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

August 5, 2026 updated by: MacRae F. Linton, MD, Vanderbilt University Medical Center
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

72

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Tennessee
      • Nashville, Tennessee, United States, 37212
        • Recruiting
        • Vanderbilt University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 69 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: 2-Hydroxybenzylamine (2-HOBA)
2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks.
Drug: 2-Hydroxybenzylamine
2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks.
Other Names:
  • 2-HOBA
Placebo Comparator: Placebo
Placebo- three tabs TID (po) for 6 weeks.
Other: Placebo
Placebo 250 mg three tabs TID (po) for 6 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline to week 6

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline to week 6
2-HOBA reduces modification of HDL by malondialdehyde (MDA).
Time Frame: Baseline to week 6
Measurement of dilysyl-MDA cross-links by mass spectrometry.
Baseline to week 6

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
Baseline to week 6
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
Baseline to week 6
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.
Baseline to week 6
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
Baseline to week 6

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vanderbilt University Medical Center

Collaborators

National Heart, Lung, and Blood Institute (NHLBI)

Investigators

  • Principal Investigator: MacRae F. Linton, MD, Vanderbilt University Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 14, 2024

Primary Completion (Estimated)

June 30, 2027

Study Completion (Estimated)

December 31, 2027

Study Registration Dates

First Submitted

June 18, 2021

First Submitted That Met QC Criteria

June 18, 2021

First Posted (Actual)

June 28, 2021

Study Record Updates

Last Update Posted (Actual)

August 7, 2026

Last Update Submitted That Met QC Criteria

August 5, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 201575
  • 2P01HL116263-06 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Individual participant data that underlie the results reported will be made available (including data dictionaries) after de-identification.

IPD Sharing Time Frame

The data will become available 3 months following publication of outcomes and will remain available for at least 5 years.

IPD Sharing Access Criteria

Data will be made available to researchers who provide a methodologically sound proposal that has been approved by the Vanderbilt Institutional Review Board and the study executive committee.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • ANALYTIC_CODE

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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