Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A
Part of paid clinical trials in Columbus, Ohio.
- Sponsor
- Nationwide Children's Hospital
- Study ID
- NCT03520751
- Phase
- PHASE1/PHASE2
- Status
- Not Yet Recruiting
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Conditions
- Charcot-Marie-Tooth Neuropathy Type 1A
Eligibility Criteria
- Sex
- ALL
- Age
- 18 Years - 35 Years
- Healthy Volunteers
- Not accepted
Interventions
- scAAV1.tMCK.NTF3 — DRUGgene vector
Study Details
This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication. Three subjects ages 18 to 35 years receiving (8.87e11 vg/kg) will be enrolled.
Key Dates
- Start date
- Apr 30, 2027
- Status verified
- Jun 2025
- Primary completion
- Apr 30, 2029
- Completion
- Apr 30, 2030
Study Design
- Enrollment
- 3 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: Dose (8.87e11 vg/kg)Three patients age 18-35 will receive intramuscular injection of recombinant AAV1 carrying a human NFT3 gene under the control of the tMCK promoter (scAAV1.tMCK.NTF3) distributed bilaterally between both limbs at a dose of 8.87e11 vg/kg.
Primary Outcome Measure
Safety based on number of participants with adverse events. [ Time Frame: 2 years ]
Central Contacts
- Dawn Scott(614)722-2715
Locations (1)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 |
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