Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Lim… (NCT03652259) | Clinical Trial Compass
TerminatedPhase 1/2
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
Stopped: Study is being terminated due to a business decision.
United States6 participantsStarted 2018-10-27
Plain-language summary
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
Who can participate
Age range4 Years – 15 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
INCLUSION CRITERIA
* Males or females of any ethnic group
* β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles
* Weakness demonstrated based on history of difficulty in running, jumping and climbing stairs
* A 100 meter walk/run (MWR) test result: ≥40 % of predicted for age-, height-, gender-, and weight-matched healthy controls at the screening visit
EXCLUSION CRITERIA
* Active viral infection based on clinical observations
* Cardiac magnetic resonance imaging (MRI) determined left ventricular ejection fraction (LVEF) \<40%
* Serological evidence of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
* Diagnosis of (or ongoing treatment for) an autoimmune disease
* Abnormal laboratory values considered clinically significant
* Concomitant illness or requirement for chronic drug treatment that, in the opinion of the Principal Investigator, creates unnecessary risks for gene transfer.
Other inclusion/exclusion criteria apply.
What they're measuring
1
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)