Trials / Completed
CompletedNCT00004809
Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 5 (planned)
- Sponsor
- National Center for Research Resources (NCRR) · NIH
- Sex
- All
- Age
- 0 Years
- Healthy volunteers
- Not accepted
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.
Detailed description
PROTOCOL OUTLINE: Autologous hepatocytes are obtained from a partial hepatectomy and transduced with a recombinant retroviral vector containing the low-density lipoprotein receptor gene. The transduced hepatocytes are infused via the inferior mesenteric vein 3 days following surgery. Traditional therapy is discontinued for 4 weeks prior to protocol therapy and may resume 6 weeks after the hepatocyte infusion.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| PROCEDURE | gene therapy |
Timeline
- Start date
- 1992-06-01
- First posted
- 2000-02-25
- Last updated
- 2005-06-24
Source: ClinicalTrials.gov record NCT00004809. Inclusion in this directory is not an endorsement.