A Study of RVT-1201 in Patients With Pulmonary Arterial Hypertension (ELEVATE 1)

A Phase 2a, Double-Blind, Placebo-Controlled Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of RVT-1201 in Patients With Pulmonary Arterial Hypertension

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.

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