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
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
Treatments tested in this trial
- 2-HOBA
Treatment groups
Locations
Sponsors and collaborators
Vanderbilt University Medical Center
Lead sponsor
National Institutes of Health (NIH)
Collaborator