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

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.

Timeline

Start

2027-04

Primary completion

2029-04

Completion

2030-04

Drugs

Indications