Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia
NCT00004809 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 5
Last updated 2005-06-24
Summary
OBJECTIVES:
I. Develop an approach for treating patients with homozygous familial hypercholesterolemia using gene therapy with autologous hepatocytes transduced with a normal low-density lipoprotein receptor gene.
Conditions
- Familial Hypercholesterolemia
Interventions
- PROCEDURE
-
gene therapy
Sponsors & Collaborators
- collaborator OTHER
-
National Center for Research Resources (NCRR)
lead NIH
Principal Investigators
-
James M. Wilson · University of Pennsylvania
Study Design
- Purpose
- TREATMENT
Eligibility
- Min Age
- 0 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 1992-06-30
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