Recruiting
Phase 2

2-HOBA

Sponsor:

Vanderbilt University Medical Center

Code:

NCT04941599

Conditions

Familial Hypercholesterolemia

Eligibility Criteria

Sex: All

Age: 18 - 69

Healthy Volunteers: Not accepted

Interventions

2-Hydroxybenzylamine

Placebo

Study Details

Brief 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.

Conditions

Familial Hypercholesterolemia

Study ID

NCT04941599

Start date

Feb 14, 2024

Status verified date

Aug, 2026

Completion date

Dec 31, 2027

Anticipated

Primary completion date

Jun 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 69

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

72 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Prevention

Interventions and Outcome Measures

Arms

active comparator: 2-Hydroxybenzylamine (2-HOBA)

2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks.

placebo comparator: Placebo

Placebo- three tabs TID (po) for 6 weeks.

Interventions

2-Hydroxybenzylamine

2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks.

Placebo

Placebo 250 mg three tabs TID (po) for 6 weeks.

Primary outcome measure

  • 2-HOBA increases HDL cholesterol efflux capacity. [ Time Frame: Baseline to week 6 ]

Central Contacts and Locations

Central contacts

Anca Ifrim, RN

6155224210anca.ifrim@vumc.org

Locations

Vanderbilt University Medical Center

Recruiting

Nashville, Tennessee, United States, 37212

More Information

Sponsor

Vanderbilt University Medical Center

Last update posted

Aug 7, 2026

Last verified

Aug, 2026

Keywords

  • Familial Hypercholesterolemia
  • HDL
  • LDL
  • 2-HOBA
  • HDL Function

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Vanderbilt University Medical Center on 2026-08-07.