Clinical Trial of 2-HOBA in Pulmonary Arterial Hypertension

NCT06176118 | Not Yet Recruiting Phase 2

• 1 other identifier: 230458
• Study Type: interventional
• Target: 12
• Locations: 0 countries
• Sites: N/A

Brief Summary

Based on existing literature and clinical trials, 2- hydroxbenzylamine (2-HOBA) has clear impact on mechanisms that much of the international field of pulmonary hypertension (PH) research agrees are central to disease progression. The investigator's preliminary data and Phase I studies demonstrate not only a clear positive impact on reducing pulmonary vascular resistances in Group I and II PH, and both cytokine and molecular biomarkers of disease, but also indicated the potential for a substantial positive effect on heart function under load stress. In this Phase II project, investigators will test the safety and efficacy of 2-HOBA in PH patients, improving the function of the right ventricle under stress in a large animal model, and effectiveness in the context of standard-of-care in mouse models and large animals, to establish the remaining data needed to proceed to commercialization.

Conditions

Pulmonary Arterial Hypertension

Keywords

2-hoba; Pulmonary Hypertension; Right Ventricular

Outcome Measures

Primary Outcomes (1)

• Change in acetylated SOD2 and LCAD in plasma

  This is a direct indication of effect; the mechanism of action of 2-HOBA is sequestration of lipids, and so this will demonstrate that it is having the desired molecular effect.

  baseline and 12-weeks

Secondary Outcomes (7)

• Change in plasma LDL, HDL, triglyceride, free fatty acid concentration measured as a concentration (mg/dL)

  baseline and 12-weeks

• Change in plasma acylcarnitine profile measured as a concentration (mg/dL)

  baseline and 12-weeks

• Change in meters walked in six-minute walk distance (meters)

  baseline and 12-weeks

• Change in Quality of Life as measured by the emPHasis-10

  baseline and 12-weeks

• Change in Estimated Right Ventricle (RV) Systolic Pressure, as assessed by echocardiogram results, expressed in mmHg

  baseline and 12-weeks

Study Arms (1)

Unblinded Intervention

EXPERIMENTAL

There is no control group in this study. Only intervention group due to phase of trial and study intentions.

Dietary Supplement: 2-HOBA

Interventions

2-HOBA DIETARY_SUPPLEMENT

2-HOBA supplement take three times daily for 12-weeks

Unblinded Intervention

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults aged 18 or older
• Diagnosed with idiopathic, heritable, simple congenital heart defect, or drug- or toxin-associated pulmonary arterial hypertension (PAH) according to World Health Organization consensus recommendations.
• Stable PAH-specific medication regimen for three months prior to enrollment. Subjects with only a single diuretic adjustment in the prior three months will be included. Adjustments in IV prostacyclin for side effect management are allowed.
• FEV1\> or = 60% predicted and no more than mild abnormalities on lung imaging
• WHO Functional Class II-IV
• Ambulatory

You may not qualify if:

• Sensitivity to 2-HOBA
• Sensitivity to aspirin or salicylates
• Aspirin-related rhinitis or wheezing
• Prohibited from normal activity due to wheelchair bound status, bed bound status, reliance on a cane/walker, activity-limiting angina, activity-limiting osteoarthritis, or other condition that limits activity
• Pregnancy
• Diagnosis of PAH etiology other than idiopathic, heritable, simple congenital heart defect, or associated with drugs or toxins
• Drug and toxin associated PAH patients with active drug use
• Prior diagnosis of cirrhosis
• Malignancy
• eGFR by MDRD \<60mL/min
• Non-english speaking

Sponsors & Collaborators

• Vanderbilt University Medical Center: lead
• National Institutes of Health (NIH): collaborator

MeSH Terms

Conditions

Pulmonary Arterial Hypertension; Hypertension, Pulmonary

Interventions

2-(aminomethyl)phenol

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Central Study Contacts

Thomas Strayer, PhD

CONTACT

615-936-0156; thomas.e.strayer@vumc.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor-Allergy, Pulmonary, and Critical Care Medicine

Study Record Dates

• First Submitted: November 30, 2023
• First Posted: December 19, 2023
• Study Start: May 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: February 18, 2026

Record last verified: 2026-02