NCT05906251TerminatedPhase 1
An Open-Label, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP 6004 Administered by Systemic Infusion in Ambulatory Subjects With Limb Girdle Muscular Dystrophy Type 2B/R2 (LGMD2B/R2, Dysferlin Related)
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 1
- Status
- Terminated
- Sponsor
- Sarepta Therapeutics, Inc.
- Enrollment
- 2
- Locations
- 1
- Primary Endpoint
- Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Study Overview
Brief Summary
The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 50 Years (Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic DYSF Deoxyribonucleic acid (DNA) gene mutations as documented prior to screening visits.
- •Participants must be ambulatory per protocol specified criteria.
- •Ability to cooperate with motor assessment testing.
- •Has accessible and intact lower and upper extremity musculature for biopsy.
- •Have adeno-associated virus rhesus serotype 74 (rAAVrh74) antibody titers < 1:400 (that is, not elevated) as determined by enzyme-linked immunosorbent assay (ELISA).
Exclusion Criteria
- •Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
- •Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- •Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
- •Note: Other inclusion or exclusion criteria could apply.
Arms & Interventions
SRP-6004
Experimental
Participants will receive single IV infusion of SRP-6004 on Day 1.
Intervention: SRP-6004 (Genetic)
Outcomes
Primary Outcomes
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Time Frame: Baseline up to Month 60
Secondary Outcomes
- Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Western Blot(Baseline, Day 90 and Month 15)
- Change from Baseline in Percent of Normal DYSF Protein Expression as Assessed by IF Percent DYSF Positive Fibers (PPF: DYSF)(Baseline, Day 90 and Month 15)
- Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Immunofluorescence (IF) Fiber Intensity(Baseline, Day 90 and Month 15)
Investigators
Study Sites (1)
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