Clinical Trial

Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A

Withdrawn Phase 1/2
View on ClinicalTrials.gov →
Why the trial stopped
Study is withdrawing to the retirement of the PI.
Summary
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.
Protocol Amendment History 3 changes
critical Withdrawn before enrolling 2026-07-30
critical Trial withdrawn: Study is withdrawing to the retirement of the PI. 2026-07-30
notable Enrollment reduced: 3 -> 0 participants 2026-07-30
Trial Details
NCT Number NCT03520751
Lead Sponsor Nationwide Children's Hospital
Conditions Charcot-Marie-Tooth Neuropathy Type 1A
Start Date 2027-04
Primary Completion 2029-04 (estimated)
Study Completion 2030-04 (estimated)
Updated on ClinicalTrials.gov 2026-07-29