RecruitingPhase 2

2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)

United States72 participantsStarted 2024-02-14

Plain-language summary

The Investigators will test the hypothesis that 2-HOBA will reduce modification of HDL and LDL and improve HDL function in humans with heterozygous FH. The Investigators plan to first study subjects with Familial Hypercholesterolemia (FH), treating them with 750 mg of 2-HOBA or placebo every 8 hours for 6 weeks.

Who can participate

Age range

18 Years – 69 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Individuals with heterozygous Familial Hypercholesterolemia. Exclusion Criteria: * Myocardial infarction or stroke within the last 6 months * unstable angina, symptoms of angina within the last 3 months * NYHA class III or IV heart failure or LVEF \< 30% * poorly controlled hypertension: SBP \> 180 mm Hg or DBP \> 110 mm Hg, * pregnancy, * evidence of a previous acute coronary syndrome, * current smokers, * individuals with Type 2 Diabetes Mellitus, obesity (BMI \> 30), * hypertriglyceridemia (fasting TG \> 250 mg/dl), * renal insufficiency (Cr \> 1.8), * hepatic disease (aspartate aminotransferase(AST) or alanine aminotransferase (ALT) \> 2x ULN), * hypothyroidism, * nephrotic syndrome, * rheumatoid arthritis, * systemic lupus erythematosus, * AIDS or HIV * history of malignancy of any organ in last 5 years.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

2-HOBA increases HDL cholesterol efflux capacity.

Timeframe: Baseline to week 6

Trial details

NCT IDNCT04941599

SponsorVanderbilt University Medical Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-06-30

Contact for this trial

Anca Ifrim, RN

6155224210 anca.ifrim@vumc.org

View on ClinicalTrials.gov More Familial Hypercholesterolemia trials