A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)
NCT ID: NCT05906251
Last Updated: 2025-09-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE1
2 participants
INTERVENTIONAL
2023-05-22
2025-06-13
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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SRP-6004
Participants will receive single IV infusion of SRP-6004 on Day 1.
SRP-6004
Single IV infusion of SRP-6004
Interventions
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SRP-6004
Single IV infusion of SRP-6004
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
18 Years
50 Years
ALL
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Sarepta Therapeutics, Inc.
Locations
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Nationwide Children's Hospital
Columbus, Ohio, United States
Countries
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Other Identifiers
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SRP-6004-102
Identifier Type: -
Identifier Source: org_study_id
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