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NCT04693598 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer Clinical Trial for Krabbe Disease
A Phase 1 study of Krabbe Disease, sponsored by Forge Biologics.
- Active
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 1
- Study location
NCT04693598: Active Phase 1 study of Krabbe Disease, sponsored by Forge Biologics.
NCT04693598 is a Phase 1 study of Krabbe Disease that is active but no longer recruiting, run by Forge Biologics. The registered enrollment target is 6 participants, below the 417-participant average among 4 other Krabbe Disease trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04693598, a Phase 1 study of Krabbe Disease, is active but no longer recruiting, sponsored by Forge Biologics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 1
- Study location
Study 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 Studied
Interventions
- BIOLOGICAL FBX-101
Study Locations (1)
Michigan
- University of Michigan Hospitals - Michigan Medicine - Ann Arbor
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2021-11-05 |
| Est. Completion | 2026-11 |
| Phase | Phase 1 |
What the registry record for NCT04693598 still lists
NCT04693598 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 417-participant average among 4 other Krabbe Disease trials with a reported enrollment target (99% lower).
The record links to 1 condition, with Krabbe Disease appearing as the primary indexed condition, and to 1 intervention - of which FBX-101 is the first listed.
NCT04693598 reports a single indexed study location in Michigan.
Frequently Asked Questions
What is clinical trial NCT04693598 about?
NCT04693598 is a clinical study titled "Gene Transfer Clinical Trial for Krabbe Disease". 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 gen...
What is the current status of trial NCT04693598?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2021-11-05. Estimated completion is 2026-11.
What conditions does trial NCT04693598 study?
This clinical trial studies the following conditions: Krabbe Disease.
What interventions are being tested in trial NCT04693598?
The interventions under investigation include: FBX-101 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04693598?
This trial is sponsored by Forge Biologics, which has 3 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04693598 being conducted?
This trial has 1 study location across Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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