2-HOBA for High Cholesterol

Overseen By Beth Meador, RN

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Vanderbilt University Medical Center

Disqualifiers: Diabetes, Obesity, Hypertension, Smoking, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 6 weeks

72 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

How does the drug 2-HOBA differ from other treatments for high cholesterol?

2-HOBA (2-Hydroxybenzylamine) is unique because it is being studied for its potential to lower cholesterol through a novel mechanism, unlike traditional drugs like statins or fibrates that primarily target cholesterol synthesis or absorption. This makes it a potentially new option for patients who may not respond well to existing treatments. ^{1 2 3 4 5}

Research Team

MacRae F. Linton, MD

Principal Investigator

Vanderbilt University Medical Center

Eligibility Criteria

This trial is for individuals with a genetic condition called heterozygous Familial Hypercholesterolemia (FH), which causes high cholesterol. It's not suitable for pregnant people, those with recent cancer, severe kidney or liver disease, uncontrolled hypertension, heart failure, recent heart attack or stroke, certain autoimmune diseases like lupus and rheumatoid arthritis, HIV/AIDS, hypothyroidism or if they are current smokers.

Inclusion Criteria

I have a genetic condition that causes high cholesterol.

I have a genetic condition that causes high cholesterol.

Exclusion Criteria

Your blood has high levels of triglycerides when you haven't eaten for a while.

I have been diagnosed with systemic lupus erythematosus.

I have Type 2 Diabetes and my BMI is over 30.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 750 mg of 2-HOBA or placebo every 8 hours for 6 weeks

6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• 2-Hydroxybenzylamine
• Placebo

Trial Overview The study is testing whether taking a compound called 2-Hydroxybenzylamine (2-HOBA) can improve the function of HDL ('good' cholesterol) in people with FH. Participants will receive either 750 mg of 2-HOBA or a placebo every eight hours for six weeks to see if there's any difference between the two groups.

Participant Groups

Group I: 2-Hydroxybenzylamine (2-HOBA) Active Control 1 Intervention

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

Group II: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Vanderbilt University Medical Center

Lead Sponsor

Trials

922

Recruited

939,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

Findings from Research

In a 26-week trial involving 39 patients with primary hypercholesterolemia, atorvastatin 10 mg significantly reduced LDL cholesterol levels by 35%, while the placebo group saw no change.

Atorvastatin also lowered triglyceride levels by 21%, and the treatment was well tolerated with no significant side effects reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The lipid-lowering effects of atorvastatin, a new HMG-CoA reductase inhibitor: results of a randomized, double-masked study. Heinonen, TM., Stein, E., Weiss, SR., et al.[2019]

Gemfibrozil, at a daily dose of 1200 mg, effectively reduces total serum cholesterol, triglycerides, and apo B levels while increasing HDL cholesterol in patients with hyperlipidemia, demonstrating its efficacy as a lipid-lowering agent.

Long-term studies indicate that gemfibrozil has a more significant impact on lowering triglycerides than cholesterol, has few side effects, and maintains its effectiveness over time, making it a promising option for managing lipid levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hyperlipoproteinemia, atherosclerosis and gemfibrozil. Nash, DT.[2017]

In a double-blind crossover trial with 20 patients suffering from hyperlipidemia, the new aryloxy derivative (clinofibrate) significantly reduced total cholesterol by 11% and triglycerides by 48% compared to placebo, which showed no significant changes.

Clinofibrate was generally well tolerated, with only one patient experiencing moderate diarrhea and abdominal pain, leading to the withdrawal of the treatment as a precaution.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Therapy of hyperlipidemia. Clinical experience with clinofibrate]. Avellone, G., Di Garbo, V., Strano, A.[2013]

In a placebo-controlled, double-blind study across multiple centers, atorvastatin significantly reduced total cholesterol, low-density lipoprotein (LDL) cholesterol, and apolipoprotein B in a dose-dependent manner.

The study reported no serious adverse events, indicating that atorvastatin is a safe option for managing cholesterol levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of atorvastatin in primary hypercholesterolemia. Japan Cholesterol Lowering Atorvastatin Study (J-CLAS) Group. [2018]

In a study involving 8 healthy male volunteers, the ACAT inhibitor CL 277,082 was well tolerated with no significant side effects, indicating a good safety profile for this drug.

However, the treatment did not significantly affect cholesterol absorption, sterol excretion rates, or plasma lipoprotein levels, suggesting that CL 277,082 may not effectively inhibit ACAT activity or lower cholesterol in humans at the tested dosage.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of the ACAT inhibitor CL 277,082 on cholesterol metabolism in humans. Harris, WS., Dujovne, CA., von Bergmann, K., et al.[2019]

References

1. United States pubmed.ncbi.nlm.nih.gov

The lipid-lowering effects of atorvastatin, a new HMG-CoA reductase inhibitor: results of a randomized, double-masked study. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Hyperlipoproteinemia, atherosclerosis and gemfibrozil. [2017]

3. Italy pubmed.ncbi.nlm.nih.gov

[Therapy of hyperlipidemia. Clinical experience with clinofibrate]. [2013]

4. United States pubmed.ncbi.nlm.nih.gov

Efficacy of atorvastatin in primary hypercholesterolemia. Japan Cholesterol Lowering Atorvastatin Study (J-CLAS) Group. [2018]

5. United States pubmed.ncbi.nlm.nih.gov

Effects of the ACAT inhibitor CL 277,082 on cholesterol metabolism in humans. [2019]