DMD: pharmacological trials- 4

  • SPITFIRE (Anti-Myostatin): A Phase 2/3, Randomized,Parallel, Double-blind, Placebo-controlled, to assess the Efficacy, Safety and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy
  • SIDEROS : A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid steroids
  • SIDEROS E: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study
  • PTC-PASS : Long-Term Observational Study of Translarna safety and effectiveness in routine care

 

Acronym

SPITFIRE
(Anti-Myostatin)

SIDEROS

 

SIDEROS E

PTC-PASS

Study type RBM RBM RBM Observationnal
Trial type Multicentre trial International multicentre trial International multicentre trial
Intervention RO7239361 Idebenone Idebenone Ataluren
Principal investigator Odile Boespflug-Tanguy Odile Boespflug-Tanguy Odile Boespflug-Tanguy Silvana De Lucia
Role Investigator Investigator Investigator Investigator
Sponsor ROCHE SANTHERA SANTHERA PTC
Fundings ROCHE SANTHERA SANTHERA PTC
Study status Closed Ongoing Ongoing Ongoing
Recruitment status Closed Ongoing Ongoing Ongoing
Population Child Child Child Child / Adult
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