Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
NOT YET RECRUITING
NCT03520751
PHASE1/PHASE2

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

Sponsor: Nationwide Children's Hospital

View on ClinicalTrials.gov

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.

Official title: Phase I/IIa Trial Evaluating scAAV1.tMCK.NTF3 for Treatment of Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A)

Key Details

Gender

All

Age Range

18 Years - 35 Years

Study Type

INTERVENTIONAL

Enrollment

3

Start Date

2027-04

Completion Date

2030-04

Last Updated

2025-06-24

Healthy Volunteers

No

Interventions

DRUG

scAAV1.tMCK.NTF3

gene vector

Locations (1)

Nationwide Children's Hospital

Columbus, Ohio, United States