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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 April 2022 |
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Main ID: |
NCT01976091 |
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Date of registration:
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24/07/2013 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
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Scientific title:
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Phase I/IIA Gene Transfer Clinical Trial for LGMD2D (Alpha-Sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA |
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Date of first enrolment:
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February 1, 2015 |
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Target sample size:
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6 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01976091 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Medical Director |
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Address:
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Telephone:
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Email:
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Affiliation:
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Sarepta Therapeutics, Inc. |
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Key inclusion & exclusion criteria
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Key Inclusion Criteria:
- Cohort 1A must be adult and wheelchair-dependent; Cohorts 1B and 2 will be
participants of age 7 or older.
- Confirmed alpha-sarcoglycan deficiency or identified sarcoglycan alpha (SGCA)
deoxyribonucleic acid (DNA) mutation.
- Participants enrolled in Cohorts 1B or 2 must be able to walk independently, but must
exhibit signs of lower extremity weakness (that is, a Gowers' sign, use a handrail for
climbing stairs) and walk =80% of predicted distance on the 6 minute walk test (6MWT)
based on normative data.
Key Exclusion Criteria:
- Active viral infection based on clinical observations.
- The presence of SGCA mutations without weakness or loss of function.
- Symptoms or signs of cardiomyopathy.
- Serological evidence of human immunodeficiency virus (HIV), Hepatitis B, or C
infection.
- Diagnosis of (or ongoing treatment for) an autoimmune disease.
- Participants with AAVrh74 or AAV8 binding antibody titers = 1:50 as determined by
enzyme-linked immunosorbent assay (ELISA) immunoassay.
Other inclusion/exclusion criteria apply.
Age minimum:
7 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Limb-Girdle Muscular Dystrophy, Type 2D
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Intervention(s)
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Genetic: SRP-9004
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Primary Outcome(s)
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Number of Participants With Adverse Events (AEs).
[Time Frame: Up to 2 Years]
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Secondary Outcome(s)
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Change From Baseline of the Distance Walked in 6 Minutes (6MWT)
[Time Frame: Baseline up to 2 Years]
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Secondary ID(s)
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5U01AR060911
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9004-101
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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