Search Clinical Trials
 
By Condition
 
By Location (USA)
 
By Location (Other)
 
By Sponsor
 
Resources
 
Privacy Policy
 
About Us
Disclaimer
Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia Clinical Trials Data presented on Clinical Trials Search is not meant to be a substitute for qualified medical advice, visits or professional assistance with a genuine dr.. We are not doctors. Always consult your mD about Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia Clinical research trials and Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia medical trials take place in many of places throughout the U.S.A.. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials usually evaluate the effectiveness of new does drugs. The purpose of the studies / projects is to solve specific human healthcare questions. Clinical trials are a popular way for mDs, government agencies, and private sector companies to find cures for all varieties of conditions, like Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia. Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia Clinical Trials and other clinical trials allow for volunteers to have health treatment options before they are available to the masses. Many times the human subjects acquire professional assistance for free of charge, and sometimes they are compensated for their time. Occasionally there is a cost for a Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia clinical trial. Test subjects typically obtain the finest healthcare available for their Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia condition. Dangers are a reality, nevertheless, and might include additional or frequent doctor trips, medical dangers (possibly life-jeopardising), and/or the treatment being ineffectual. Trials are federally regulated with strict guidelines to protect clinical trials patients.

Home > "P" Clinical Trials Conditions > Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia

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



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

For Condition: Familial Hypercholesterolemia
Status: Completed
Sponsor(s): National Center for Research Resources (NCRR) , University of Pennsylvania
Synopsis: 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.
Details: 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.
Eligibility:
Study Type:  Interventional, Treatment
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Homozygous familial hypercholesterolemia, i.e.: Low-density lipoprotein (LDL) cholesterol greater than 500 mg/dL Autosomal dominant inheritance Early-onset tendon and tuberous xanthomas LDL receptor negative, i.e.: Receptor binding in cultured fibroblasts no more than 20% of normal OR Genotype with 2 previously described, disease-causing alleles Advanced coronary heart disease with relatively poor prognosis, i.e: Angina pectoris History of myocardial infarction Positive exercise tolerance test Atherosclerotic disease in proximal aorta or coronary arteries by ultrasound or angiogram None of the following: Unstable angina pectoris Left ventricular ejection fraction less than 30% Decompensated congestive heart failure Untreated ventricular tachycardia Moderate to severe aortic stenosis Other dyslipidemia Obstructive hepatobiliary disease --Prior/Concurrent Therapy At least 2 weeks since the following: Drugs affecting cholesterol metabolism Plasma exchange LDL apheresis --Patient Characteristics-- Age: Any age Renal: No azotemia No significant proteinuria Other: No hypothyroidism No diabetes
Total Enrollment: 5

Location and Contact Information:

Overall Study Official:
JamesWilson,  Study Chair,  University of Pennsylvania


Additional Information:
Study ID Numbers:  199/12011;  UPHS-28040
Study Start Date: June 1992
Record last reviewed: December 2001
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00004809

Other Familial Hypercholesterolemia Studies:
1. Implitapide in Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy

2. Implitapide in Patients with Heterozygous Familial Hypercholesterolemia (HeFH) on Maximal Concurrent Lipid-Lowering Therapy

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

Related Studies:

Other Familial Hypercholesterolemia Clinical Trials
Other Clinical Trials
Other Clinical Trials

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

  Other Familial Hypercholesterolemia Clinical Trials
  Other Clinical Trials
  Other Clinical Trials


Warning: include(/var/www/cgi-bin/traxis/counter.php) [function.include]: failed to open stream: No such file or directory in /home/cts/domains/clinicaltrialssearch.org/public_html/index.php on line 103

Warning: include() [function.include]: Failed opening '/var/www/cgi-bin/traxis/counter.php' for inclusion (include_path='.:/usr/local/lib/php') in /home/cts/domains/clinicaltrialssearch.org/public_html/index.php on line 103