Homozygous Familial HypercholesterolemiaNCT03135184
HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia
A study testing HDL Therapeutics PDS-2 System in people with Homozygous Familial Hypercholesterolemia and HoFH, run by HDL Therapeutics at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia, HoFH
- What is being tested?
- HDL Therapeutics PDS-2 System
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Jan 2018to Aug 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
Cedars-Sinai Medical Center (CSMC)
Los Angeles, California, 90048
About this study
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
Read the 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.
- Conditions
- Homozygous Familial Hypercholesterolemia, HoFH
- Being tested
- HDL Therapeutics PDS-2 System
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HDL Therapeutics PDS-2 SystemDevice
Delipidation of plasma HDL to reduce coronary atheroma
| Study group | Role | What they receive |
|---|---|---|
| HDL Therapeutics PDS-2™ System | Experimental | Serial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System Receives: Device: HDL Therapeutics PDS-2 System |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 2 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2017
- Study start26 Jan 2018
- Primary completion7 Jan 2019
- Study completionAug 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Homozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Prospective, unblinded, interventional study
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- HDL Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- MedStar Heart and Vascular Institute
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03135184
- Sponsor's protocol ID
- HALO-FH
- Status last verified
- May 2019
- First submitted
- 26 Apr 2017
- Last updated
- 23 May 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia
- ClinicalTrials.gov ID
- NCT03135184
- Acronym / sponsor ID
- HALO-FH
- Conditions
- Homozygous Familial Hypercholesterolemia, HoFH
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (actual)
- Lead sponsor
- HDL TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- MedStar Heart and Vascular Institute
- Study start
- 26 Jan 2018 (actual)
- Primary completion
- 7 Jan 2019 (actual)
- Study completion
- Aug 2019 (estimated)
- First posted
- 1 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Prospective, unblinded, interventional study
| Arm | Type | Receives |
|---|---|---|
| HDL Therapeutics PDS-2™ System | Experimental | Device: HDL Therapeutics PDS-2 SystemSerial infusions of autologous selectively delipidated HDL/preβ enriched plasma following use of HDL Therapeutics PDS-2™ System |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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 (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HDL Therapeutics PDS-2 SystemDelipidation of plasma HDL to reduce coronary atheroma | Device | HDL Therapeutics PDS-2™ System |
Primary outcome
Change in coronary atheroma
Time frame: 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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2019.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing HDL Therapeutics PDS-2 System in people with Homozygous Familial Hypercholesterolemia and HoFH, run by HDL Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in coronary atheroma (time frame: 2 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 May 2019.