DMD: pharmacological trials- 4

  • 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
  • U7 : A phase I/IIa, escalating dose, open label study to evaluate Safety, Tolerability and Efficacy of G1U007 locoregional injection in patients with Duchenne Muscular Dystrophy for exon 53 skipping
  • 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
  • PTC-PASS : Long-Term Observational Study of Translarna safety and effectiveness in routine care

 

Acronym

Anti-Myostatin

U7

SIDEROS

PTC-PASS

Study type RBM RBM RBM Observationnal
Trial type Multicentre trial Single-centre trial International multicentre trial International multicentre trial
Intervention BMS-986089 vs Placebo G1U007 Idebenone Ataluren
Principal investigator Teresa Gidaro Laurent Servais Teresa Gidaro Teresa Gidaro
Role Investigator Sponsor and Investigator Investigator Investigator
Sponsor BMS Généthon SANTHERA PTC
Fundings BMS Généthon SANTHERA PTC
Study status Ongoing In preparation Ongoing Ongoing
Recruitment status Ongoing In preparation Ongoing Ongoing
Population Child Child Child / Adult Child / Adult
Contact t.gidaro@institut-myologie.org l.servais@institut-myologie.org t.gidaro@institut-myologie.org t.gidaro@institut-myologie.org
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