DMD: pharmacological trials-2

  •  Essence : A Double-blind, Placebo-Controlled, Multicenter Study With and Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
  • SRP4658-302 : Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy
    Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
  • SRP 4856-102: An open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with DMD Amenable to Exon 51 Skipping
  • SRP 4856-102-OLE: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
  • SRP4658-402: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

 

Acronym

Essence

SRP4658-302

SRP 4658-102

SRP 4856-102-OLE

SRP4658-402

Study type BMR BMR BMR BMR BMR
Trial type International multicentre trial International multicentre trial
Intervention Casimersen, Golodirsen and placebo Casimersen, Golodirsen Étéplirsen Étéplirsen Étéplirsen
Principal investigator
Andreea Seferian Andreea Seferian Andreea Seferian Andreea Seferian Andreea Seferian
Sponsor SAREPTA SAREPTA SAREPTA SAREPTA SAREPTA
Fundings SAREPTA SAREPTA SAREPTA SAREPTA SAREPTA
Study status Ongoing In preparation Ongoing Ongoing In preparation
Recruitment status Ongoing Ongoing Ongoing
Population Child Child Child Child Child
+ infos on clinicaltrials.org + infos on clinicaltrials.gov