Actively Recruiting

Phase 2
Age: 18Years - 69Years
All Genders
ID04941599

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

Led by Vanderbilt University Medical Center · Updated on 2026-05-06

72

Participants Needed

1

Research Sites

26 weeks

Total Duration

On this page

Sponsors

V

Vanderbilt University Medical Center

Lead Sponsor

N

National Heart, Lung, and Blood Institute (NHLBI)

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the effects of 2-Hydroxybenzylamine (2-HOBA) on patients with heterozygous Familial Hypercholesterolemia (FH). The study aims to test whether 2-HOBA can reduce harmful modifications to HDL and LDL cholesterol and improve HDL function in these individuals. This is a Phase 2 clinical trial sponsored by Vanderbilt University Medical Center. Participants will be randomly assigned to receive either 2-HOBA or a placebo. Those in the treatment group will take 750 mg of 2-HOBA daily, divided into three doses of 250 mg each, for a total of 6 weeks. The placebo group will take matching placebo tablets with the same dosing schedule. Both treatments are taken by mouth three times a day. During the study, participants will undergo assessments to measure changes in HDL cholesterol efflux capacity from baseline to week 6, along with evaluations of modifications in HDL by specific biochemical markers. The trial includes monitoring for safety and efficacy throughout the treatment period, which lasts 6 weeks. This study does not include healthy volunteers, and participant involvement is carefully tracked to assess the impact of the investigational drug on cholesterol function.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years - 69Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Individuals with heterozygous Familial Hypercholesterolemia
  • Age 18 to 69 years
Not Eligible

You will not qualify if you...

  • Myocardial infarction or stroke within the last 6 months
  • Unstable angina or symptoms of angina within the last 3 months
  • NYHA class III or IV heart failure or left ventricular ejection fraction less than 30%
  • Poorly controlled hypertension: systolic blood pressure over 180 mm Hg or diastolic blood pressure over 110 mm Hg
  • Pregnancy
  • Evidence of a previous acute coronary syndrome
  • Current smokers
  • Type 2 Diabetes Mellitus
  • Obesity (BMI over 30)
  • Hypertriglyceridemia (fasting triglycerides over 250 mg/dl)
  • Renal insufficiency (creatinine over 1.8)
  • Hepatic disease (aspartate aminotransferase or alanine aminotransferase over 2 times the upper limit of normal)
  • Hypothyroidism
  • Nephrotic syndrome
  • Rheumatoid arthritis
  • Systemic lupus erythematosus
  • AIDS or HIV
  • History of malignancy of any organ in last 5 years

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - 6 weeks

Participants receive either 2-Hydroxybenzylamine or placebo three times a day by mouth for 6 weeks.

Visits during treatment occur as scheduled by the study team

Trial Site Locations

Total: 1 location

1

Vanderbilt University Medical Center

Nashville, Tennessee, United States, 37212

Actively Recruiting

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Research Team

A

Anca Ifrim, RN

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

PREVENTION

Number of Arms

2

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