A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

Plain-language summary

This multicenter, global Phase 3 study by Sarepta Therapeutics examines the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4). It includes both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2), with eligibility based on cohort-specific mobility test thresholds and the presence of 1 homozygous or 2 heterozygous changes. Participants also receive a glucocorticoid (prednisone or equivalent); the trial is active but no longer recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 3

Status

Active, not recruiting

Sponsor

Sarepta Therapeutics, Inc.

Intervention

SRP-9003 (scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec), Glucocorticoid (Prednisone or equivalent)

Start date

1/15/2024

Estimated completion

11/30/2029

Countries

United States, Belgium, Germany, Italy, Spain, United Kingdom

Locations

• La Jolla, United States
• Columbus, United States
• Philadelphia, United States
• Norfolk, United States
• Leuven, Belgium
• Ghent, Belgium
• Essen, Germany
• Milan, Italy
• Barcelona, Spain
• Newcastle upon Tyne, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Cohort 1, only ambulatory participants:

  * Able to walk without assistive aid
  * 10MWR \<30 seconds
  * NSAD ≥25
* Cohort 2, only non-ambulatory participants:

  * 10MWR ≥30 seconds or unable to perform
  * PUL 2.0 entry scale score ≥3
* Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
* Able to cooperate with muscle testing
* Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers \<1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.

Exclusion Criteria:

* Left ventricular ejection fraction \< 40% or clinical signs and/or symptoms of cardiomyopathy
* Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
* Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
* Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)

Other inclusion/exclusion criteria apply.