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A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD)
study id #: NCT04626674
condition: Muscular Dystrophy, Duchenne
status: not yet recruitingpurpose:
This is an open-label gene delivery study evaluating the safety of and expression from SRP-9001 in individuals with Duchenne Muscular Dystrophy over 260 weeks.
mechanism of action: Gene therapy to introduce a version of dystrophin
last updated: November 18, 2020
start date: November 11, 2020
estimated completion: May 31, 2021
phase of development: Phase 1
size / enrollment: 10
- Part 1: Change From Baseline in Quantity of Micro-dystrophin Protein Expression Measured by Western Blot [ Time Frame: Baseline up to Week 12 ]
- Vector Shedding as Measured by Polymerase Chain Reaction in Urine, Saliva and Stool Samples Post-infusion [ Time Frame: Baseline up to Week 260 ]
- Immunogenicity of SRP-9001 as Assessed by Antibody Titers to rAAVrh74 [ Time Frame: Screening up to Week 260 ]
- Incidence of Treatment Emergent Adverse Events (TEAEs), Serious AEs and Adverse Events of Special Interest [ Time Frame: Baseline up to Week 260 ]
- Number of Participants with Clinically Significant Abnormalities in Vital Signs, Physical Examinations, Laboratory Parameters, Electrocardiograms (ECG) and Echocardiograms (ECHO) Findings [ Time Frame: Baseline up to Week 260 ]
• Eligible Sexes: male
• Definitive diagnosis of Duchenne Muscular Dystrophy based on documented clinical findings and prior genetic testing
• Ability to cooperate with motor assessment testing.
• Stable dose equivalent of oral corticosteroids for at least 12 weeks.
• rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
• Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
• Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
• Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
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