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NCT Number: NCT04941599

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

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.

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Key information

Conditions

Familial Hypercholesterolemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year–69 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location status: Recruiting

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

2-hydroxybenzylamine

Drug

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

Other names: 2-HOBA

Placebo

Other

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

Primary outcomes

  1. 2-HOBA increases HDL cholesterol efflux capacity.

    Time frame: Baseline to week 6

    Change in HDL cholesterol efflux capacity will be measured by macrophage cholesterol efflux assay.

Secondary outcomes

  1. 2-HOBA reduces modification of HDL by Isolevuglandin (Iso-LG).

    Time frame: Baseline to week 6

    Measurement of the Iso-LG-lysine lactam by mass spectrometry.

  2. 2-HOBA reduces modification of HDL by malondialdehyde (MDA).

    Time frame: Baseline to week 6

    Measurement of dilysyl-MDA cross-links by mass spectrometry.

Other outcomes

  1. Change in HDL anti-inflammatory function in an in vitro assay of macrophage cytokine production (IL-1B, TNFa, IL-6).

    Time frame: Baseline to week 6

    Measurement of changes in LPS-stimulated macrophage cytokine production(IL-1B, TNFa, IL-6).

  2. Change in HDL anti-oxidant function in an in vitro assay of macrophage reactive oxygen species production.

    Time frame: Baseline to week 6

    Measurement of changes in H2O2-stimulated macrophage reactive oxygen species

  3. Change in HDL microRNA and small noncoding ribonucleic acid (sRNA) composition

    Time frame: Baseline to week 6

    HDL microRNA and sRNA will be measured through high-throughput sequencing with quantitative polymerase chain reaction (qPCR) validation.

  4. Effects of 2-HOBA on HDL and LDL subpopulation sizes

    Time frame: Baseline to week 6

    HDL and LDL subpopulation sizes and particle numbers will be measured by NMR

Study contacts

Contact information is provided by the study sponsor or research team.

Anca Ifrim, RN

CONTACT

[email protected]

6155224210

Sponsors and collaborators

Lead sponsor

Vanderbilt University Medical Center

Other

Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

Registry information

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Jun 28, 2021
Registry last updated
May 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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