Health TrialsbyHealthPanel

Clinical Trial of 2-HOBA in Pulmonary Arterial Hypertension

ConditionPulmonary Arterial Hypertension
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorVanderbilt University Medical Center

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • 2-HOBA

    Dietary supplement

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

Treatment groups

12 Participants
are divided into 1 treatment group
Group A: Unblinded InterventionExperimental treatment 1 intervention

Trial outcomes

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.

Time frame
baseline and 12-weeks

Secondary outcomes

1

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

Plasma concentrations based measures in mg/dL

Time frame
baseline and 12-weeks
2

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

Time frame
baseline and 12-weeks
3

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

The 6MWT measures the distance (in meters), a participant can walk at a comfortable speed on a flat, hard surface in 6 minutes. The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out daily physical activities.

Time frame
baseline and 12-weeks
4

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

The emPHasis-10 is a short and easy questionnaire that consists of 10 items that address breathlessness, fatigue, control, and confidence. Each item is scored on a semantic differential six-point scale (0-5), with contrasting adjectives at each end. A total emPHasis-10 score is derived using simple aggregation of the 10 items. emPHasis-10 scores range from 0 to 50, higher scores indicate worse quality of life.

Time frame
baseline and 12-weeks

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Vanderbilt University Medical Center

Lead sponsor

National Institutes of Health (NIH)

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: