Clinical Trials Logo
  1. Home
  2. Familial Hypercholesterolemia
  3. NCT00004809

Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia

Familial Hypercholesterolemia Clinical Trial

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00004809
Study type Interventional
Source Office of Rare Diseases (ORD)
Contact
Status Completed
Phase Phase 1
Start date June 1992
View Details
See also
  Status Clinical Trial Phase
Enrolling by invitation NCT05284513 - Collaborative Approach to Reach Everyone With Familial Hypercholesterolemia (CARE-FH) N/A
Enrolling by invitation NCT05271305 - Pilot Study for a National Screening for Familial Hypercholesterolemia
Completed NCT02750527 - Pediatric Population Screening for Type 1 Diabetes and Familial Hypercholesterolemia in Lower Saxony, Germany
Not yet recruiting NCT00924339 - Soy Food Intervention Trial N/A
Withdrawn NCT00751608 - Effect of APL180 on Endothelial Function in Familial Hypercholesterolemia Patients Phase 2
Terminated NCT00079846 - Implitapide in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy Phase 2
Completed NCT02624869 - Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) Phase 3
Recruiting NCT05758779 - The Danish Familial Hypercholesterolemia Organized Coronary Screening Trial N/A
Enrolling by invitation NCT04929457 - Evaluation of a Digiphysical Screening Method to Identify and Diagnose Familial Hypercholesterolemia
Not yet recruiting NCT04455581 - A Study to Determine the Safety, Tolerability, and Efficacy of SHR-1209 in Patients With Familial Hypercholesterolemia Phase 2
Recruiting NCT04101149 - Genetic Causes of Familial Hypercholesterolemia
Completed NCT00943306 - Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia Phase 3
Completed NCT02462655 - Effects of LDL Apheresis System on the Expression of Genes Involved in Lipoprotein Metabolism and Inflammation in Homozygotes for Familial Hypercholesterolemia N/A
Terminated NCT00079859 - Implitapide in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) on Maximal Concurrent Lipid-Lowering Therapy Phase 2
Recruiting NCT05066932 - Advanced Lipoproptein Profiling and Cardiovascular Risk Stratification in Familial Hypercholesterolemia
Not yet recruiting NCT04958629 - A Prospective Cohort Study on Familial Hypercholesterolemia in Health Examination Population
Completed NCT02709850 - Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia Phase 1
Active, not recruiting NCT03832985 - Pediatric Reporting of Adult-Onset Genomic Results Early Phase 1
Terminated NCT02013713 - French Observatory of Familial Hypercholesterolemia in Cardiology
Recruiting NCT02009345 - Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale Canada