Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS). TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE. Patients entering the OLE continued...
Saved in:
| Published in | Genetics in medicine Vol. 26; no. 7; p. 101138 |
|---|---|
| Main Authors | , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Elsevier Inc
01.07.2024
|
| Subjects | |
| Online Access | Get full text |
| ISSN | 1098-3600 1530-0366 1530-0366 |
| DOI | 10.1016/j.gim.2024.101138 |
Cover
| Abstract | Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS).
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE. Patients entering the OLE continued elamipretide 40 mg subcutaneous daily. OLE primary endpoints were safety and tolerability; secondary endpoints included change from baseline in the 6-minute walk test (6MWT) and BarTH Syndrome Symptom Assessment (BTHS-SA) Total Fatigue score. Muscle strength, physician- and patient-assessed outcomes, echocardiographic parameters, and biomarkers, including cardiolipin (CL) and monolysocardiolipin (MLCL), were assessed.
Ten patients entered the OLE; 8 reached the week 168 visit. Elamipretide was well tolerated, with injection-site reactions being the most common adverse events. Significant improvements from OLE baseline on 6MWT occurred at all OLE time points (cumulative 96.1 m of improvement [week 168, P = .003]). Mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE time points. Three-dimensional (3D) left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to week 168. MLCL/CL values showed improvement, correlating to important clinical outcomes.
Elamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS. |
|---|---|
| AbstractList | Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS).PURPOSEEvaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS).TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE. Patients entering the OLE continued elamipretide 40 mg subcutaneous daily. OLE primary endpoints were safety and tolerability; secondary endpoints included change from baseline in the 6-minute walk test (6MWT) and BarTH Syndrome Symptom Assessment (BTHS-SA) Total Fatigue score. Muscle strength, physician- and patient-assessed outcomes, echocardiographic parameters, and biomarkers, including cardiolipin (CL) and monolysocardiolipin (MLCL), were assessed.METHODSTAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE. Patients entering the OLE continued elamipretide 40 mg subcutaneous daily. OLE primary endpoints were safety and tolerability; secondary endpoints included change from baseline in the 6-minute walk test (6MWT) and BarTH Syndrome Symptom Assessment (BTHS-SA) Total Fatigue score. Muscle strength, physician- and patient-assessed outcomes, echocardiographic parameters, and biomarkers, including cardiolipin (CL) and monolysocardiolipin (MLCL), were assessed.Ten patients entered the OLE; 8 reached the week 168 visit. Elamipretide was well tolerated, with injection-site reactions being the most common adverse events. Significant improvements from OLE baseline on 6MWT occurred at all OLE time points (cumulative 96.1 m of improvement [week 168, P = .003]). Mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE time points. Three-dimensional (3D) left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to week 168. MLCL/CL values showed improvement, correlating to important clinical outcomes.RESULTSTen patients entered the OLE; 8 reached the week 168 visit. Elamipretide was well tolerated, with injection-site reactions being the most common adverse events. Significant improvements from OLE baseline on 6MWT occurred at all OLE time points (cumulative 96.1 m of improvement [week 168, P = .003]). Mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE time points. Three-dimensional (3D) left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to week 168. MLCL/CL values showed improvement, correlating to important clinical outcomes.Elamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS.CONCLUSIONElamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS. Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS). TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE. Patients entering the OLE continued elamipretide 40 mg subcutaneous daily. OLE primary endpoints were safety and tolerability; secondary endpoints included change from baseline in the 6-minute walk test (6MWT) and BarTH Syndrome Symptom Assessment (BTHS-SA) Total Fatigue score. Muscle strength, physician- and patient-assessed outcomes, echocardiographic parameters, and biomarkers, including cardiolipin (CL) and monolysocardiolipin (MLCL), were assessed. Ten patients entered the OLE; 8 reached the week 168 visit. Elamipretide was well tolerated, with injection-site reactions being the most common adverse events. Significant improvements from OLE baseline on 6MWT occurred at all OLE time points (cumulative 96.1 m of improvement [week 168, P = .003]). Mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE time points. Three-dimensional (3D) left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to week 168. MLCL/CL values showed improvement, correlating to important clinical outcomes. Elamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS. |
| ArticleNumber | 101138 |
| Author | Vaz, Frédéric M. Abbruscato, Anthony Manuel, Ryan Campbell, John Carr, Jim Vernon, Hilary J. Thompson, William R. Hornby, Brittany |
| Author_xml | – sequence: 1 givenname: William R. surname: Thompson fullname: Thompson, William R. organization: The Blalock-Taussig-Thomas Pediatric and Congenital Heart Center, Johns Hopkins University School of Medicine, Baltimore, MD – sequence: 2 givenname: Ryan surname: Manuel fullname: Manuel, Ryan organization: Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD – sequence: 3 givenname: Anthony surname: Abbruscato fullname: Abbruscato, Anthony organization: Stealth BioTherapeutics, Inc., Newton, MA – sequence: 4 givenname: Jim surname: Carr fullname: Carr, Jim organization: Stealth BioTherapeutics, Inc., Newton, MA – sequence: 5 givenname: John surname: Campbell fullname: Campbell, John organization: Stealth BioTherapeutics, Inc., Newton, MA – sequence: 6 givenname: Brittany surname: Hornby fullname: Hornby, Brittany organization: Department of Physical Therapy, Kennedy Krieger, Baltimore, MD – sequence: 7 givenname: Frédéric M. surname: Vaz fullname: Vaz, Frédéric M. organization: Amsterdam UMC Location University of Amsterdam, Department of Clinical Chemistry and Pediatrics, Laboratory Genetic Metabolic Diseases, Emma Children’s Hospital, Meibergdreef 9, Amsterdam, The Netherlands – sequence: 8 givenname: Hilary J. orcidid: 0000-0001-9940-9866 surname: Vernon fullname: Vernon, Hilary J. email: hvernon1@jhmi.edu organization: Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/38602181$$D View this record in MEDLINE/PubMed |
| BookMark | eNp9kUtv1DAUhS3Uij7gB7BBXrLJcB0ncQZWpSoPaaQiVFSpG8uxr4uHxE5tD2V2_HQ8mnbTRTd-SOc7i_OdkAMfPBLyhsGCAeverxe3blrUUDe7P-P9C3LMWg4V8K47KG9Y9hXvAI7ISUprACZ4DS_JEe87qFnPjsm_VfC3VcY4UbTWaaW3VHlDk7KYtzRYiqOa3BwxO4PUeTqr7NDnRO9d_kU_qVjOtPUmhgk_UNb11T3ibxpm9NWoBhwp_s3okwueRkybsaCl9urs5vvl9cWPV-TQqjHh64f7lPz8fHF1_rVaXX75dn62qjRvea40cjDKMGwFb1grRFdzFLUx_RIapoTuB9P1wwAApmZWWG0Yb5RuTW1N01l-St7te-cY7jaYspxc0jiOymPYJMmBC75sRd2U6NuH6GaY0Mg5uknFrXxcrQTEPqBjSCmildrlMkvwOSo3SgZy50euZfEjd37k3k8h2RPysfw55uOewTLPH4dRJl0MaDQuos7SBPcM_R8zcKfm |
| CitedBy_id | crossref_primary_10_1038_s41392_024_02044_3 crossref_primary_10_1016_j_jlr_2025_100750 crossref_primary_10_1093_ehjcr_ytaf030 crossref_primary_10_1002_ccr3_9116 crossref_primary_10_1093_ajhp_zxae242 crossref_primary_10_3390_ijms26030944 |
| Cites_doi | 10.1038/ng0496-385 10.2217/fca-2023-0008 10.1681/ASN.2012121216 10.1002/jimd.12453 10.1161/CIRCHEARTFAILURE.113.000406 10.1097/FJC.0000000000000155 10.1007/s10741-020-10031-3 10.1002/jimd.12425 10.14814/phy2.14215 10.1111/bph.12461 10.1186/s13023-022-02469-5 10.1097/01823246-201324030-00004 10.3389/fcvm.2019.00167 10.1016/j.jbc.2021.101005 10.1016/j.jpeds.2019.09.074 10.1007/s00246-022-02846-8 10.1038/s41436-020-01006-8 10.1186/1750-1172-8-23 10.1164/rccm.202208-1547OC 10.1038/s42003-020-1101-3 10.1164/rccm.201212-2191ED 10.1186/s13023-019-1200-8 10.1016/j.gene.2019.144148 10.1086/514886 10.1093/ehjqcco/qcx031 10.1373/clinchem.2007.095711 10.1007/s10557-018-6805-y 10.1074/jbc.RA119.012094 10.1212/WNL.0000000000005255 10.1002/jcsm.12559 10.1007/s10741-021-10177-8 10.1007/s00246-015-1260-z 10.1186/1750-1172-8-70 10.1161/CIRCHEARTFAILURE.115.002206 10.1074/jbc.M606100200 |
| ContentType | Journal Article |
| Copyright | 2024 The Authors Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved. |
| Copyright_xml | – notice: 2024 The Authors – notice: Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved. |
| DBID | 6I. AAFTH AAYXX CITATION NPM 7X8 |
| DOI | 10.1016/j.gim.2024.101138 |
| DatabaseName | ScienceDirect Open Access Titles Elsevier:ScienceDirect:Open Access CrossRef PubMed MEDLINE - Academic |
| DatabaseTitle | CrossRef PubMed MEDLINE - Academic |
| DatabaseTitleList | MEDLINE - Academic PubMed |
| Database_xml | – sequence: 1 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 |
| DeliveryMethod | fulltext_linktorsrc |
| Discipline | Medicine Biology |
| EISSN | 1530-0366 |
| ExternalDocumentID | 38602181 10_1016_j_gim_2024_101138 S1098360024000716 |
| Genre | Journal Article |
| GroupedDBID | --- .-D ..I .GJ 08G 0SF 39C 3V. 4Q1 4Q2 4Q3 53G 5GY 5RE 5VS 6I. 70F 7X7 88E 8FI 8FJ AAFTH AAKAS AAWBL AAXUO AAYEP AAZLF ABAWZ ABJNI ABLJU ABUWG ACBMV ACBRV ACBYP ACGFO ACGFS ACIGE ACKTT ACRQY ACTTH ACVWB ACZOJ ADBBV ADBIZ ADHDB ADMDM ADZCM AE3 AEFTE AEJRE AENEX AEXYK AFKRA AFSHS AFTRI AGAYW AGEZK AGGBP AHMBA AHSBF AHVBC AILAN AITUG AIZYK AJDOV AJRNO AKRWK ALFFA ALIPV ALMA_UNASSIGNED_HOLDINGS AMRAJ AWKKM AXYYD BENPR BKKNO BPHCQ BS7 BVXVI CCPQU CS3 DNIVK DU5 EBS EE. EIOEI EJD EX3 F5P FDB FDQFY FERAY FIZPM FSGXE FYUFA H0~ HMCUK JF9 JG8 JK3 JSO K-O KD2 M1P M41 N9A NAO NQJWS N~M OAG OAH ODA OK1 OLG OVD OWU OWV OWW OWX OWY OWZ P-K P2P PQQKQ PROAC PSQYO R58 RNT RNTTT ROL S4R SNX SNYQT SOHCF SOJ SRMVM SWTZT T8P TAOOD TBHMF TDRGL TEORI TSG UKHRP VVN W3M WOQ WOW XXN XYM YFH ZFV AAFWJ AALRI AAYWO AAYXX ACVFH ADCNI ADVLN AEUPX AFETI AFJKZ AFPUW AGCQF AIGII AKBMS AKYEP APXCP CITATION PHGZM PHGZT EFKBS NPM 7X8 EBLON |
| ID | FETCH-LOGICAL-c353t-ce30dad1e57341577623e72dd89041a7c8bd68bb000d21f7fcd134ac5d2fd46f3 |
| ISSN | 1098-3600 1530-0366 |
| IngestDate | Sat Sep 27 17:12:32 EDT 2025 Mon Jul 21 06:00:13 EDT 2025 Thu Apr 24 23:04:25 EDT 2025 Tue Jul 01 02:59:24 EDT 2025 Sat Jun 29 15:30:49 EDT 2024 |
| IsDoiOpenAccess | true |
| IsOpenAccess | true |
| IsPeerReviewed | true |
| IsScholarly | true |
| Issue | 7 |
| Keywords | Barth syndrome Cardiolipin Elamipretide Cardiomyopathy Mitochondrial disease |
| Language | English |
| License | This is an open access article under the CC BY-NC-ND license. Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved. |
| LinkModel | OpenURL |
| MergedId | FETCHMERGED-LOGICAL-c353t-ce30dad1e57341577623e72dd89041a7c8bd68bb000d21f7fcd134ac5d2fd46f3 |
| Notes | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 ObjectType-Undefined-3 |
| ORCID | 0000-0001-9940-9866 |
| OpenAccessLink | https://dx.doi.org/10.1016/j.gim.2024.101138 |
| PMID | 38602181 |
| PQID | 3037395724 |
| PQPubID | 23479 |
| ParticipantIDs | proquest_miscellaneous_3037395724 pubmed_primary_38602181 crossref_citationtrail_10_1016_j_gim_2024_101138 crossref_primary_10_1016_j_gim_2024_101138 elsevier_sciencedirect_doi_10_1016_j_gim_2024_101138 |
| ProviderPackageCode | CITATION AAYXX |
| PublicationCentury | 2000 |
| PublicationDate | 2024-07-01 |
| PublicationDateYYYYMMDD | 2024-07-01 |
| PublicationDate_xml | – month: 07 year: 2024 text: 2024-07-01 day: 01 |
| PublicationDecade | 2020 |
| PublicationPlace | United States |
| PublicationPlace_xml | – name: United States |
| PublicationTitle | Genetics in medicine |
| PublicationTitleAlternate | Genet Med |
| PublicationYear | 2024 |
| Publisher | Elsevier Inc |
| Publisher_xml | – name: Elsevier Inc |
| References | Machiraju, Wang, Sabouny (bib24) 2019; 6 Sabbah, Gupta, Szekely (bib34) 2014; 130 Sabbah, Taylor, Vernon (bib11) 2023; 19 Clarke, Bowron, Gonzalez (bib4) 2013; 8 Bione, D’Adamo, Maestrini, Gedeon, Bolhuis, Toniolo (bib8) 1996; 12 Anzmann, Sniezek, Pado (bib16) 2021; 297 Kulik, van Lenthe, Stet (bib36) 2008; 54 Moutchia, McClelland, Al-Naamani (bib32) 2023; 207 Thompson, Jefferies, Wang (bib9) 2022; 45 Mazar, Stokes, Ollis (bib3) 2019; 14 Reid Thompson, Hornby, Manuel (bib27) 2021; 23 D’Adamo, Fassone, Gedeon (bib7) 1997; 61 Sabbah (bib13) 2021; 26 Dai, Hsieh, Chen (bib21) 2013; 6 Allen, Pennington, Perry (bib25) 2020; 3 Braun, Hamstra, Messner, Fajardo (bib17) 2019; 7 Kang, Forsey, Dudley, Steward, Tsai-Goodman (bib33) 2016; 37 Szeto (bib20) 2014; 171 Birk, Liu, Soong (bib23) 2013; 24 Xu, Malhotra, Ren, Schlame (bib15) 2006; 281 Rigaud, Lebre, Touraine (bib6) 2013; 8 Miller, Ren, Schlame, Toth, Phoon (bib1) 2020; 217 Dai, Shi, Gupta, Sabbah, Hale, Kloner (bib22) 2014; 64 Vaz, van Lenthe, Vervaart (bib29) 2022; 45 Karaa, Haas, Goldstein, Vockley, Weaver, Cohen (bib37) 2018; 90 Ferreira, Thompson, Vernon (bib5) 1993 Sabbah, Gupta, Singh-Gupta, Zhang, Lanfear (bib35) 2018; 32 Chowdhury, Jackson, Byrne (bib2) 2022; 43 Hornby, Thompson, Almuqbil (bib28) 2022; 17 Bredy, Ministeri, Kempny (bib12) 2018; 4 bib39 Shoemaker, Curtis, Vangsnes, Dickinson (bib31) 2013; 24 Garlid, Schaffer, Kim, Bhatt, Guevara-Gonzalez, Ping (bib14) 2020; 726 Holland, Nici (bib30) 2013; 187 Sabbah (bib10) 2022; 27 Eirin, Ebrahimi, Kwon (bib18) 2016; 5 Sabbah, Gupta, Kohli, Wang, Hachem, Zhang (bib19) 2016; 9 Mitchell, Ng, Tamucci (bib26) 2020; 295 Karaa, Haas, Goldstein, Vockley, Cohen (bib38) 2020; 11 Xu (10.1016/j.gim.2024.101138_bib15) 2006; 281 Anzmann (10.1016/j.gim.2024.101138_bib16) 2021; 297 Rigaud (10.1016/j.gim.2024.101138_bib6) 2013; 8 Sabbah (10.1016/j.gim.2024.101138_bib13) 2021; 26 Clarke (10.1016/j.gim.2024.101138_bib4) 2013; 8 Eirin (10.1016/j.gim.2024.101138_bib18) 2016; 5 Birk (10.1016/j.gim.2024.101138_bib23) 2013; 24 Reid Thompson (10.1016/j.gim.2024.101138_bib27) 2021; 23 Moutchia (10.1016/j.gim.2024.101138_bib32) 2023; 207 Karaa (10.1016/j.gim.2024.101138_bib37) 2018; 90 Bione (10.1016/j.gim.2024.101138_bib8) 1996; 12 Kang (10.1016/j.gim.2024.101138_bib33) 2016; 37 Karaa (10.1016/j.gim.2024.101138_bib38) 2020; 11 Szeto (10.1016/j.gim.2024.101138_bib20) 2014; 171 Mitchell (10.1016/j.gim.2024.101138_bib26) 2020; 295 Sabbah (10.1016/j.gim.2024.101138_bib34) 2014; 130 Ferreira (10.1016/j.gim.2024.101138_bib5) 1993 Mazar (10.1016/j.gim.2024.101138_bib3) 2019; 14 Shoemaker (10.1016/j.gim.2024.101138_bib31) 2013; 24 Thompson (10.1016/j.gim.2024.101138_bib9) 2022; 45 Dai (10.1016/j.gim.2024.101138_bib22) 2014; 64 Sabbah (10.1016/j.gim.2024.101138_bib10) 2022; 27 Miller (10.1016/j.gim.2024.101138_bib1) 2020; 217 Sabbah (10.1016/j.gim.2024.101138_bib11) 2023; 19 Braun (10.1016/j.gim.2024.101138_bib17) 2019; 7 Allen (10.1016/j.gim.2024.101138_bib25) 2020; 3 Kulik (10.1016/j.gim.2024.101138_bib36) 2008; 54 Machiraju (10.1016/j.gim.2024.101138_bib24) 2019; 6 Sabbah (10.1016/j.gim.2024.101138_bib35) 2018; 32 Bredy (10.1016/j.gim.2024.101138_bib12) 2018; 4 Sabbah (10.1016/j.gim.2024.101138_bib19) 2016; 9 Hornby (10.1016/j.gim.2024.101138_bib28) 2022; 17 Vaz (10.1016/j.gim.2024.101138_bib29) 2022; 45 Holland (10.1016/j.gim.2024.101138_bib30) 2013; 187 Dai (10.1016/j.gim.2024.101138_bib21) 2013; 6 Chowdhury (10.1016/j.gim.2024.101138_bib2) 2022; 43 Garlid (10.1016/j.gim.2024.101138_bib14) 2020; 726 D’Adamo (10.1016/j.gim.2024.101138_bib7) 1997; 61 |
| References_xml | – volume: 171 start-page: 2029 year: 2014 end-page: 2050 ident: bib20 article-title: First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics publication-title: Br J Pharmacol – volume: 6 start-page: 1067 year: 2013 end-page: 1076 ident: bib21 article-title: Global proteomics and pathway analysis of pressure-overload-induced heart failure and its attenuation by mitochondrial-targeted peptides publication-title: Circ Heart Fail – volume: 14 start-page: 243 year: 2019 ident: bib3 article-title: Understanding the life experience of Barth syndrome from the perspective of adults: a qualitative one-on-one interview study publication-title: Orphanet J Rare Dis – volume: 9 year: 2016 ident: bib19 article-title: Chronic therapy with elamipretide (MTP-131), a novel mitochondria-targeting peptide, improves left ventricular and mitochondrial function in dogs with advanced heart failure publication-title: Circ Heart Fail – volume: 54 start-page: 371 year: 2008 end-page: 378 ident: bib36 article-title: Bloodspot assay using HPLC-tandem mass spectrometry for detection of Barth syndrome publication-title: Clin Chem – volume: 23 start-page: 471 year: 2021 end-page: 478 ident: bib27 article-title: A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism publication-title: Genet Med – volume: 3 start-page: 389 year: 2020 ident: bib25 article-title: The cardiolipin-binding peptide elamipretide mitigates fragmentation of cristae networks following cardiac ischemia reperfusion in rats publication-title: Commun Biol – volume: 11 start-page: 909 year: 2020 end-page: 918 ident: bib38 article-title: A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy publication-title: J Cachexia Sarcopenia Muscle – volume: 61 start-page: 862 year: 1997 end-page: 867 ident: bib7 article-title: The X-linked gene G4.5 is responsible for different infantile dilated cardiomyopathies publication-title: Am J Hum Genet – volume: 8 start-page: 23 year: 2013 ident: bib4 article-title: Barth syndrome publication-title: Orphanet J Rare Dis – volume: 17 start-page: 336 year: 2022 ident: bib28 article-title: Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome publication-title: Orphanet J Rare Dis – volume: 7 year: 2019 ident: bib17 article-title: SERCA2a tyrosine nitration coincides with impairments in maximal SERCA activity in left ventricles from tafazzin-deficient mice publication-title: Physiol Rep – volume: 45 start-page: 29 year: 2022 end-page: 37 ident: bib29 article-title: An improved functional assay in blood spot to diagnose Barth syndrome using the monolysocardiolipin/cardiolipin ratio publication-title: J Inherit Metab Dis – volume: 90 start-page: e1212 year: 2018 end-page: e1221 ident: bib37 article-title: Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy publication-title: Neurology – volume: 5 year: 2016 ident: bib18 article-title: Restoration of mitochondrial cardiolipin attenuates cardiac damage in swine renovascular hypertension publication-title: J Am Heart Assoc – volume: 726 year: 2020 ident: bib14 article-title: TAZ encodes tafazzin, a transacylase essential for cardiolipin formation and central to the etiology of Barth syndrome publication-title: Gene – volume: 187 start-page: 335 year: 2013 end-page: 336 ident: bib30 article-title: The return of the minimum clinically important difference for 6-minute-walk distance in chronic obstructive pulmonary disease publication-title: Am J Respir Crit Care Med – start-page: 1 year: 1993 end-page: 28 ident: bib5 article-title: Barth syndrome publication-title: GeneReviews – volume: 27 start-page: 1911 year: 2022 end-page: 1923 ident: bib10 article-title: Elamipretide for Barth syndrome cardiomyopathy: gradual rebuilding of a failed power grid publication-title: Heart Fail Rev – volume: 19 start-page: 211 year: 2023 end-page: 225 ident: bib11 article-title: Temporal evolution of the heart failure phenotype in Barth syndrome and treatment with elamipretide publication-title: Future Cardiol – volume: 4 start-page: 51 year: 2018 end-page: 58 ident: bib12 article-title: New York Heart Association (NYHA) classification in adults with congenital heart disease: relation to objective measures of exercise and outcome publication-title: Eur Heart J Qual Care Clin Outcomes – volume: 217 start-page: 139 year: 2020 end-page: 144 ident: bib1 article-title: A bayesian analysis to determine the prevalence of Barth syndrome in the pediatric population publication-title: J Pediatr – volume: 37 start-page: 167 year: 2016 end-page: 176 ident: bib33 article-title: Clinical characteristics and outcomes of cardiomyopathy in Barth syndrome: the UK experience publication-title: Pediatr Cardiol – volume: 32 start-page: 319 year: 2018 end-page: 328 ident: bib35 article-title: Abnormalities of mitochondrial dynamics in the failing heart: normalization following long-term therapy with elamipretide publication-title: Cardiovasc Drugs Ther – volume: 12 start-page: 385 year: 1996 end-page: 389 ident: bib8 article-title: A novel X-linked gene, G4.5. is responsible for Barth syndrome publication-title: Nat Genet – volume: 281 start-page: 39217 year: 2006 end-page: 39224 ident: bib15 article-title: The enzymatic function of tafazzin publication-title: J Biol Chem – volume: 24 start-page: 21 year: 2013 end-page: 29 ident: bib31 article-title: Clinically meaningful change estimates for the six-minute walk test and daily activity in individuals with chronic heart failure publication-title: Cardiopulm Phys Ther J – volume: 8 start-page: 70 year: 2013 ident: bib6 article-title: Natural history of Barth syndrome: a national cohort study of 22 patients publication-title: Orphanet J Rare Dis – volume: 45 start-page: 17 year: 2022 end-page: 28 ident: bib9 article-title: Current and future treatment approaches for Barth syndrome publication-title: J Inherit Metab Dis – volume: 26 start-page: 237 year: 2021 end-page: 253 ident: bib13 article-title: Barth syndrome cardiomyopathy: targeting the mitochondria with elamipretide publication-title: Heart Fail Rev – volume: 24 start-page: 1250 year: 2013 end-page: 1261 ident: bib23 article-title: The mitochondrial-targeted compound SS-31 re-energizes ischemic mitochondria by interacting with cardiolipin publication-title: J Am Soc Nephrol – volume: 207 start-page: 1070 year: 2023 end-page: 1079 ident: bib32 article-title: Minimal clinically important difference in the 6-minute-walk distance for patients with pulmonary arterial hypertension publication-title: Am J Respir Crit Care Med – volume: 43 start-page: 1251 year: 2022 end-page: 1263 ident: bib2 article-title: Longitudinal observational study of cardiac outcome risk factor prediction in children, adolescents, and adults with Barth syndrome publication-title: Pediatr Cardiol – volume: 295 start-page: 7452 year: 2020 end-page: 7469 ident: bib26 article-title: The mitochondria-targeted peptide SS-31 binds lipid bilayers and modulates surface electrostatics as a key component of its mechanism of action publication-title: J Biol Chem – ident: bib39 article-title: FDA guidance on conduct of clinical trials of medical products during the COVID-19 public health emergency: guidance for industry, investigators, and institutional review boards. Food and Drug Administration – volume: 297 year: 2021 ident: bib16 article-title: Diverse mitochondrial abnormalities in a new cellular model of TAFFAZZIN deficiency are remediated by cardiolipin-interacting small molecules publication-title: J Biol Chem – volume: 64 start-page: 543 year: 2014 end-page: 553 ident: bib22 article-title: Bendavia, a mitochondria-targeting peptide, improves postinfarction cardiac function, prevents adverse left ventricular remodeling, and restores mitochondria-related gene expression in rats publication-title: J Cardiovasc Pharmacol – volume: 6 start-page: 167 year: 2019 ident: bib24 article-title: SS-31 peptide reverses the mitochondrial fragmentation present in fibroblasts from patients with DCMA, a mitochondrial cardiomyopathy publication-title: Front Cardiovasc Med – volume: 130 year: 2014 ident: bib34 article-title: Bendavia (MTP-131), a mitochondria targeting peptide, normalizes dysregulation of mitochondria fission and fusion proteins in myocardium of dogs with chronic heart failure publication-title: Circulation – volume: 12 start-page: 385 issue: 4 year: 1996 ident: 10.1016/j.gim.2024.101138_bib8 article-title: A novel X-linked gene, G4.5. is responsible for Barth syndrome publication-title: Nat Genet doi: 10.1038/ng0496-385 – volume: 19 start-page: 211 issue: 4 year: 2023 ident: 10.1016/j.gim.2024.101138_bib11 article-title: Temporal evolution of the heart failure phenotype in Barth syndrome and treatment with elamipretide publication-title: Future Cardiol doi: 10.2217/fca-2023-0008 – volume: 24 start-page: 1250 issue: 8 year: 2013 ident: 10.1016/j.gim.2024.101138_bib23 article-title: The mitochondrial-targeted compound SS-31 re-energizes ischemic mitochondria by interacting with cardiolipin publication-title: J Am Soc Nephrol doi: 10.1681/ASN.2012121216 – volume: 45 start-page: 17 issue: 1 year: 2022 ident: 10.1016/j.gim.2024.101138_bib9 article-title: Current and future treatment approaches for Barth syndrome publication-title: J Inherit Metab Dis doi: 10.1002/jimd.12453 – volume: 6 start-page: 1067 issue: 5 year: 2013 ident: 10.1016/j.gim.2024.101138_bib21 article-title: Global proteomics and pathway analysis of pressure-overload-induced heart failure and its attenuation by mitochondrial-targeted peptides publication-title: Circ Heart Fail doi: 10.1161/CIRCHEARTFAILURE.113.000406 – volume: 64 start-page: 543 issue: 6 year: 2014 ident: 10.1016/j.gim.2024.101138_bib22 article-title: Bendavia, a mitochondria-targeting peptide, improves postinfarction cardiac function, prevents adverse left ventricular remodeling, and restores mitochondria-related gene expression in rats publication-title: J Cardiovasc Pharmacol doi: 10.1097/FJC.0000000000000155 – volume: 26 start-page: 237 issue: 2 year: 2021 ident: 10.1016/j.gim.2024.101138_bib13 article-title: Barth syndrome cardiomyopathy: targeting the mitochondria with elamipretide publication-title: Heart Fail Rev doi: 10.1007/s10741-020-10031-3 – volume: 45 start-page: 29 issue: 1 year: 2022 ident: 10.1016/j.gim.2024.101138_bib29 article-title: An improved functional assay in blood spot to diagnose Barth syndrome using the monolysocardiolipin/cardiolipin ratio publication-title: J Inherit Metab Dis doi: 10.1002/jimd.12425 – volume: 7 issue: 16 year: 2019 ident: 10.1016/j.gim.2024.101138_bib17 article-title: SERCA2a tyrosine nitration coincides with impairments in maximal SERCA activity in left ventricles from tafazzin-deficient mice publication-title: Physiol Rep doi: 10.14814/phy2.14215 – volume: 171 start-page: 2029 issue: 8 year: 2014 ident: 10.1016/j.gim.2024.101138_bib20 article-title: First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics publication-title: Br J Pharmacol doi: 10.1111/bph.12461 – volume: 17 start-page: 336 issue: 1 year: 2022 ident: 10.1016/j.gim.2024.101138_bib28 article-title: Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome publication-title: Orphanet J Rare Dis doi: 10.1186/s13023-022-02469-5 – volume: 24 start-page: 21 issue: 3 year: 2013 ident: 10.1016/j.gim.2024.101138_bib31 article-title: Clinically meaningful change estimates for the six-minute walk test and daily activity in individuals with chronic heart failure publication-title: Cardiopulm Phys Ther J doi: 10.1097/01823246-201324030-00004 – volume: 6 start-page: 167 year: 2019 ident: 10.1016/j.gim.2024.101138_bib24 article-title: SS-31 peptide reverses the mitochondrial fragmentation present in fibroblasts from patients with DCMA, a mitochondrial cardiomyopathy publication-title: Front Cardiovasc Med doi: 10.3389/fcvm.2019.00167 – volume: 297 issue: 3 year: 2021 ident: 10.1016/j.gim.2024.101138_bib16 article-title: Diverse mitochondrial abnormalities in a new cellular model of TAFFAZZIN deficiency are remediated by cardiolipin-interacting small molecules publication-title: J Biol Chem doi: 10.1016/j.jbc.2021.101005 – volume: 217 start-page: 139 year: 2020 ident: 10.1016/j.gim.2024.101138_bib1 article-title: A bayesian analysis to determine the prevalence of Barth syndrome in the pediatric population publication-title: J Pediatr doi: 10.1016/j.jpeds.2019.09.074 – volume: 43 start-page: 1251 issue: 6 year: 2022 ident: 10.1016/j.gim.2024.101138_bib2 article-title: Longitudinal observational study of cardiac outcome risk factor prediction in children, adolescents, and adults with Barth syndrome publication-title: Pediatr Cardiol doi: 10.1007/s00246-022-02846-8 – volume: 23 start-page: 471 issue: 3 year: 2021 ident: 10.1016/j.gim.2024.101138_bib27 article-title: A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism publication-title: Genet Med doi: 10.1038/s41436-020-01006-8 – volume: 8 start-page: 23 year: 2013 ident: 10.1016/j.gim.2024.101138_bib4 article-title: Barth syndrome publication-title: Orphanet J Rare Dis doi: 10.1186/1750-1172-8-23 – volume: 207 start-page: 1070 issue: 8 year: 2023 ident: 10.1016/j.gim.2024.101138_bib32 article-title: Minimal clinically important difference in the 6-minute-walk distance for patients with pulmonary arterial hypertension publication-title: Am J Respir Crit Care Med doi: 10.1164/rccm.202208-1547OC – volume: 3 start-page: 389 issue: 1 year: 2020 ident: 10.1016/j.gim.2024.101138_bib25 article-title: The cardiolipin-binding peptide elamipretide mitigates fragmentation of cristae networks following cardiac ischemia reperfusion in rats publication-title: Commun Biol doi: 10.1038/s42003-020-1101-3 – volume: 187 start-page: 335 issue: 4 year: 2013 ident: 10.1016/j.gim.2024.101138_bib30 article-title: The return of the minimum clinically important difference for 6-minute-walk distance in chronic obstructive pulmonary disease publication-title: Am J Respir Crit Care Med doi: 10.1164/rccm.201212-2191ED – volume: 14 start-page: 243 issue: 1 year: 2019 ident: 10.1016/j.gim.2024.101138_bib3 article-title: Understanding the life experience of Barth syndrome from the perspective of adults: a qualitative one-on-one interview study publication-title: Orphanet J Rare Dis doi: 10.1186/s13023-019-1200-8 – volume: 726 year: 2020 ident: 10.1016/j.gim.2024.101138_bib14 article-title: TAZ encodes tafazzin, a transacylase essential for cardiolipin formation and central to the etiology of Barth syndrome publication-title: Gene doi: 10.1016/j.gene.2019.144148 – volume: 61 start-page: 862 issue: 4 year: 1997 ident: 10.1016/j.gim.2024.101138_bib7 article-title: The X-linked gene G4.5 is responsible for different infantile dilated cardiomyopathies publication-title: Am J Hum Genet doi: 10.1086/514886 – volume: 4 start-page: 51 issue: 1 year: 2018 ident: 10.1016/j.gim.2024.101138_bib12 article-title: New York Heart Association (NYHA) classification in adults with congenital heart disease: relation to objective measures of exercise and outcome publication-title: Eur Heart J Qual Care Clin Outcomes doi: 10.1093/ehjqcco/qcx031 – volume: 130 issue: 2 year: 2014 ident: 10.1016/j.gim.2024.101138_bib34 article-title: Bendavia (MTP-131), a mitochondria targeting peptide, normalizes dysregulation of mitochondria fission and fusion proteins in myocardium of dogs with chronic heart failure publication-title: Circulation – volume: 54 start-page: 371 issue: 2 year: 2008 ident: 10.1016/j.gim.2024.101138_bib36 article-title: Bloodspot assay using HPLC-tandem mass spectrometry for detection of Barth syndrome publication-title: Clin Chem doi: 10.1373/clinchem.2007.095711 – volume: 32 start-page: 319 issue: 4 year: 2018 ident: 10.1016/j.gim.2024.101138_bib35 article-title: Abnormalities of mitochondrial dynamics in the failing heart: normalization following long-term therapy with elamipretide publication-title: Cardiovasc Drugs Ther doi: 10.1007/s10557-018-6805-y – volume: 295 start-page: 7452 issue: 21 year: 2020 ident: 10.1016/j.gim.2024.101138_bib26 article-title: The mitochondria-targeted peptide SS-31 binds lipid bilayers and modulates surface electrostatics as a key component of its mechanism of action publication-title: J Biol Chem doi: 10.1074/jbc.RA119.012094 – volume: 90 start-page: e1212 issue: 14 year: 2018 ident: 10.1016/j.gim.2024.101138_bib37 article-title: Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy publication-title: Neurology doi: 10.1212/WNL.0000000000005255 – volume: 11 start-page: 909 issue: 4 year: 2020 ident: 10.1016/j.gim.2024.101138_bib38 article-title: A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy publication-title: J Cachexia Sarcopenia Muscle doi: 10.1002/jcsm.12559 – volume: 5 issue: 6 year: 2016 ident: 10.1016/j.gim.2024.101138_bib18 article-title: Restoration of mitochondrial cardiolipin attenuates cardiac damage in swine renovascular hypertension publication-title: J Am Heart Assoc – volume: 27 start-page: 1911 issue: 5 year: 2022 ident: 10.1016/j.gim.2024.101138_bib10 article-title: Elamipretide for Barth syndrome cardiomyopathy: gradual rebuilding of a failed power grid publication-title: Heart Fail Rev doi: 10.1007/s10741-021-10177-8 – volume: 37 start-page: 167 issue: 1 year: 2016 ident: 10.1016/j.gim.2024.101138_bib33 article-title: Clinical characteristics and outcomes of cardiomyopathy in Barth syndrome: the UK experience publication-title: Pediatr Cardiol doi: 10.1007/s00246-015-1260-z – volume: 8 start-page: 70 year: 2013 ident: 10.1016/j.gim.2024.101138_bib6 article-title: Natural history of Barth syndrome: a national cohort study of 22 patients publication-title: Orphanet J Rare Dis doi: 10.1186/1750-1172-8-70 – volume: 9 issue: 2 year: 2016 ident: 10.1016/j.gim.2024.101138_bib19 article-title: Chronic therapy with elamipretide (MTP-131), a novel mitochondria-targeting peptide, improves left ventricular and mitochondrial function in dogs with advanced heart failure publication-title: Circ Heart Fail doi: 10.1161/CIRCHEARTFAILURE.115.002206 – start-page: 1 year: 1993 ident: 10.1016/j.gim.2024.101138_bib5 article-title: Barth syndrome – volume: 281 start-page: 39217 issue: 51 year: 2006 ident: 10.1016/j.gim.2024.101138_bib15 article-title: The enzymatic function of tafazzin publication-title: J Biol Chem doi: 10.1074/jbc.M606100200 |
| SSID | ssj0017320 |
| Score | 2.4939926 |
| Snippet | Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS).... Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome... |
| SourceID | proquest pubmed crossref elsevier |
| SourceType | Aggregation Database Index Database Enrichment Source Publisher |
| StartPage | 101138 |
| SubjectTerms | Barth syndrome Cardiolipin Cardiomyopathy Elamipretide Mitochondrial disease |
| Title | Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER |
| URI | https://dx.doi.org/10.1016/j.gim.2024.101138 https://www.ncbi.nlm.nih.gov/pubmed/38602181 https://www.proquest.com/docview/3037395724 |
| Volume | 26 |
| hasFullText | 1 |
| inHoldings | 1 |
| isFullTextHit | |
| isPrint | |
| journalDatabaseRights | – providerCode: PRVLSH databaseName: Elsevier Journals customDbUrl: mediaType: online eissn: 1530-0366 dateEnd: 99991231 omitProxy: true ssIdentifier: ssj0017320 issn: 1098-3600 databaseCode: AKRWK dateStart: 19981101 isFulltext: true providerName: Library Specific Holdings |
| link | http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1db9MwFLVKJ9BeEAwY5UtG4okqVeIkTspbmYamjQGqOm3iJXL8MXV0KWobofHEr-D3ch3bSSh0YrxEVZS4bu7pzfG91-ci9Irpju-5kp4YBkMvUsz3WC58b5hSRRWHFyDXG5yPP9CDk-jwLD7rdH62qpbKVT7g3_-6r-R_rArnwK56l-wNLFsPCifgM9gXjmBhOP6Tjd_Pi3NP-1ZdlgFPmxsxpSVT0hRaSLD3VJcUTkUlDmJVVO2WtrdM52ycZkEVG6Cp903KL33dVMsDgMhZvwqT65haH1bm5czUfkxGnz99PN0ft7mtVrCuRJ_hi9ZT9k3dyZ4TguiPB004vChNtcD4qkHrKM8X5ZKz1bylctDkTBYV1g6nl-3ABYnqIlfna7WUaUh9v-2MzfZ5C7qk5VnBdQRGB-YPp2_iDxeD86mWFiDRoLn2d4HttRdfXY7oKt0uMhgi00NkZohbaIsklJIu2hodjU-P6vxUEhKjc2F_gsuXV5WDa_PYxHg2rWgqZjO5h-7aJQkeGXzdRx1Z7KDbpknp1Q66c2xt-QD9qAGHHeAwAA4bwOG5wm3A4WmBHeCwBhyuAIcd4N5gBzfcwA3XcMMWbnpYB7eH6OTd_mTvwLMtPDwexuHK4zL0BROBjBOgS3ECr95QJkSIdOhHAUt4mgua5pqKChKoRHERhBHjsSBKRFSFj1C3mBfyMcIpcF9Y_BJKxDBSMWU8ZWEgc5XTlImU9pDvHnPGrb69brMyyzaat4de17d8NeIu110cOdtllp0a1pkBDq-77aWzcwaeW6fjWCHn5TID8lhlyUnUQ7sGAPUsQt0aDsj3k5vM8Cnabv5nz1B3tSjlc6DMq_yFxe8v1bHBaw |
| linkProvider | Library Specific Holdings |
| 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=Long-term+efficacy+and+safety+of+elamipretide+in+patients+with+Barth+syndrome%3A+168-week+open-label+extension+results+of+TAZPOWER&rft.jtitle=Genetics+in+medicine&rft.au=Thompson%2C+William+R.&rft.au=Manuel%2C+Ryan&rft.au=Abbruscato%2C+Anthony&rft.au=Carr%2C+Jim&rft.date=2024-07-01&rft.issn=1098-3600&rft.volume=26&rft.issue=7&rft.spage=101138&rft_id=info:doi/10.1016%2Fj.gim.2024.101138&rft.externalDBID=n%2Fa&rft.externalDocID=10_1016_j_gim_2024_101138 |
| thumbnail_l | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=1098-3600&client=summon |
| thumbnail_m | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=1098-3600&client=summon |
| thumbnail_s | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=1098-3600&client=summon |