Open access

Research Article

30 January 2020

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

Authors: Eugenio Mercuri [email protected], Francesco Muntoni, Andrés Nascimento Osorio, Már Tulinius, Filippo Buccella, Lauren P Morgenroth, Heather Gordish-Dressman, … Show All … , Joel Jiang, Panayiota Trifillis, Jin Zhu, Allan Kristensen, Claudio L Santos, Erik K Henricson, Craig M McDonald, Isabelle Desguerre, and on behalf of the STRIDE CINRG Duchenne Natural History InvestigatorsAuthor Info & Affiliations

Publication: Journal of Comparative Effectiveness Research

Volume 9, Number 5

https://doi.org/10.2217/cer-2019-0171

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Abstract

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.

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