Using 2-HOBA to improve HDL function in patients with Familial Hypercholesterolemia

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

Phase 2 Interventional Vanderbilt University Medical Center · NCT04941599

This study is testing if a new treatment called 2-HOBA can help improve cholesterol function in people with Familial Hypercholesterolemia.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment72 (estimated)
Ages18 Years to 69 Years
SexAll
SponsorVanderbilt University Medical Center Academic / other
Locations1 site (Nashville, Tennessee)
Trial IDNCT04941599 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effects of 2-Hydroxybenzylamine (2-HOBA) on individuals with heterozygous Familial Hypercholesterolemia (FH). Participants will receive either 750 mg of 2-HOBA or a placebo every 8 hours for a duration of 6 weeks. The primary aim is to determine if 2-HOBA can reduce the modification of HDL and LDL cholesterol and enhance HDL function. The study is designed to provide insights into potential new treatments for managing cholesterol levels in FH patients.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals diagnosed with heterozygous Familial Hypercholesterolemia.

Not a fit: Patients with recent cardiovascular events, poorly controlled hypertension, or other significant comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve cholesterol management and cardiovascular health for patients with Familial Hypercholesterolemia.

How similar studies have performed: While there have been studies on HDL function, the specific use of 2-HOBA in this context is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria
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.

Where this trial is running

Nashville, Tennessee

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Familial HypercholesterolemiaHDLLDL2-HOBAHDL Function
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.