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NCT02651675 · ClinicalTrials.gov registry record · Phase 1
A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
A Phase 1 study, sponsored by REGENXBIO.
- Terminated
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
NCT02651675: Clinical Trial Phase 1 study, sponsored by REGENXBIO.
NCT02651675 is a Phase 1 study that was terminated before completion, run by REGENXBIO. The registered enrollment target is 9 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02651675, a Phase 1 study, was terminated before completion, sponsored by REGENXBIO.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
Study 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).
Primary Outcome
Physical examinations; Clinical laboratory parameters; and adverse event reporting
Interventions
- GENETIC AAV directed hLDLR gene therapy
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2016-03 |
| Est. Completion | 2020-11-27 |
| Phase | Phase 1 |
Why NCT02651675 stopped before completion
NCT02651675 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 1 intervention - of which AAV directed hLDLR gene therapy is the first listed.
NCT02651675 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02651675 about?
NCT02651675 is a clinical study titled "A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)". 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).
What is the current status of trial NCT02651675?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2016-03. Estimated completion is 2020-11-27.
What interventions are being tested in trial NCT02651675?
The interventions under investigation include: AAV directed hLDLR gene therapy (GENETIC).
Who is sponsoring clinical trial NCT02651675?
This trial is sponsored by REGENXBIO, which has 9 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02651675's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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