a study of rvt-1201 in patients with pulmonary arterial hypertension (elevate 1)

Conditions: Pulmonary Arterial Hypertension

Summary: this is an exploratory phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of rvt-1201 in patients with pulmonary arterial hypertension (pah).

this is an exploratory phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of rvt-1201 in patients with pulmonary arterial hypertension (pah). study participation for each patient will last approximately 3 months and will consist of a screening period (up to 28 days in duration), a baseline period (day 1, pre-dose), a 6-week treatment period, and a 2-week follow-up period. the study will enroll approximately 36 patients at approximately 20 centers across the united states and canada.

Eligibility criteria:
key inclusion criteria: * symptomatic pah belonging to one of the following types: * idiopathic * heritable * drug- or toxin- induced * associated with one of the following: connective tissue disease or congenital heart disease * world health organization (who) functional class (fc) ii or iii * pah diagnosed by right heart cardiac catheterization prior to screening * receiving standard of care treatment for pah with oral monotherapy or dual therapy for at least 12 weeks prior to screening at a dose which has been stable for at least 8 weeks prior to screening * if on a diuretic, dose must be stable for at least 4 weeks prior to screening, with no changes anticipated during study participation * 6-minute walk distance (6mwd) between 150 and 500 meters at screening and baseline visits * plasma n-terminal pro b-type natriuretic peptide (nt-probnp) level ≥ 300 pg/ml at screening * ability and willingness to give written informed consent and to comply with the requirements of the study key exclusion criteria: * pah associated with human immunodeficiency virus (hiv) infection, portal hypertension or schistosomiasis * other types of pulmonary hypertension (ph): * pulmonary hypertension due to left heart disease (who ph group 2) * pulmonary hypertension due to lung diseases and/or hypoxia (who ph group 3) * chronic thromboembolic pulmonary hypertension (who ph group 4) * pulmonary hypertension with unclear multifactorial mechanisms (who ph group 5) * hospitalization for pulmonary hypertension within 12 weeks of screening * cardiopulmonary rehabilitation program based on exercise (planned, or started ≤ 12 weeks prior to screening) * prostanoid or prostacyclin receptor agonist therapy within 12 weeks of screening * evidence of left-sided heart disease * if pulmonary function tests were done prior to screening, pulmonary function tests demonstrate obstructive or restrictive lung disease * use of telotristat (xermelo®) within the last 6 months * use of any investigational drug within 30 days or five half-lives (whichever is longer) prior to screening, or 90 days if an investigational drug for pah * have uncontrolled atrial fibrillation (afib) or other uncontrolled arrhythmias * body mass index (bmi) \>45 kg/m2 * women of childbearing potential who are pregnant, planning to become pregnant, or lactating or female/male patients unwilling to use effective contraception

Keywords: pulmonary arterial hypertension (pah) pulmonary hypertension (ph) rvt-1201 rodatristat ethyl tryptophan hydroxylase (tph) serotonin reduction elevate 1 kar5417