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A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

NCT02651675 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2023-07-13

Study results available
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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).

Conditions

  • Homozygous Familial Hypercholesterolemia (HoFH)

Interventions

GENETIC

AAV directed hLDLR gene therapy

AAV directed hLDLR gene therapy is a novel adeno-associated viral (AAV8) vector with human low-density lipoprotein receptor (hLDLR) gene

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • REGENXBIO Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-03-31
Primary Completion
2020-11-27
Completion
2020-11-27
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Italy
  • Netherlands

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02651675 on ClinicalTrials.gov