HoFH, the International Clinical Collaborators Registry
Recruiting
Observational Study
Homozygous Familial Hypercholesterolemia
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 1,000 (estimated)
- Sponsor
- University of Pennsylvania · Other
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About This Trial
Homozygous familial hypercholesterolemia (HoFH), a rare inherited disorder caused by bi-allelic mutations in the LDL Receptor pathway, is characterized by extremely elevated levels of low-density lipoprotein cholesterol (LDL-C) from birth and premature atherosclerotic cardiovascular disease (ASCVD). Our current knowledge about HoFH is disjointed and largely stems from relatively small case series …
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Diagnosis of homozygous familial hypercholesterolemia (HoFH) clinically of genetically determined
Exclusion Criteria:
* No diagnosis of HoFH
Contacts