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

Phase 2

Recruiting

• Conditions: Familial Hypercholesterolemia
• Interventions: Drug: 2-Hydroxybenzylamine; Other: Placebo

• Registration Number: NCT04941599
• Lead Sponsor: Vanderbilt University Medical Center

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-06-18
• Study First Submitted QC: 2021-06-18
• Study First Posted: 2021-06-28
• Study Start: 2024-02-14
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-11
• Last Update Posted: 2024-12-13
• Primary Completion: 2025-07
• Study Completion: 2025-11

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 72

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

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
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.

Secondary Outcome Measures

Name	Time	Method
2-HOBA reduces modification of HDL by Isolevuglandin (Iso-LG).	Baseline to week 6	Measurement of the Iso-LG-lysine lactam by mass spectrometry.
2-HOBA reduces modification of HDL by malondialdehyde (MDA).	Baseline to week 6	Measurement of dilysyl-MDA cross-links by mass spectrometry.

Trial Locations

Locations (1)

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States