Trial Search Results

Study of SRP-4045 and SRP-4053 in DMD Patients

The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

Stanford is currently accepting patients for this trial.

Lead Sponsor:

Sarepta Therapeutics, Inc.

Intervention(s):

  • Drug: SRP-4045
  • Drug: SRP-4053
  • Drug: Placebo

Phase:

Phase 3

Eligibility


Inclusion Criteria:

   - Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53
   skipping

   - Stable dose of oral corticosteroids for at least 24 weeks

   - Intact right and left biceps or 2 alternative upper muscle groups

   - Mean 6MWT greater than or equal 300 meters and less than or equal to 450 meters

   - Stable pulmonary function: forced vital capacity (FVC) equal to or greater than 50%
   predicted

Exclusion Criteria:

   - Treatment with gene therapy at any time

   - Previous treatment with SMT C1100, PRO045 (BMN 045), PRO053 (BMN 053) or PRO051 (BMN
   051) within 24 weeks prior to Week 1

   - Current or previous treatment with any other experimental treatment (other than
   deflazacort) within 12 weeks prior to Week 1

   - Major surgery within 3 months prior to Week 1

   - Presence of other clinically significant illness

Other inclusion/exclusion criteria may apply

Ages Eligible for Study

7 Years - 13 Years

Genders Eligible for Study

Male

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Recruiting