Trials / Enrolling By Invitation
Enrolling By InvitationNCT05152823
Gene Therapy for IGHMBP2-Related Diseases
Phase I/IIa Intrathecal Gene Delivery Clinical Trial for IGHMBP2-Related Diseases
- Status
- Enrolling By Invitation
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- Sponsor
- Megan Waldrop · Academic / Other
- Sex
- All
- Age
- 2 Months – 14 Years
- Healthy volunteers
- Not accepted
Summary
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Gene Therapy | AAV9 carrying the IGHMBP2 gene. |
Timeline
- Start date
- 2021-11-04
- Primary completion
- 2028-07-01
- Completion
- 2030-07-01
- First posted
- 2021-12-10
- Last updated
- 2025-09-30
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05152823. Inclusion in this directory is not an endorsement.