NCT05152823View on ClinicalTrials.gov

Gene Therapy for IGHMBP2-Related Diseases

PHASE1/PHASE2Enrolling by invitationINTERVENTIONAL

Enrollment

10

Participants

Timeline

Start Date

November 4, 2021

Primary Completion Date

July 31, 2028

Study Completion Date

July 31, 2030

Conditions

SMARD1CMT2S

Interventions

BIOLOGICAL

Gene Therapy

AAV9 carrying the IGHMBP2 gene.

Trial Locations (1)

43205

Nationwide Children's Hospital, Columbus

All Listed Sponsors

lead

Megan Waldrop

OTHER