Familial HypercholesterolemiaPhase 2NCT04941599
2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)
A Phase 2 study testing 2-Hydroxybenzylamine in people with Familial Hypercholesterolemia, run by Vanderbilt University Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 69 years
- Conditions the study is for
- Familial Hypercholesterolemia
- What is being tested?
- 2-Hydroxybenzylamine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 72 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Feb 2024to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 69 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Individuals with heterozygous Familial Hypercholesterolemia.
Who cannot join
- 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,
Show all 17Show fewer
- 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 locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
About this study
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.
- Conditions
- Familial Hypercholesterolemia
- Being tested
- 2-Hydroxybenzylamine, Placebo
- Keywords
- Familial Hypercholesterolemia, HDL, LDL, 2-HOBA, HDL Function
- Medical subject headings
- Hyperlipoproteinemia Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
2-HydroxybenzylamineDrug
Also called 2-HOBA
2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks.
PlaceboOther
Placebo 250 mg three tabs TID (po) for 6 weeks.
| Study group | Role | What they receive |
|---|---|---|
| 2-Hydroxybenzylamine (2-HOBA) | Active comparator | 2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks. Receives: Drug: 2-Hydroxybenzylamine |
| Placebo | Placebo comparator | Placebo- three tabs TID (po) for 6 weeks. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-HOBA increases HDL cholesterol efflux capacity.
Change in HDL cholesterol efflux capacity will be measured by macrophage cholesterol efflux assay.
Measured: Baseline to week 6
Secondary outcomes (2)
2-HOBA reduces modification of HDL by Isolevuglandin (Iso-LG).
Measurement of the Iso-LG-lysine lactam by mass spectrometry.
Measured: Baseline to week 6
2-HOBA reduces modification of HDL by malondialdehyde (MDA).
Measurement of dilysyl-MDA cross-links by mass spectrometry.
Measured: Baseline to week 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2021
- Study start14 Feb 2024
- Primary completion30 Jun 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Subjects will be randomized to treatment with either 2-Hydroxybenzylamine (2-HOBA) , a naturally occuring dicarbonyl scavenger, or placebo for 6 weeks.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, MacRae F. Linton, MD, Vanderbilt University Medical Center
- Investigators
- MacRae F. Linton, MD, Principal investigator, Vanderbilt University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04941599
- Sponsor's protocol ID
- 201575
- Other IDs
- 2P01HL116263-06
- Status last verified
- Aug 2026
- First submitted
- 18 Jun 2021
- Last updated
- 7 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data that underlie the results reported will be made available (including data dictionaries) after de-identification.
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)
- ClinicalTrials.gov ID
- NCT04941599
- Acronym / sponsor ID
- 201575
- Conditions
- Familial Hypercholesterolemia
- Keywords
- Familial Hypercholesterolemia, HDL, LDL, 2-HOBA, HDL Function
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 14 Feb 2024 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 28 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Subjects will be randomized to treatment with either 2-Hydroxybenzylamine (2-HOBA) , a naturally occuring dicarbonyl scavenger, or placebo for 6 weeks.
| Arm | Type | Receives |
|---|---|---|
| 2-Hydroxybenzylamine (2-HOBA) | Active comparator | Drug: 2-Hydroxybenzylamine2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks. |
| Placebo | Placebo comparator | Other: PlaceboPlacebo- three tabs TID (po) for 6 weeks. |
Who may qualify
- Ages
- 18 to 69 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Individuals with heterozygous Familial Hypercholesterolemia.
Key exclusion criteria (17 listed)
- 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,
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 2-HydroxybenzylamineAlso: 2-HOBA2-Hydroxybenzylamine (2-HOBA) 250 mg three tabs TID (po) for 6 weeks. | Drug | 2-Hydroxybenzylamine (2-HOBA) |
| PlaceboPlacebo 250 mg three tabs TID (po) for 6 weeks. | Other | Placebo |
Primary outcome
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.
2 secondary outcomes
2-HOBA reduces modification of HDL by Isolevuglandin (Iso-LG).
Time frame: Baseline to week 6
2-HOBA reduces modification of HDL by malondialdehyde (MDA).
Time frame: Baseline to week 6
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2026.
- Individual participant dataSharing planned: Yes. Individual participant data that underlie the results reported will be made available (including data dictionaries) after de-identification.
Related trials for Familial Hypercholesterolemia
Active, not recruitingObservational study
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Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia
Not yet recruitingObservational study
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia
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Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)
Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
MacRae F. Linton, MD principal investigator
Vanderbilt University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 2-Hydroxybenzylamine in people with Familial Hypercholesterolemia, run by Vanderbilt University Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Anca Ifrim, RN (6155224210, anca.ifrim@vumc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2-HOBA increases HDL cholesterol efflux capacity. (time frame: Baseline to week 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2026.