A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
Public ClinicalTrials.gov record NCT02651675. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)
Study identification
- NCT ID
- NCT02651675
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Enrollment
- 9 participants
Conditions and interventions
Interventions
- AAV directed hLDLR gene therapy Genetic
Genetic
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 29, 2016
- Primary completion
- Nov 26, 2020
- Completion
- Nov 26, 2020
- Last update posted
- Jul 12, 2023
2016 – 2020
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Boca Raton location | Boca Raton | Florida | 33434 | — |
| Kansas City Location | Kansas City | Kansas | 66160 | — |
| Portland location | Portland | Oregon | 97239 | — |
| Philadelphia Location | Philadelphia | Pennsylvania | 19104 | — |
| Nashville location | Nashville | Tennessee | 37232 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 4 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02651675, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 12, 2023 · Synced Apr 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02651675 live on ClinicalTrials.gov.