Trials / Active Not Recruiting
Active Not RecruitingNCT04693598
Gene Transfer Clinical Trial for Krabbe Disease
A Phase 1/2 Clinical Study of Intravenous Gene Transfer With an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation (RESKUE)
- Status
- Active Not Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 6 (estimated)
- Sponsor
- Forge Biologics, Inc · Industry
- Sex
- All
- Age
- 1 Day – 12 Months
- Healthy volunteers
- Not accepted
Summary
This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cell transplantation for Krabbe disease, followed by a single infusion of an adeno-associated virus gene therapy product. Extensive natural history subjects will be used to compare as control group.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | FBX-101 | A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (GALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein. |
Timeline
- Start date
- 2021-11-05
- Primary completion
- 2026-11-01
- Completion
- 2026-11-01
- First posted
- 2021-01-05
- Last updated
- 2025-01-30
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04693598. Inclusion in this directory is not an endorsement.