At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 3 enrolled
Drug / intervention
scAAV9.U7.ACCAbiological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular Dystrophy
In Brief
A Phase 2 clinical trial evaluating scAAV9.U7.ACCA for Duchenne Muscular Dystrophy. Completed, enrolled 3 participants across 1 site.
Detailed Summary
Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDuchenne Muscular Dystrophy
CountriesUnited States
CollaboratorsAudentes Therapeutics, Astellas Pharma Inc
Timeline
Phase 2CompletedFinished
2020202120222023202420252026
Enrollment StartJan 2020
First PostedJan 2020
Primary CompletionNov 2023
Study CompletionJul 2025
TodayJul 2026
First PostedJan 27, 2020
Enrollment StartJan 15, 2020
Primary CompletionNov 13, 2023
Study CompletionJul 1, 2025
TodayJul 2, 2026
Enrollment to primary: 3.8 yearsPosted 6.4 years ago
Interventions
scAAV9.U7.ACCAbiological
A single dose of scAAV9.U7.ACCA will be systemically delivered via a peripheral vein injection.