Limb Girdle Muscular Dystrophy Clinical Trial
Official title:
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)
Verified date | November 2023 |
Source | Sarepta Therapeutics, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Active, not recruiting |
Enrollment | 2 |
Est. completion date | August 31, 2028 |
Est. primary completion date | August 31, 2028 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 50 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
United States | Nationwide Children's Hospital | Columbus | Ohio |
Lead Sponsor | Collaborator |
---|---|
Sarepta Therapeutics, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs) | Baseline up to Month 60 | ||
Secondary | Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Western Blot | Baseline, Day 90 and Month 24 | ||
Secondary | Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Immunofluorescence (IF) Fiber Intensity | Baseline, Day 90 and Month 24 | ||
Secondary | 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 24 |
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