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

Vanderbilt University Medical Center

Status and phase: Enrolling; Phase 2

Conditions: Familial Hypercholesterolemia

Treatments: Drug: 2-Hydroxybenzylamine; Other: Placebo

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT04941599

201575

2P01HL116263-06 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Enrollment: 72 estimated patients

Sex: All

Ages: 18 to 69 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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72 participants in 2 patient groups, including a placebo group

2-Hydroxybenzylamine (2-HOBA)

Active Comparator group

Description:

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

Treatment:

Drug: 2-Hydroxybenzylamine

Placebo

Placebo Comparator group

Description:

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

Treatment:

Other: Placebo