HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

HDL Therapeutics

Status: Unknown

Conditions: HoFH; Homozygous Familial Hypercholesterolemia

Treatments: Device: HDL Therapeutics PDS-2 System

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT03135184

HALO-FH

Details and patient eligibility

About

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

Full 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.

Enrollment: 6 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

HDL Therapeutics PDS-2™ System

Experimental group

Description:

Serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System

Treatment:

Device: HDL Therapeutics PDS-2 System