drugset / Trial / NCT02651675
A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
Phase 1/2
Terminated
9 enrolled
REGENXBIO Inc.
National Heart, Lung, and Blood Institute (NHLBI) · collab
Non-randomizedSequentialOpen-labelTreatment
Summary
This first-in-human study is intended to evaluate the safety and preliminary effectiveness of AAV (Adeno-associated virus)-based liver-directed gene therapy in the treatment of adults with Homozygous Familial Hypercholesterolemia (HoFH).
Timeline
- Start
- 2016-03
- Primary completion
- 2020-11-27
- Completion
- 2020-11-27
Outcome
Outcome not reported
Stopped (Business): “Terminated by Sponsor for Business Reasons”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV directed hLDLR gene therapy | Gene therapy (AAV / viral vector) | 2.5e+12 vg/kg | — |
| Subject | AAV directed hLDLR gene therapy | Gene therapy (AAV / viral vector) | 7.5e+12 vg/kg | — |