An observational study in Homozygous Familial Hypercholesterolemia, sponsored by The Rogosin Institute. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-04-06.
Sponsored by The Rogosin Institute · Observational
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Among 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States — 7 open to participants now, 9 under way, 4 of them phase 3. Homozygous Familial Hypercholesterolemia · United States.
Among the 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States, this one is the only one that started in 2010, is one of 5 observational ones, and was the 8th registered.
68 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 13 are open to participants now.
Browse Homozygous Familial Hypercholesterolemia studies →7 of the 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States are open now. Recruiting first, the newest first:
Arrowhead Pharmaceuticals · Sites in United States, Denmark, Poland
Ages 12–17
University of Wisconsin, Madison · Sites in United States
Ages 18+
Novartis Pharmaceuticals · Sites in United States, Austria, China and 8 more countries
Ages 2–11
CRISPR Therapeutics AG · Sites in United States, Australia, New Zealand and 1 more country
Ages 18–75
Novartis Pharmaceuticals · Sites in United States, Argentina, Brazil and 23 more countries
Ages 2–100
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 12 Homozygous Familial Hypercholesterolemia studies recruiting now, 6 (50%) set no upper age limit and 5 (42%) stop at 75 or younger; 8 take children under 18; none take healthy volunteers.
This study: all sexes · no healthy volunteers.
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36 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 2 · Phase 2 11 · Phase 3 18
First in 2003; this study’s year is marked. 1 started in 2010.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 60 is below the median of 167 across 14 observational studies indexed under Homozygous Familial Hypercholesterolemia.
Median enrollment 29 across the 31 interventional studies, 60 across the 5 observational. Of the 26 that completed or were terminated, 19 (73%) have results posted.
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32 of 36 have an industry lead sponsor. The 10 most frequent:
The Rogosin Institute is the lead sponsor of 24 studies on the registry; 3 are open to participants now.
The Rogosin Institute has 1 study filed under Homozygous Familial Hypercholesterolemia — fewer than ten, so there is no comparison to draw.
3 of the studies in Homozygous Familial Hypercholesterolemia with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07037771Phase 3Arrowhead PharmaceuticalsA Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE) | 2027-08-20 |
| NCT04815005University of PennsylvaniaHoFH, the International Clinical Collaborators Registry | 2026-12-31 |
| NCT07491172CRISPR Therapeutics AGA Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias | 2027-06 |
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Among the 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States, from their registry updates:
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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.
Detailed information of "standard of care" procedures will be compiled in a database. These include medical history and physical exam, lipid profiles and other standard blood tests, dietary evaluation and counseling, cardiology evaluation including EKG and echocardiogram,ultrasound of carotids and femoral arteries, CT angiogram and, if indicated, intracoronary angiography (ICA) with intravascular ultrasound (IVUS) and stress echo or nuclear stress testing.
The recommendation for treatment will be individualized. Current options are a) FDA approved cholesterol-lowering medications: statins, ezetimibe b) LDL-apheresis c) liver transplant d) portacaval shunt e) investigational drugs. Treatment of vascular and/or valvular disease may include aspirin, beta blockers, clopidogrel, angioplasty with metal stent, coronary artery bypass surgery, aortic valve repair/replacement.
Research procedures will include medical photos of skin xanthomas, blood assays (apolipoproteins A and B, LDL particle size, homocysteine, TNF, IL-6, insulin, glucose, ICAM, VCAM, P and E selectin, and endothelial progenitor cells), and DNA analysis of the genes for the LDL receptor and other lipid-related genes.
Males and females of any age with severely elevated LDL cholesterol levels without secondary causes and a family history consistent with an autosomal dominant disorder.
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1. Patients of any age and sex who meet clinical or genetic criteria for hoFH as follows:
Documented, untreated fasting LDL cholesterol level of > 500 mg/dL and triglycerides \< 200 mg/dL on a cholesterol-lowering diet for at least 8 weeks with secondary causes excluded, AND:
Exclusion criteria:
1. Inability of patient, or, if less than 18, a parent, to sign .
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Change in disease progression
Time frame: 10 years
Started Jun 2010 (actual). Primary completion is estimated for May 2030, 239 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Homozygous Familial Hypercholesterolemia →
The Rogosin Institute