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2-Hydroxybenzylamine (2-HOBA) for Familial Hypercholesterolemia (FH)

This study is testing a drug called 2-Hydroxybenzylamine (2-HOBA) to see if it can improve how "good" cholesterol (HDL) works in people with Familial Hypercholesterolemia (FH). FH is a genetic condition that causes very high cholesterol levels. Researchers believe 2-HOBA might help reduce changes to HDL and LDL cholesterol and make HDL more effective. You could be eligible if you have heterozygous FH, are between 18 and 69 years old, and don't have certain heart conditions or uncontrolled high blood pressure. The main goal is to see if 2-HOBA increases HDL's ability to remove cholesterol from the body. The current recruitment status is unclear, but the study plans to enroll 72 participants.

Study design
This is an interventional study, meaning participants will receive either the study drug or a placebo. It plans to enroll 72 participants.
What's involved
Participants will take 2-Hydroxybenzylamine (2-HOBA) or a placebo three times a day by mouth for 6 weeks.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint is measured from baseline to week 6.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04941599

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

Recruiting
PHASE2Ages 18–69InterventionalPrevention
Vanderbilt University Medical Center
~72 participants
Updated 2026-08-07 on ClinicalTrials.gov
What's tested:2-HydroxybenzylaminePlacebo

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
2-HOBA increases HDL cholesterol efflux capacity.
Measured over Baseline to week 6

Conditions

Familial Hypercholesterolemia

Explore related trials

Hyperlipoproteinemia, Type IIa

NCT04941599

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Vanderbilt University Medical Center

    Nashville, Tennesseeno site contact published

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • MacRae F. Linton, MD · PRINCIPAL_INVESTIGATOR · Vanderbilt University Medical Center

Who to contact

Anca Ifrim, RN
[email protected]
6155224210
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

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Eligibility criteria

Inclusion

Individuals with heterozygous Familial Hypercholesterolemia.

Exclusion

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.

What this trial measures

  • 2-HOBA increases HDL cholesterol efflux capacity.Baseline to week 6

    Change in HDL cholesterol efflux capacity will be measured by macrophage cholesterol efflux assay.