_00571_9 crossref_primary_10_1007_s44162_023_00021_y crossref_primary_10_1016_j_gim_2025_101386 crossref_primary_10_1186_s13023_022_02460_0 crossref_primary_10_1007_s13311_023_01418_9 crossref_primary_10_1038_s41390_021_01410_z crossref_primary_10_1002_ggn2_202100047 crossref_primary_10_1016_j_gim_2023_100831 crossref_primary_10_1016_j_ymgme_2022_05_001 crossref_primary_10_1097_WCO_0000000000000743 crossref_primary_10_1002_humu_24107 crossref_primary_10_1016_j_nmd_2019_09_012 crossref_primary_10_1007_s40271_020_00482_z crossref_primary_10_1016_j_ymgme_2022_02_006 crossref_primary_10_3390_toxics10020056 crossref_primary_10_1002_crt2_41 crossref_primary_10_1148_rg_2020200052 crossref_primary_10_1097_CND_0000000000000303 crossref_primary_10_1016_j_mito_2019_10_010 crossref_primary_10_1093_hmg_ddz023 crossref_primary_10_1186_s13023_022_02286_w crossref_primary_10_2139_ssrn_4057358 crossref_primary_10_1186_s13023_020_01443_3 crossref_primary_10_1002_ana_26260 crossref_primary_10_1016_j_ajo_2019_03_009 crossref_primary_10_1093_brain_awae308 crossref_primary_10_1002_jimd_12281 crossref_primary_10_1186_s13023_024_03320_9 crossref_primary_10_1016_j_clinthera_2024_03_008 crossref_primary_10_1007_s11332_023_01132_1 crossref_primary_10_1186_s41687_022_00534_y crossref_primary_10_1186_s13023_023_02754_x crossref_primary_10_1002_jimd_12320 |
| Cites_doi | 10.1016/0022-510X(90)90015-F 10.1186/s13023-016-0445-8 10.1378/chest.13-0606 10.1097/WCO.0000000000000243 10.1038/gim.2014.177 10.1016/j.cct.2015.11.006 10.1177/0883073813481622 10.4103/2229-3485.159936 10.1186/1750-1172-8-44 10.1177/0883073810371509 10.1093/brain/aww303 10.1016/j.cct.2013.04.004 10.1016/j.mito.2011.05.002 10.1016/j.mito.2012.11.002 10.1038/nrneurol.2013.129 10.1002/ana.24362 10.1517/14728214.2015.1041375 10.2217/pme.11.7 10.1007/s13311-012-0174-1 10.1016/j.jval.2013.04.004 10.1007/s13311-013-0176-7 10.1186/s12883-015-0408-z 10.1111/dmcn.12119 10.1016/j.ymgme.2007.11.018 10.1016/j.addr.2008.05.001 10.1097/WCO.0000000000000245 10.1016/j.ymgme.2009.11.005 10.1016/j.ymgme.2016.09.002 10.1016/j.parkreldis.2015.07.018 10.1038/nrdp.2016.80 10.1007/s11940-009-0046-0 10.1016/j.cct.2012.02.012 10.1016/j.mito.2007.03.008 10.1093/ckj/sfv060 10.1146/annurev-genom-091212-153525 10.1212/01.wnl.0000218155.46739.90 |
| ContentType | Journal Article |
| Copyright | COPYRIGHT 2018 Public Library of Science 2018 Zolkipli-Cunningham et al. This is an open access article distributed under the terms of the Creative Commons Attribution License: http://creativecommons.org/licenses/by/4.0/ (the “License”), which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. 2018 Zolkipli-Cunningham et al 2018 Zolkipli-Cunningham et al |
| Copyright_xml | – notice: COPYRIGHT 2018 Public Library of Science – notice: 2018 Zolkipli-Cunningham et al. This is an open access article distributed under the terms of the Creative Commons Attribution License: http://creativecommons.org/licenses/by/4.0/ (the “License”), which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License. – notice: 2018 Zolkipli-Cunningham et al 2018 Zolkipli-Cunningham et al |
| DBID | AAYXX CITATION CGR CUY CVF ECM EIF NPM IOV ISR 3V. 7QG 7QL 7QO 7RV 7SN 7SS 7T5 7TG 7TM 7U9 7X2 7X7 7XB 88E 8AO 8C1 8FD 8FE 8FG 8FH 8FI 8FJ 8FK ABJCF ABUWG AEUYN AFKRA ARAPS ATCPS AZQEC BBNVY BENPR BGLVJ BHPHI C1K CCPQU D1I DWQXO FR3 FYUFA GHDGH GNUQQ H94 HCIFZ K9. KB. KB0 KL. L6V LK8 M0K M0S M1P M7N M7P M7S NAPCQ P5Z P62 P64 PATMY PDBOC PHGZM PHGZT PIMPY PJZUB PKEHL PPXIY PQEST PQGLB PQQKQ PQUKI PTHSS PYCSY RC3 7X8 5PM DOA |
| DOI | 10.1371/journal.pone.0197513 |
| DatabaseName | CrossRef Medline MEDLINE MEDLINE (Ovid) MEDLINE MEDLINE PubMed Gale In Context: Opposing Viewpoints Gale In Context: Science ProQuest Central (Corporate) Animal Behavior Abstracts Bacteriology Abstracts (Microbiology B) Biotechnology Research Abstracts Nursing & Allied Health Database (Proquest) Ecology Abstracts Entomology Abstracts (Full archive) Immunology Abstracts Meteorological & Geoastrophysical Abstracts Nucleic Acids Abstracts Virology and AIDS Abstracts Agricultural Science Collection Health & Medical Collection ProQuest Central (purchase pre-March 2016) Medical Database (Alumni Edition) ProQuest Pharma Collection Proquest Public Health Database Technology Research Database ProQuest SciTech Collection ProQuest Technology Collection ProQuest Natural Science Collection Hospital Premium Collection Hospital Premium Collection (Alumni Edition) ProQuest Central (Alumni) (purchase pre-March 2016) Materials Science & Engineering Collection ProQuest Central ProQuest One Sustainability ProQuest Central UK/Ireland Advanced Technologies & Aerospace Collection Agricultural & Environmental Science Collection ProQuest Central Essentials Biological Science Collection ProQuest Central Technology Collection (via ProQuest SciTech Premium Collection) Natural Science Collection Environmental Sciences and Pollution Management ProQuest One ProQuest Materials Science Collection ProQuest Central Engineering Research Database Proquest Health Research Premium Collection Health Research Premium Collection (Alumni) ProQuest Central Student AIDS and Cancer Research Abstracts SciTech Premium Collection ProQuest Health & Medical Complete (Alumni) Materials Science Database Nursing & Allied Health Database (Alumni Edition) Meteorological & Geoastrophysical Abstracts - Academic ProQuest Engineering Collection ProQuest Biological Science Collection Agricultural Science Database ProQuest Health & Medical Collection Medical Database Algology Mycology and Protozoology Abstracts (Microbiology C) Biological Science Database Engineering Database (Proquest) Nursing & Allied Health Premium Advanced Technologies & Aerospace Database ProQuest Advanced Technologies & Aerospace Collection Biotechnology and BioEngineering Abstracts Environmental Science Database Materials Science Collection ProQuest Central Premium ProQuest One Academic (New) Publicly Available Content Database ProQuest Health & Medical Research Collection ProQuest One Academic Middle East (New) ProQuest One Health & Nursing ProQuest One Academic Eastern Edition (DO NOT USE) ProQuest One Applied & Life Sciences ProQuest One Academic ProQuest One Academic UKI Edition Engineering Collection Environmental Science Collection Genetics Abstracts MEDLINE - Academic PubMed Central (Full Participant titles) DOAJ Directory of Open Access Journals |
| DatabaseTitle | CrossRef MEDLINE Medline Complete MEDLINE with Full Text PubMed MEDLINE (Ovid) Agricultural Science Database Publicly Available Content Database ProQuest Central Student ProQuest Advanced Technologies & Aerospace Collection ProQuest Central Essentials Nucleic Acids Abstracts SciTech Premium Collection Environmental Sciences and Pollution Management ProQuest One Applied & Life Sciences ProQuest One Sustainability Health Research Premium Collection Meteorological & Geoastrophysical Abstracts Natural Science Collection Health & Medical Research Collection Biological Science Collection ProQuest Central (New) ProQuest Medical Library (Alumni) Engineering Collection Advanced Technologies & Aerospace Collection Engineering Database Virology and AIDS Abstracts ProQuest Biological Science Collection ProQuest One Academic Eastern Edition Agricultural Science Collection ProQuest Hospital Collection ProQuest Technology Collection Health Research Premium Collection (Alumni) Biological Science Database Ecology Abstracts ProQuest Hospital Collection (Alumni) Biotechnology and BioEngineering Abstracts Environmental Science Collection Entomology Abstracts Nursing & Allied Health Premium ProQuest Health & Medical Complete ProQuest One Academic UKI Edition Environmental Science Database ProQuest Nursing & Allied Health Source (Alumni) Engineering Research Database ProQuest One Academic Meteorological & Geoastrophysical Abstracts - Academic ProQuest One Academic (New) Technology Collection Technology Research Database ProQuest One Academic Middle East (New) Materials Science Collection ProQuest Health & Medical Complete (Alumni) ProQuest Central (Alumni Edition) ProQuest One Community College ProQuest One Health & Nursing ProQuest Natural Science Collection ProQuest Pharma Collection ProQuest Central ProQuest Health & Medical Research Collection Genetics Abstracts ProQuest Engineering Collection Biotechnology Research Abstracts Health and Medicine Complete (Alumni Edition) ProQuest Central Korea Bacteriology Abstracts (Microbiology B) Algology Mycology and Protozoology Abstracts (Microbiology C) Agricultural & Environmental Science Collection AIDS and Cancer Research Abstracts Materials Science Database ProQuest Materials Science Collection ProQuest Public Health ProQuest Nursing & Allied Health Source ProQuest SciTech Collection Advanced Technologies & Aerospace Database ProQuest Medical Library Animal Behavior Abstracts Materials Science & Engineering Collection Immunology Abstracts ProQuest Central (Alumni) MEDLINE - Academic |
| DatabaseTitleList | MEDLINE - Academic Agricultural Science Database MEDLINE |
| Database_xml | – sequence: 1 dbid: DOA name: DOAJ Directory of Open Access Journals url: https://www.doaj.org/ sourceTypes: Open Website – sequence: 2 dbid: NPM name: PubMed url: https://proxy.k.utb.cz/login?url=http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=PubMed sourceTypes: Index Database – sequence: 3 dbid: EIF name: MEDLINE url: https://proxy.k.utb.cz/login?url=https://www.webofscience.com/wos/medline/basic-search sourceTypes: Index Database – sequence: 4 dbid: 8FG name: ProQuest Technology Collection url: https://search.proquest.com/technologycollection1 sourceTypes: Aggregation Database |
| DeliveryMethod | fulltext_linktorsrc |
| Discipline | Sciences (General) Medicine Public Health |
| DocumentTitleAlternate | Mitochondrial disease clinical trial patient perspective |
| EISSN | 1932-6203 |
| ExternalDocumentID | 2040744989 oai_doaj_org_article_1a22933ebde349409113d28f859963aa PMC5957366 A539145456 29771953 10_1371_journal_pone_0197513 |
| Genre | Comparative Study Research Support, Non-U.S. Gov't Journal Article Research Support, N.I.H., Extramural |
| GeographicLocations | New York United States--US Pennsylvania |
| GeographicLocations_xml | – name: New York – name: Pennsylvania – name: United States--US |
| GrantInformation_xml | – fundername: NICHD NIH HHS grantid: U54 HD086984 – fundername: NIDDK NIH HHS grantid: K12 DK094723 – fundername: NIGMS NIH HHS grantid: T32 GM008638 – fundername: NIDDK NIH HHS grantid: K23 DK102659 – fundername: NINDS NIH HHS grantid: U54 NS078059 – fundername: NICHD NIH HHS grantid: U24 HD093483 – fundername: ; grantid: T32-GM008638 |
| GroupedDBID | --- 123 29O 2WC 53G 5VS 7RV 7X2 7X7 7XC 88E 8AO 8C1 8CJ 8FE 8FG 8FH 8FI 8FJ A8Z AAFWJ AAUCC AAWOE AAYXX ABDBF ABIVO ABJCF ABUWG ACGFO ACIHN ACIWK ACPRK ACUHS ADBBV ADRAZ AEAQA AENEX AEUYN AFKRA AFPKN AFRAH AHMBA ALIPV ALMA_UNASSIGNED_HOLDINGS AOIJS APEBS ARAPS ATCPS BAWUL BBNVY BCNDV BENPR BGLVJ BHPHI BKEYQ BPHCQ BVXVI BWKFM CCPQU CITATION CS3 D1I D1J D1K DIK DU5 E3Z EAP EAS EBD EMOBN ESX EX3 F5P FPL FYUFA GROUPED_DOAJ GX1 HCIFZ HH5 HMCUK HYE IAO IEA IGS IHR IHW INH INR IOV IPY ISE ISR ITC K6- KB. KQ8 L6V LK5 LK8 M0K M1P M48 M7P M7R M7S M~E NAPCQ O5R O5S OK1 OVT P2P P62 PATMY PDBOC PHGZM PHGZT PIMPY PQQKQ PROAC PSQYO PTHSS PV9 PYCSY RNS RPM RZL SV3 TR2 UKHRP WOQ WOW ~02 ~KM CGR CUY CVF ECM EIF IPNFZ NPM PJZUB PPXIY PQGLB RIG BBORY PMFND 3V. 7QG 7QL 7QO 7SN 7SS 7T5 7TG 7TM 7U9 7XB 8FD 8FK AZQEC C1K DWQXO FR3 GNUQQ H94 K9. KL. M7N P64 PKEHL PQEST PQUKI RC3 7X8 ESTFP PUEGO 5PM AAPBV ABPTK BBAFP N95 |
| ID | FETCH-LOGICAL-c692t-5141821242530af6eca48f802835afea35e7b7175336d07255db9e2875139b3f3 |
| IEDL.DBID | M48 |
| ISSN | 1932-6203 |
| IngestDate | Mon Dec 05 23:08:08 EST 2022 Wed Aug 27 01:23:34 EDT 2025 Thu Aug 21 13:29:40 EDT 2025 Mon Sep 08 14:10:18 EDT 2025 Fri Jul 25 11:20:46 EDT 2025 Tue Jun 17 21:08:31 EDT 2025 Tue Jun 10 20:49:52 EDT 2025 Fri Jun 27 04:05:43 EDT 2025 Fri Jun 27 04:07:25 EDT 2025 Thu May 22 21:24:19 EDT 2025 Mon Jul 21 05:59:09 EDT 2025 Tue Jul 01 03:51:23 EDT 2025 Thu Apr 24 23:04:03 EDT 2025 |
| IsDoiOpenAccess | true |
| IsOpenAccess | true |
| IsPeerReviewed | true |
| IsScholarly | true |
| Issue | 5 |
| Language | English |
| License | This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. Creative Commons Attribution License |
| LinkModel | DirectLink |
| MergedId | FETCHMERGED-LOGICAL-c692t-5141821242530af6eca48f802835afea35e7b7175336d07255db9e2875139b3f3 |
| Notes | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 ObjectType-Article-2 ObjectType-Feature-1 content type line 23 Competing Interests: The authors have declared that no competing interests exist. |
| ORCID | 0000-0002-1723-6728 |
| OpenAccessLink | http://journals.scholarsportal.info/openUrl.xqy?doi=10.1371/journal.pone.0197513 |
| PMID | 29771953 |
| PQID | 2040744989 |
| PQPubID | 1436336 |
| PageCount | e0197513 |
| ParticipantIDs | plos_journals_2040744989 doaj_primary_oai_doaj_org_article_1a22933ebde349409113d28f859963aa pubmedcentral_primary_oai_pubmedcentral_nih_gov_5957366 proquest_miscellaneous_2041631018 proquest_journals_2040744989 gale_infotracmisc_A539145456 gale_infotracacademiconefile_A539145456 gale_incontextgauss_ISR_A539145456 gale_incontextgauss_IOV_A539145456 gale_healthsolutions_A539145456 pubmed_primary_29771953 crossref_primary_10_1371_journal_pone_0197513 crossref_citationtrail_10_1371_journal_pone_0197513 |
| ProviderPackageCode | CITATION AAYXX |
| PublicationCentury | 2000 |
| PublicationDate | 2018-05-17 |
| PublicationDateYYYYMMDD | 2018-05-17 |
| PublicationDate_xml | – month: 05 year: 2018 text: 2018-05-17 day: 17 |
| PublicationDecade | 2010 |
| PublicationPlace | United States |
| PublicationPlace_xml | – name: United States – name: San Francisco – name: San Francisco, CA USA |
| PublicationTitle | PloS one |
| PublicationTitleAlternate | PLoS One |
| PublicationYear | 2018 |
| Publisher | Public Library of Science Public Library of Science (PLoS) |
| Publisher_xml | – name: Public Library of Science – name: Public Library of Science (PLoS) |
| References | PW Stacpoole (ref6) 2011; 11 PB Shieh (ref12) 2015; 28 NS Sharma (ref35) 2015; 6 L Merlini (ref13) 2015; 15 E McCormick (ref18) 2013; 10 EO Lillie (ref30) 2011; 8 PP Koay (ref39) 2013; 14 S Koene (ref34) 2013; 13 S Henrard (ref40) 2015; 13 ref17 CM McDonald (ref25) 2010; 25 SC Groft (ref38) 2013; 144 RT Wang (ref37) 2015; 28 N Bresolin (ref3) 1990; 100 S Koene (ref27) 2013; 55 M Picillo (ref41) 2015; 21 N Kayadjanian (ref14) 2013; 8 RH Haas (ref7) 2007; 7 RH Haas (ref19) 2008; 94 RR Weaver (ref36) 2015 DS Kerr (ref29) 2013; 10 G Pfeffer (ref5) 2013; 9 ref23 A Baker (ref32) 2015; 8 GS Gorman (ref2) 2015; 77 B Wirth (ref15) 2015; 20 F Distelmaier (ref20) 2017; 140 HL Peay (ref42) 2016; 46 K Sofou (ref31) 2013; 28 G Pfeffer (ref4) 2012; 4 RL Richesson (ref21) 2012; 33 PA Merkel (ref22) 2016; 11 KM Camp (ref28) 2016; 119 S Parikh (ref10) 2009; 11 MA Tarnopolsky (ref8) 2008; 60 S Parikh (ref11) 2015; 17 EA de Blieck (ref16) 2013; 35 EJ Higgs (ref33) 2015 DS Kerr (ref9) 2010; 99 DR Lynch (ref26) 2006; 66 GS Gorman (ref1) 2016; 2 LS Matza (ref24) 2013; 16 |
| References_xml | – volume: 100 start-page: 70 issue: 1–2 year: 1990 ident: ref3 article-title: Ubidecarenone in the treatment of mitochondrial myopathies: a multi-center double-blind trial publication-title: J Neurol Sci doi: 10.1016/0022-510X(90)90015-F – volume: 11 start-page: 66 issue: 1 year: 2016 ident: ref22 article-title: The partnership of patient advocacy groups and clinical investigators in the rare diseases clinical research network publication-title: Orphanet J Rare Dis doi: 10.1186/s13023-016-0445-8 – volume: 144 start-page: 16 issue: 1 year: 2013 ident: ref38 article-title: Rare diseases research: expanding collaborative translational research opportunities publication-title: Chest doi: 10.1378/chest.13-0606 – volume: 28 start-page: 542 issue: 5 year: 2015 ident: ref12 article-title: Duchenne muscular dystrophy: clinical trials and emerging tribulations publication-title: Curr Opin Neurol doi: 10.1097/WCO.0000000000000243 – volume: 17 start-page: 689 issue: 9 year: 2015 ident: ref11 article-title: Diagnosis and management of mitochondrial disease: a consensus statement from the Mitochondrial Medicine Society publication-title: Genet Med doi: 10.1038/gim.2014.177 – volume: 46 start-page: 1 year: 2016 ident: ref42 article-title: "Watching time tick by …": Decision making for Duchenne muscular dystrophy trials publication-title: Contemp Clin Trials doi: 10.1016/j.cct.2015.11.006 – volume: 28 start-page: 663 issue: 5 year: 2013 ident: ref31 article-title: Mitochondrial disease: a challenge for the caregiver, the family, and society publication-title: J Child Neurol doi: 10.1177/0883073813481622 – volume: 6 start-page: 134 issue: 3 year: 2015 ident: ref35 article-title: Patient centric approach for clinical trials: Current trend and new opportunities publication-title: Perspect Clin Res doi: 10.4103/2229-3485.159936 – volume: 8 start-page: 44 year: 2013 ident: ref14 article-title: SMA- EUROPE workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe publication-title: Orphanet J Rare Dis doi: 10.1186/1750-1172-8-44 – volume: 25 start-page: 1130 issue: 9 year: 2010 ident: ref25 article-title: Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy publication-title: J Child Neurol doi: 10.1177/0883073810371509 – volume: 140 start-page: e11 issue: Pt 2 year: 2017 ident: ref20 article-title: Treatable mitochondrial diseases: cofactor metabolism and beyond publication-title: Brain doi: 10.1093/brain/aww303 – volume: 35 start-page: 48 issue: 2 year: 2013 ident: ref16 article-title: Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates publication-title: Contemp Clin Trials doi: 10.1016/j.cct.2013.04.004 – volume: 11 start-page: 679 issue: 5 year: 2011 ident: ref6 article-title: Why are there no proven therapies for genetic mitochondrial diseases? publication-title: Mitochondrion doi: 10.1016/j.mito.2011.05.002 – volume: 13 start-page: 15 issue: 1 year: 2013 ident: ref34 article-title: Developing outcome measures for pediatric mitochondrial disorders: which complaints and limitations are most burdensome to patients and their parents? publication-title: Mitochondrion doi: 10.1016/j.mito.2012.11.002 – volume: 9 start-page: 474 issue: 8 year: 2013 ident: ref5 article-title: New treatments for mitochondrial disease-no time to drop our standards publication-title: Nat Rev Neurol doi: 10.1038/nrneurol.2013.129 – ident: ref17 – volume: 77 start-page: 753 issue: 5 year: 2015 ident: ref2 article-title: Prevalence of nuclear and mitochondrial DNA mutations related to adult mitochondrial disease publication-title: Ann Neurol doi: 10.1002/ana.24362 – volume: 20 start-page: 353 issue: 3 year: 2015 ident: ref15 article-title: Moving towards treatments for spinal muscular atrophy: hopes and limits publication-title: Expert Opin Emerg Drugs doi: 10.1517/14728214.2015.1041375 – volume: 13 start-page: 302 issue: 2 year: 2015 ident: ref40 article-title: Participation of people with haemophilia in clinical trials of new treatments: an investigation of patients’ motivations and existing barriers publication-title: Blood Transfus – volume: 8 start-page: 161 issue: 2 year: 2011 ident: ref30 article-title: The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? publication-title: Per Med doi: 10.2217/pme.11.7 – volume: 4 start-page: CD004426 year: 2012 ident: ref4 article-title: Treatment for mitochondrial disorders publication-title: Cochrane Database Syst Rev – volume: 10 start-page: 251 issue: 2 year: 2013 ident: ref18 article-title: Molecular genetic testing for mitochondrial disease: from one generation to the next publication-title: Neurotherapeutics doi: 10.1007/s13311-012-0174-1 – volume: 16 start-page: 461 issue: 4 year: 2013 ident: ref24 article-title: Pediatric patient-reported outcome instruments for research to support medical product labeling: report of the ISPOR PRO good research practices for the assessment of children and adolescents task force publication-title: Value Health doi: 10.1016/j.jval.2013.04.004 – volume: 10 start-page: 307 issue: 2 year: 2013 ident: ref29 article-title: Review of clinical trials for mitochondrial disorders: 1997–2012 publication-title: Neurotherapeutics doi: 10.1007/s13311-013-0176-7 – volume: 15 start-page: 153 year: 2015 ident: ref13 article-title: Improving clinical trial design for Duchenne muscular dystrophy publication-title: BMC Neurol doi: 10.1186/s12883-015-0408-z – volume: 55 start-page: 698 issue: 8 year: 2013 ident: ref27 article-title: Towards the harmonization of outcome measures in children with mitochondrial disorders publication-title: Dev Med Child Neurol doi: 10.1111/dmcn.12119 – year: 2015 ident: ref36 article-title: Reconciling evidence-based medicine and patient-centred care: defining evidence-based inputs to patient-centred decisions publication-title: J Eval Clin Pract – year: 2015 ident: ref33 article-title: ’A short time but a lovely little short time’: Bereaved parents’ experiences of having a child with spinal muscular atrophy type 1 publication-title: J Paediatr Child Health – volume: 94 start-page: 16 issue: 1 year: 2008 ident: ref19 article-title: The in-depth evaluation of suspected mitochondrial disease publication-title: Mol Genet Metab doi: 10.1016/j.ymgme.2007.11.018 – ident: ref23 – volume: 60 start-page: 1561 issue: 13–14 year: 2008 ident: ref8 article-title: The mitochondrial cocktail: rationale for combined nutraceutical therapy in mitochondrial cytopathies publication-title: Adv Drug Deliv Rev doi: 10.1016/j.addr.2008.05.001 – volume: 28 start-page: 535 issue: 5 year: 2015 ident: ref37 article-title: What can Duchenne Connect teach us about treating Duchenne muscular dystrophy? publication-title: Curr Opin Neurol doi: 10.1097/WCO.0000000000000245 – volume: 99 start-page: 246 issue: 3 year: 2010 ident: ref9 article-title: Treatment of mitochondrial electron transport chain disorders: a review of clinical trials over the past decade publication-title: Mol Genet Metab doi: 10.1016/j.ymgme.2009.11.005 – volume: 119 start-page: 187 issue: 3 year: 2016 ident: ref28 article-title: Nutritional interventions in primary mitochondrial disorders: Developing an evidence base publication-title: Mol Genet Metab doi: 10.1016/j.ymgme.2016.09.002 – volume: 21 start-page: 1041 issue: 9 year: 2015 ident: ref41 article-title: Recruitment strategies and patient selection in clinical trials for Parkinson’s disease: Going viral and keeping science and ethics at the highest standards publication-title: Parkinsonism Relat Disord doi: 10.1016/j.parkreldis.2015.07.018 – volume: 2 start-page: 16080 year: 2016 ident: ref1 article-title: Mitochondrial diseases publication-title: Nat Rev Dis Primers doi: 10.1038/nrdp.2016.80 – volume: 11 start-page: 414 issue: 6 year: 2009 ident: ref10 article-title: A modern approach to the treatment of mitochondrial disease publication-title: Curr Treat Options Neurol doi: 10.1007/s11940-009-0046-0 – volume: 33 start-page: 647 issue: 4 year: 2012 ident: ref21 article-title: The Rare Diseases Clinical Research Network Contact Registry update: features and functionality publication-title: Contemp Clin Trials doi: 10.1016/j.cct.2012.02.012 – volume: 7 start-page: S136 issue: Suppl year: 2007 ident: ref7 article-title: The evidence basis for coenzyme Q therapy in oxidative phosphorylation disease publication-title: Mitochondrion doi: 10.1016/j.mito.2007.03.008 – volume: 8 start-page: 531 issue: 5 year: 2015 ident: ref32 article-title: Understanding the physical and emotional impact of early-stage ADPKD: experiences and perspectives of patients and physicians publication-title: Clin Kidney J doi: 10.1093/ckj/sfv060 – volume: 14 start-page: 579 year: 2013 ident: ref39 article-title: The role of patient advocacy organizations in shaping genomic science publication-title: Annu Rev Genomics Hum Genet doi: 10.1146/annurev-genom-091212-153525 – volume: 66 start-page: 1711 issue: 11 year: 2006 ident: ref26 article-title: Measuring Friedreich ataxia: complementary features of examination and performance measures publication-title: Neurology doi: 10.1212/01.wnl.0000218155.46739.90 |
| SSID | ssj0053866 |
| Score | 2.4736395 |
| Snippet | Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of... Background Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous... BACKGROUND:Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous... Background Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous... |
| SourceID | plos doaj pubmedcentral proquest gale pubmed crossref |
| SourceType | Open Website Open Access Repository Aggregation Database Index Database Enrichment Source |
| StartPage | e0197513 |
| SubjectTerms | Adolescent Adult Aged Antioxidants Attitude to Health Care and treatment Child Child, Preschool Children & youth Clinical trials Clinical Trials as Topic - economics Clinical Trials as Topic - psychology Cohort Studies Collaboration Design Design factors Developmental Disabilities - etiology Developmental Disabilities - psychology Disease Drug development Epilepsy - etiology Epilepsy - psychology Fatigue Female Genetic aspects Health care facilities Health Expenditures Hospitals Humans Infant Informed Consent Intolerance Lead Male Medical research Medicine Medicine and Health Sciences Middle Aged Mitochondria Mitochondrial diseases Mitochondrial Diseases - drug therapy Mitochondrial Diseases - psychology Mitochondrial DNA Molecular chains Motivation Muscle Weakness - etiology Muscle Weakness - psychology Muscles Muscular dystrophy Muscular fatigue Patient Acceptance of Health Care - psychology Patients People and Places Phlebotomy - psychology Public health Rare diseases Research and Analysis Methods Research Design Severity of Illness Index Software Surveys and Questionnaires Symptom Assessment Young Adult |
| SummonAdditionalLinks | – databaseName: DOAJ Directory of Open Access Journals dbid: DOA link: http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwrV1Lb9QwELbQnrggWh4NbalBSMAhbWzHcXxsEVVBKkhA0d4sO3agUpusmt3_z0ziRA2qVA4c9rKerHbn4flmPf6GkDellxlz3KU-uCzNpaxSXfos9VqWPreZ5w7_hzz_Upxd5J-Xcnlr1Bf2hA30wIPijpjlkJFEcD4gkwqkNyY8L-sSeUWE7aERpLGxmBr2YIjioogX5YRiR9Euh6u2CYcAapRkYpaIer7-aVderK7a7i7I-Xfn5K1UdPqYPIoYkh4P332LPAjNNtmKUdrRd5FK-v0TsjyHgIUNrvHoZzSextBIpkqvp-FmHbWNp87e4AC7jq5bkBkbrgO9bOh4g5L2Yz66p-Ti9OOPD2dpnKWQVoXm6xRwEVQSDAsMkdm6CMhmXpeILqStgxUyKKeQtlMUPlNQaHinA5RToCntRC2ekUUD2tshtNa-Yt5anpc6L7hzFcAQ55iu4MUtT4gYFWuqSDSO8y6uTH96pqDgGPRk0BwmmiMh6fTUaiDauEf-BG02ySJNdv8GOI-JzmPuc56EHKDFzXDndAp2cyyFZjmCy4S87iWQKqPBXpxfdtN15tPXn_8g9P3bTOhtFKpbUEdl4_0H-E1IwTWT3JtJQsBXs-Ud9M9RK53hsBGrPNelhidHn717-dW0jB-K_XVNaDe9DABzZG9LyPPBxSfNcigR8LA1IWrm_DPVz1eay989U7nUUomiePE_bLVLHgJYLbFzg6k9sljfbMI-AMK1e9nH_h_2H11e priority: 102 providerName: Directory of Open Access Journals – databaseName: ProQuest Technology Collection dbid: 8FG link: http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwjV3di9QwEA-6gggi3qp31VOjCOpDzyZp2uZJTnE9hVVQT-4tJE16Hpztut39_51p02rlUB_6spku7Xx1Jpn5DSFPCicTZrmNnbdJnEpZxqpwSeyULFxqEsct7kMuP2RHx-n7E3kSNtzaUFY5-MTOUbumxD1ySNIh9UhTVaiXqx8xTo3C09UwQuMyucI4aBJ2ii_eDp4YbDnLQrucyNmLIJ2DVVP7AwhtcsnE5HPUofaPvnm2Om_aiwLPP-snf_sgLW6SGyGSpIe96HfIJV_PydVlOCufk-v9jhztG43mZCeYcUufBazp57fIyRIsGjxg7VARaTiuoQFtlX4fp5-11NSOWrPGCXct3TRAM1Rke3pW06HFknZzQNrb5Hjx5svrozgMW4jLTPFNDIETpBoMMxCRmCrzCHdeFRh-SFN5I6TPbY64niJzSQ6ZiLPKQ74FTFRWVOIOmdXA2D1CK-VK5ozhaaHSjFtbguSsZaqEixseETHwXJcBiRwHYpzr7ngth4ykZ6FGSekgqYjE412rHonjH_SvUJwjLeJodz8061MdzFIzwyHeEd46jzg9EDwx4Ti8N6LWCGMi8hCVQfdNqaM30IdSKJZi9BmRxx0FYmnUWKxzarZtq999_PofRJ8_TYieBqKqAXaUJjRIwDshRteEcn9CCR6hnCzvoeoOXGn1L9uBOwd1vnj50biMf4oFeLVvth0NRO4I7xaR3V77R85yyCHwNDYi-cQuJqyfrtRn3zooc6lkLrLs7t8f6x65BnFqgUUbLN8ns8166-9DLLixDzqD_wn6lV4V priority: 102 providerName: ProQuest |
| Title | Mitochondrial disease patient motivations and barriers to participate in clinical trials |
| URI | https://www.ncbi.nlm.nih.gov/pubmed/29771953 https://www.proquest.com/docview/2040744989 https://www.proquest.com/docview/2041631018 https://pubmed.ncbi.nlm.nih.gov/PMC5957366 https://doaj.org/article/1a22933ebde349409113d28f859963aa http://dx.doi.org/10.1371/journal.pone.0197513 |
| Volume | 13 |
| hasFullText | 1 |
| inHoldings | 1 |
| isFullTextHit | |
| isPrint | |
| journalDatabaseRights | – providerCode: PRVFSB databaseName: Free Full-Text Journals in Chemistry customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: HH5 dateStart: 20060101 isFulltext: true titleUrlDefault: http://abc-chemistry.org/ providerName: ABC ChemistRy – providerCode: PRVAFT databaseName: Open Access Digital Library customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: KQ8 dateStart: 20060101 isFulltext: true titleUrlDefault: http://grweb.coalliance.org/oadl/oadl.html providerName: Colorado Alliance of Research Libraries – providerCode: PRVAFT databaseName: Open Access Digital Library customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: KQ8 dateStart: 20061001 isFulltext: true titleUrlDefault: http://grweb.coalliance.org/oadl/oadl.html providerName: Colorado Alliance of Research Libraries – providerCode: PRVAON databaseName: DOAJ Directory of Open Access Journals customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: DOA dateStart: 20060101 isFulltext: true titleUrlDefault: https://www.doaj.org/ providerName: Directory of Open Access Journals – providerCode: PRVEBS databaseName: EBSCOhost Academic Search Ultimate customDbUrl: https://search.ebscohost.com/login.aspx?authtype=ip,shib&custid=s3936755&profile=ehost&defaultdb=asn eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: ABDBF dateStart: 20080101 isFulltext: true titleUrlDefault: https://search.ebscohost.com/direct.asp?db=asn providerName: EBSCOhost – providerCode: PRVBFR databaseName: Free Medical Journals customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: DIK dateStart: 20060101 isFulltext: true titleUrlDefault: http://www.freemedicaljournals.com providerName: Flying Publisher – providerCode: PRVFQY databaseName: GFMER Free Medical Journals customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: GX1 dateStart: 20060101 isFulltext: true titleUrlDefault: http://www.gfmer.ch/Medical_journals/Free_medical.php providerName: Geneva Foundation for Medical Education and Research – providerCode: PRVHPJ databaseName: ROAD: Directory of Open Access Scholarly Resources customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: M~E dateStart: 20060101 isFulltext: true titleUrlDefault: https://road.issn.org providerName: ISSN International Centre – providerCode: PRVAQN databaseName: PubMed Central customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: RPM dateStart: 20060101 isFulltext: true titleUrlDefault: https://www.ncbi.nlm.nih.gov/pmc/ providerName: National Library of Medicine – providerCode: PRVPQU databaseName: Health & Medical Collection customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: 7X7 dateStart: 20061201 isFulltext: true titleUrlDefault: https://search.proquest.com/healthcomplete providerName: ProQuest – providerCode: PRVPQU databaseName: ProQuest Central customDbUrl: http://www.proquest.com/pqcentral?accountid=15518 eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: BENPR dateStart: 20061201 isFulltext: true titleUrlDefault: https://www.proquest.com/central providerName: ProQuest – providerCode: PRVPQU databaseName: ProQuest Public Health Database customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: 8C1 dateStart: 20061201 isFulltext: true titleUrlDefault: https://search.proquest.com/publichealth providerName: ProQuest – providerCode: PRVPQU databaseName: ProQuest Technology Collection customDbUrl: eissn: 1932-6203 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: 8FG dateStart: 20061201 isFulltext: true titleUrlDefault: https://search.proquest.com/technologycollection1 providerName: ProQuest – providerCode: PRVFZP databaseName: Scholars Portal Journals: Open Access customDbUrl: eissn: 1932-6203 dateEnd: 20250930 omitProxy: true ssIdentifier: ssj0053866 issn: 1932-6203 databaseCode: M48 dateStart: 20061201 isFulltext: true titleUrlDefault: http://journals.scholarsportal.info providerName: Scholars Portal |
| link | http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwjV3db9MwELe27oUXxPhaYBSDEB8PqRI7ieMHhLZpZSB1oEFR3yI7dsakknRNK8F_z13qRAQVsYfmoT4nyvls_y53_h0hL1ITB6Fm2jdWB34Ux7kvUxP4RsapiVRgmMbvkJPz5GwafZzFsx3SBtqdAuutrh3Wk5ou56Of17_ewYR_21RtEGHbabSoSjsCyCLikL9cXPtYWgpDsK7Oxi7Zg-2KoelPoi7UABO-CWgikPETFnB3vu5fN-7tXw3Nf7eYDxbzqt6GVP9OuPxjBxvfIbcd9KRHG1vZJzu2vEv23eSu6WvHQP3mHplNYJ6DakqD5kldEIc6Dlb6o6uJVlNVGqrVEuve1XRVgUybp23pVUnbg5e0qQ5S3yfT8enXkzPflWDw80SylQ9wChyQEP0SHqgisUiCXqQISmJVWMVjK7RAtk-emECAf2K0tOCFgaak5gV_QAYlaO-A0EKaPDRKsSiVUcK0zgG9aB3KHH5MMY_wVrFZ7vjJsUzGPGuCbgL8lI2eMhyOzA2HR_yu12LDz_Ef-WMcs04W2bWbP6rlZeYmaxYqBiiIW20ssvcApAq5YfDeyGXDlfLIUxzxbHNUtVsjsqOYyzBCTOqR540EMmyUmMJzqdZ1nX349O0GQl8uekKvnFBRgTpy5Y5NwDshc1dP8rAnCetE3ms-QPtstVJnDNZvEUUyldCztdntzc-6ZrwppuWVtlo3MoDnkfTNIw83Jt5ploFngTFaj4ie8fdU328pr743BOexjAVPkkc3eO5jcgsgbIr5HKE4JIPVcm2fAExc6SHZFTMB1_QkxOv4_ZDsHZ-ef74YNh9ehs0y8Bs4Hmw6 |
| linkProvider | Scholars Portal |
| linkToHtml | http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwtV1Lb9QwEB6VRQIkhGh5dKFQg0DAISWx8zwgVB7VLu0WibZob8GOnVKpJMtmV4g_xW9kJnECQRVw6WEv8cTajMfjb2zPNwCPYh24nuLK0Ua5jh8EmZPE2nV0EsTal67mivYhJ_vh6Mh_Nw2mK_CjzYWha5WtT6wdtS4z2iPHIB1DD99P4uTl7KtDVaPodLUtodGYxa75_g1DturF-A2O72POd94evh45tqqAk4UJXziIEBBTewS1hSvz0BCvdx7TOhvI3EgRmEhFRGApQu1GCLm1SgwGFgGCJSVygf1egIu-cH3i6o-mXYCHviMMbXqeiLzn1hq2ZmVhthBKUQ-95a-uEtCtBYPZaVmdBXT_vK_52wK4cx2uWeTKthtTW4UVU6zBpYk9m1-Dq80OIGsSm9Zg1bqNij213NbPbsB0gh4EPW6hyfCZPR5ilt2VfemqrVVMFpopOaeKehVblCjT3gA37KRgbUonq-uOVDfh6FyG4RYMClTsOrA80ZmnpeR-nPghVypDS1HKSzL8ccmHIFqdp5llPqcCHKdpfZwXYQTUqDClkUrtSA3B6d6aNcwf_5B_RcPZyRJvd_2gnB-n1g2knuSIr4RR2hAvEII1T2iO300sOULKIWySMaRNEmznfdLtQCSeT2h3CA9rCeLuKOhy0LFcVlU6fv_xP4QOPvSEnlihvER1ZNImZOA3ESdYT3KjJ4keKOs1r5Pptlqp0l9zFd9szfns5gddM3VKF_4KUy5rGYwUiE5uCLcb6-80yzFmodPfIUS9edFTfb-lOPlcU6cHSRCJMLzz97-1CZdHh5O9dG-8v3sXriBGjunCiBdtwGAxX5p7iEMX6n49-Rl8Om9v8xMTQJiD |
| linkToPdf | http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwtV1Lb9QwEB6VRaqQEKLLowuFGgQCDmkTO88DQoVSdSlbEFC0t2DHTqlUkmWzK8Rf49cxkziBoAq49LCXeGJtxuPxN_b4G4AHsQ5cT3HlaKNcxw-CzEli7To6CWLtS1dzRfuQk8Nw_8h_NQ2mK_CjvQtDaZWtT6wdtS4z2iPHIB1DD99P4mQ7t2kRb3f3ns2-OlRBik5a23IajYkcmO_fMHyrno53cawfcr738sOLfcdWGHCyMOELB9EC4muPYLdwZR4a4vjOY1pzA5kbKQITqYjILEWo3Qjht1aJwSAjQOCkRC6w3wtwMRK-oHSyaNoFe-hHwtBe1RORt20tY2tWFmYLYRX10FsK64oB3bowmJ2W1Vmg98_czd8Ww72rcMWiWLbTmN0arJhiCKsTe04_hMvNbiBrLjkNYc26kIo9tjzXT67BdILeBL1voWkSMHtUxCzTK_vSVV6rmCw0U3JO1fUqtihRps0GN-ykYO31TlbXIKmuw9G5DMMNGBSo2HVgeaIzT0vJ_TjxQ65UhlajlJdk-OOSj0C0Ok8zy4JOxThO0_poL8JoqFFhSiOV2pEagdO9NWtYQP4h_5yGs5MlDu_6QTk_Tq1LSD3JEWsJo7QhjiAEbp7QHL-bGHOElCPYJGNImwuxnSdKdwKReD4h3xHcryWIx6OgGXEsl1WVjt98_A-h9-96Qo-sUF6iOjJpL2fgNxE_WE9yoyeJ3ijrNa-T6bZaqdJf8xbfbM357OZ7XTN1Ssl_hSmXtQxGDUQtN4KbjfV3muUYv9BJ8Aii3rzoqb7fUpx8rmnUgySIRBje-vvf2oRV9DPp6_HhwW24hHA5ptwRL9qAwWK-NHcQki7U3XruM_h03s7mJ3_7nL4 |
| openUrl | ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=Mitochondrial+disease+patient+motivations+and+barriers+to+participate+in+clinical+trials&rft.jtitle=PloS+one&rft.au=Zolkipli-Cunningham%2C+Zarazuela&rft.au=Xiao%2C+Rui&rft.au=Stoddart%2C+Amy&rft.au=McCormick%2C+Elizabeth+M&rft.date=2018-05-17&rft.issn=1932-6203&rft.eissn=1932-6203&rft.volume=13&rft.issue=5&rft.spage=e0197513&rft_id=info:doi/10.1371%2Fjournal.pone.0197513&rft.externalDBID=NO_FULL_TEXT |
| thumbnail_l | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=1932-6203&client=summon |
| thumbnail_m | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=1932-6203&client=summon |
| thumbnail_s | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=1932-6203&client=summon |