HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

Not Applicable

• Conditions: HoFH; Homozygous Familial Hypercholesterolemia
• Interventions: Device: HDL Therapeutics PDS-2 System

• Registration Number: NCT03135184
• Lead Sponsor: HDL Therapeutics

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.

Study Timeline

• Study First Submitted: 2017-04-26
• Study First Submitted QC: 2017-04-26
• Study First Posted: 2017-05-01
• Study Start: 2018-01-26
• Primary Completion: 2019-01-07
• Status Verified: 2019-05
• Last Update Submitted: 2019-05-21
• Last Update Posted: 2019-05-23
• Study Completion: 2019-08

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 6

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Cedars-Sinai Medical Center (CSMC); 🇺🇸 Los Angeles, California, United States