Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A
Status:
Suspended
Trial end date:
2023-07-01
Target enrollment:
Participant gender:
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.