Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of...

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Published in	Journal of comparative effectiveness research Vol. 9; no. 5; pp. 341 - 360
Main Authors	Mercuri, Eugenio, Muntoni, Francesco, Osorio, Andrés Nascimento, Tulinius, Már, Buccella, Filippo, Morgenroth, Lauren P, Gordish-Dressman, Heather, Jiang, Joel, Trifillis, Panayiota, Zhu, Jin, Kristensen, Allan, Santos, Claudio L, Henricson, Erik K, McDonald, Craig M, Desguerre, Isabelle
Format	Journal Article
Language	English
Published	England Future Medicine Ltd 01.04.2020
Subjects	Age ataluren Blood pressure Clinical trials Codon, Nonsense - genetics Data collection dystrophin Dystrophin - genetics effectiveness Enrollments Humans Hypertension Medical laboratories Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - genetics Mutation Neurosciences Neurovetenskaper nonsense mutation Duchenne muscular dystrophy Oxadiazoles - adverse effects Oxadiazoles - therapeutic use Patients Proteins Registries safety STRIDE Registry Studies Treatment Outcome effectiveness nonsense mutation Duchenne muscular dystrophy dystrophin STRIDE Registry safety ataluren
Online Access	Get full text
ISSN	2042-6305 2042-6313 2042-6313
DOI	10.2217/cer-2019-0171

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Abstract	Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype–phenotype/–ataluren benefit correlations and ataluren safety. Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Kaplan–Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype–phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. NCT02369731. NCT02369731.
AbstractList	Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. NCT02369731. NCT02369731. Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype–phenotype/–ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan–Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype–phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731. Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731. Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p <= 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731. Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype–phenotype/–ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan–Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype–phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
Author	Zhu, Jin Trifillis, Panayiota Muntoni, Francesco McDonald, Craig M Tulinius, Már Mercuri, Eugenio Kristensen, Allan Desguerre, Isabelle Buccella, Filippo Gordish-Dressman, Heather Henricson, Erik K Santos, Claudio L Osorio, Andrés Nascimento Morgenroth, Lauren P Jiang, Joel
AuthorAffiliation	3Dubowitz Neuromuscular Centre & MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, 30 Guildford Street, London, WC1N 1EH, UK 12APHP Necker – Enfants Malades Hospital, Paris V Descartes University, Neuromuscular Network FILNEMUS, Paris, France 10PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA 6Department of Pediatrics, Gothenburg University, Queen Silvia Children’s Hospital, Gothenburg, Sweden 8Therapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA 11University of California Davis School of Medicine, Davis, CA, USA 4NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK 5Hospital Sant Joan de Déu Unidad de Patología Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain 7Parent Project Italy APS, Rome, Italy 9Cen
AuthorAffiliation_xml	– name: 1Department of Pediatric Neurology, Catholic University, Rome, Italy – name: 6Department of Pediatrics, Gothenburg University, Queen Silvia Children’s Hospital, Gothenburg, Sweden – name: 3Dubowitz Neuromuscular Centre & MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, 30 Guildford Street, London, WC1N 1EH, UK – name: 2Centro Clinico Nemo, Policlinico Universitario A Gemelli IRCCS, Rome, Italy – name: 5Hospital Sant Joan de Déu Unidad de Patología Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain – name: 4NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK – name: 7Parent Project Italy APS, Rome, Italy – name: 10PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA – name: 11University of California Davis School of Medicine, Davis, CA, USA – name: 12APHP Necker – Enfants Malades Hospital, Paris V Descartes University, Neuromuscular Network FILNEMUS, Paris, France – name: 8Therapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA – name: 9Center for Genetic Medicine, Children’s National Health System & the George Washington, Washington, DC, USA
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Notes	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 14 content type line 23 Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study Investigators (sorted by country): Argentina: A Dubrovsky (Buenos Aires). Australia: A Kornberg, M Ryan (Melbourne, VIC); R Webster (Sydney, NSW). Canada: WD Biggar, LC McAdam (Toronto, ON); JK Mah (Calgary, AB); H Kolski (Edmonton, AB). India: V Vishwanathan, S Chidambaranathan (Chennai). Israel: Y Nevo (Jerusalem). Italy: K Gorni (Milan). Puerto Rico: J Carlo (San Juan). Sweden: M Tulinius (Göteborg). USA: CM McDonald, EK Henricson, RT Abresch, NC Joyce (Sacramento, CA); A Cnaan, LP Morgenroth, R Leshner, C Tesi-Rocha, M Thangarajh, T Duong (Washington, DC); PR Clemens, H Abdel-Hamid (Pittsburgh, PA); AM Connolly, A Pestronk (St Louis, MO); J Teasley, A Harper (Richmond, VA); TE Bertorini (Memphis, TN); N Kuntz, S Driscoll (Rochester, MN); JW Day, P Karachunski (Minneapolis, MN); T Lotze (Houston, TX). STRIDE Registry Investigators (sorted by country): Austria: G Bernert, M Gosk-Tomek, A Ille, A Kellersmann, S Weiss (Vienna); V Pilshofer (Linz). Czech Republic: Z Bálintová, P Danhofer, P Fabulová, L Juříková (Brno); P Fuchsová, J Haberlová (Prague). France: F Laffargue, C Sarret, B Pontier (Clermont Ferrand); R Bellance, E Sarrazin (Fort de France, Martinique); P Sabouraud (Reims); A Magot, S Mercier, Y Péréon (Nantes); J-M Cuisset, S Coopman-Degryse (former investigator) (Lille); E Enaud, M-L Jacquemont, A Perville, M Renouil (former investigator), V Trommsdorff, D Verheulpen (Saint-Pierre); S Fontaine-Carbonnel, C Vuillerot (Bron); S Peudenier, J Ropars (Brest); F Audic, B Chabrol (Marseille); S Chabrier, G Gousse (Saint-Etienne); E Lagrue (Tours); K Aragon (former investigator), C Barnerias, LV Brande, S De Lucia, I Desguerre, T Gidaro, A Seferian, L Servais (Paris); V Laugel (Strasbourg); C Espil-Taris (Bordeaux); H Mecili, E Raffo, S Ragot-Mandry (Vandoeuvre-lès-Nancy). Germany: S Borrell, J Kirschner (Freiburg); A Gangfuss, M Henrich, H Kölbel, U Schara, N Spönemann, E Temme (Essen); J Seeger (Frankfurt); A Hirsch (Fürth), J Denecke, J Johannsen, A Neu (Hamburg); D Osinski, S Rügner, S Schüssler, R Trollmann (Erlangen); A Kaindl, JB Schneider, C Stoltenburg, C Weiss (Berlin); G Schreiber (Kassel); A Hahn, M Grzybowski (Gießen). Greece: E Pavlidou, E Pavlou (Thessaloniki). Hungary: S Dobner, Z Liptai (Budapest). Israel: T Dor (Jerusalem). Italy: C Brogna, M Catteruccia, A D’Amico, E Mercuri, M Pane (Rome); L Bello, E Pegoraro, C Semplicini (Padua); E Albamonte, G Baranello, G Comi, A Govoni, A Lerario, F Magri, R Masson, E Mauri, V Sansone (Milan); C Brusa, T Mongini, F Ricci, M Vacchetti (Turin); C Bruno, C Paniucci, M Pedemonte (Genoa); M Giannotta, A Pini (Bologna); S Messina, M Sframeli, G Vita, G Vita (Messina); L Ruggiero, L Santoro (Naples). Romania: D Craiu (former investigator), C Motoescu, C Sandu, R Teleanu, D Vasile (Bucharest); Sweden: M Tulinius (Gothenburg). UK: I Hughes (Manchester), A-M Childs (Leeds), Z Alhaswani, H Roper (former investigator), D Parasuraman (Birmingham); C DeGoede (Preston); V Gowda, A Manzur, P Munot, A Sarkokzy (London); C Charlesworth, J Lemon (former investigator), L Turner (former investigator), S Spinty (Liverpool).
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Snippet	Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding... Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence... Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence...
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SubjectTerms	Age ataluren Blood pressure Clinical trials Codon, Nonsense - genetics Data collection dystrophin Dystrophin - genetics effectiveness Enrollments Humans Hypertension Medical laboratories Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - genetics Mutation Neurosciences Neurovetenskaper nonsense mutation Duchenne muscular dystrophy Oxadiazoles - adverse effects Oxadiazoles - therapeutic use Patients Proteins Registries safety STRIDE Registry Studies Treatment Outcome
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