The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

Part of paid clinical trials in New York, New York.

Sponsor
The Rogosin Institute
Study ID
NCT01109368
Status
Recruiting

Conditions

  • Homozygous Familial Hypercholesterolemia

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

This repository will establish for the first time a system to carefully assess and monitor over time the general health and the amount of cholesterol in the arteries of U.S. children and adults with homozygous familial hypercholesterolemia (hoFH). Patients with this very rare disorder have very high blood levels of cholesterol from birth due to the inheritance of an abnormal gene from each parent. As a result, if untreated, heart attacks and sudden death occur in childhood. Treatments such as LDL-apheresis and liver transplant will lower the cholesterol level, but the best treatment and the best way to monitor the effect of the treatment on the arteries are unknown. The collection of clinical data and blood for analysis of known and yet-to-be discovered markers and predictors of arterial disease will yield new information about the natural history of the disorder and response to treatment. The repository will greatly aid the development of specific protocols that seek to learn more about this disease and new therapies.

Key Dates

Start date
Jun 30, 2010
Status verified
Mar 2026
Primary completion
May 31, 2030
Completion
May 31, 2030

Study Design

Enrollment
60 participants (estimated)

Primary Outcome Measure

Change in disease progression [ Time Frame: 10 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Weill Cornell Medical CollegeNew YorkNew York10021
Lisa C. Hudgins, M.D.
212-746-3561
Lisa Hudgins, M.D. (PRINCIPAL_INVESTIGATOR)
Daniel Levine, Ph.D. (SUB_INVESTIGATOR)
Geoffrey Bergman, M.D. (SUB_INVESTIGATOR)
Sheila Carroll, M.D. (SUB_INVESTIGATOR)
Ajay Mirani, M.D. (SUB_INVESTIGATOR)
Akiko Maehera, M.D. (SUB_INVESTIGATOR)

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