search
Back to results

HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

Primary Purpose

Homozygous Familial Hypercholesterolemia, HoFH

Status
Unknown status
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
HDL Therapeutics PDS-2 System
Sponsored by
HDL Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Homozygous Familial Hypercholesterolemia

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Cedars-Sinai Medical Center (CSMC)

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

HDL Therapeutics PDS-2™ System

Arm Description

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

Outcomes

Primary Outcome Measures

Change in coronary atheroma
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

Secondary Outcome Measures

Full Information

First Posted
April 26, 2017
Last Updated
May 21, 2019
Sponsor
HDL Therapeutics
Collaborators
MedStar Heart and Vascular Institute
search

1. Study Identification

Unique Protocol Identification Number
NCT03135184
Brief Title
HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia
Official Title
HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia
Study Type
Interventional

2. Study Status

Record Verification Date
May 2019
Overall Recruitment Status
Unknown status
Study Start Date
January 26, 2018 (Actual)
Primary Completion Date
January 7, 2019 (Actual)
Study Completion Date
August 2019 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
HDL Therapeutics
Collaborators
MedStar Heart and Vascular Institute

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
Yes
Device Product Not Approved or Cleared by U.S. FDA
Yes
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Homozygous Familial Hypercholesterolemia, HoFH

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
Prospective, unblinded, interventional study
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
HDL Therapeutics PDS-2™ System
Arm Type
Experimental
Arm Description
Serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System
Intervention Type
Device
Intervention Name(s)
HDL Therapeutics PDS-2 System
Intervention Description
Delipidation of plasma HDL to reduce coronary atheroma
Primary Outcome Measure Information:
Title
Change in coronary atheroma
Description
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
Time Frame
2 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
Cedars-Sinai Medical Center (CSMC)
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

We'll reach out to this number within 24 hrs