Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia
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|ClinicalTrials.gov Identifier: NCT00004809|
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : June 24, 2005
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.
|Condition or disease||Intervention/treatment||Phase|
|Familial Hypercholesterolemia||Procedure: gene therapy||Phase 1|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||5 participants|
|Study Start Date :||June 1992|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004809
|Study Chair:||James M. Wilson||University of Pennsylvania|