HoFH, Homozygous Familial Hypercholesterolemia
Conditions
Brief summary
Assess the effect on coronary atheroma of serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System
Detailed description
The PDS-2™ System is intended to reduce coronary atheroma in patients with Homozygous Familial Hypercholesterolemia (HoFH). Subjects will receive serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics' PDS-2 System.
Interventions
DEVICEHDL Therapeutics PDS-2 System
Delipidation of plasma HDL to reduce coronary atheroma
Sponsors
MedStar Heart and Vascular Institute
HDL Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Prospective, unblinded, interventional study
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Clinical diagnosis of Homozygous Familial Hypercholesterolemia (HoFH) due to a defect in the LDL receptor, or the identification of a defect in apoB-100, or a gain of function of PCSK9, or a genetic defect resulting in the HoFH clinical phenotype * No other condition that would preclude the subject from successfully completing the series of plasmapheresis visits in the investigator's opinion * At least one (1) coronary artery study segment will be identified for each subject and all will remain constant throughout the study. The qualifying study segment(s) will have 20% to 40% stenosis. Key
Exclusion criteria
* Planned change in current lipid lowering therapy * Use of oral anticoagulants, unless the dose has been stable for 4 weeks * LDL or plasma apheresis within 1 week prior to enrollment and through 8-week primary endpoint * New York Heart Association (NYHA) class III or IV or last known left ventricular ejection fraction \< 30% * Myocardial infarction, unstable angina, percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG) or stroke within 3 months of enrollment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in coronary atheroma | 2 months | Assessed by coronary imaging in study coronary artery segments following serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System as compared to baseline |
Countries
United States
Outcome results
None listed