Clareo Health | 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
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
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Sarepta Therapeutics, Inc.
Age
4–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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)
