Screening Protocol for a Gene Therapy Trial in Subjects With Homozygous Familial Hypercholesterolemia

Completed

Conditions studied: Hypercholesterolemia, Familial

In brief

The purpose of this protocol is to identify and screen potential candidates for future enrollment in a gene therapy clinical trial for HoFH.

Key facts

Study ID

NCT03018678

Run by

University of Pennsylvania

People needed

21

Starts

2016-03-01

Expected to finish

2019-12-03

Last updated by the study team

2020-03-06

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Males and females ≥ 18 years of age
• Clinical presentation consistent with homozygous FH
• Subjects must be able to comprehend and willing to provide a signed IRB approved Informed Consent Form

You may not qualify if…

• Known to carry confirmed mutations in genes affecting LDL receptor functionality other than the LDLR gene
• History of cirrhosis based on documented histological evaluation or non-invasive imaging
• Documented diagnosis of any of the following liver diseases: Hepatitis B or C; Biopsy-proven nonalcoholic steatohepatitis; Biopsy-proven alcoholic liver disease; Autoimmune hepatitis; Primary biliary cirrhosis; Primary sclerosing cholangitis; Wilson's disease; Hemochromatosis; alpha1 anti-trypsin deficiency
• History of immunodeficiency diseases, including a positive HIV test result
• Previous organ transplantation
• Serious or unstable medical or psychological conditions that, in the opinion of the investigator, would compromise the subject's safety or successful participation in the study
• Inability to participate

Where it is running

• University of Pennsylvania — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.