Gene Therapy for IGHMBP2-Related Diseases
Part of paid clinical trials in Columbus, Ohio.
- Sponsor
- Megan Waldrop
- Study ID
- NCT05152823
- Phase
- PHASE1/PHASE2
- Status
- Enrolling By Invitation
Conditions
- CMT2S
- SMARD1
Eligibility Criteria
- Sex
- ALL
- Age
- 2 Months - 14 Years
- Healthy Volunteers
- Not accepted
Interventions
- Gene Therapy — BIOLOGICALAAV9 carrying the IGHMBP2 gene.
Study Details
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.
Key Dates
- Start date
- Nov 4, 2021
- Status verified
- Sep 2025
- Primary completion
- Jul 31, 2028
- Completion
- Jul 31, 2030
Study Design
- Enrollment
- 10 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: Single Intrathecal Delivery
Primary Outcome Measure
Monitoring for the development of unacceptable toxicity. [ Time Frame: 3 years ]
Locations (1)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | - |
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