A Study of RVT-1201 in Patients With Pulmonary Arterial Hypertension (ELEVATE 1)
Purpose
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).
Condition
- Pulmonary Arterial Hypertension
Eligibility
- Eligible Ages
- Between 18 Years and 75 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
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
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- Following screening assessments, PAH patients who meet all entrance criteria will be randomly assigned to receive one of the following treatments in a ratio of 2:1: Arm 1 (n=24) - RVT-1201 Treatment: RVT-1201 immediate-release tablets will be administered orally, at a dose of 600 mg twice daily (BID), for a total of 6 weeks in addition to the patient's current standard of care (SOC) medication(s) for PAH. Arm 2 (n=12) - Placebo Treatment: Matching placebo tablets will be administered orally, at a dose of 600 mg twice daily (BID), for a total of 6 weeks in addition to the patient's current SOC medication(s) for PAH. Participants will be followed in face-to-face visits with trial personnel every 2 weeks for 8 weeks (6 weeks of treatment plus a 2-week follow-up), with an additional phone call at Week 1, to assess drug effects and monitor safety during their treatments.
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental RVT-1201 |
RVT-1201 600 mg immediate-release tablet, administered orally twice daily with food for 6 weeks, in addition to the patient's current standard of care medication(s) for PAH (n=24 [Anticipated]) |
|
|
Placebo Comparator Placebo |
Matching placebo tablet, administered orally twice daily with food for 6 weeks, in addition to the patient's current standard of care medication(s) for PAH (n=12 [Anticipated]) |
|
More Details
- Status
- Terminated
- Sponsor
- Altavant Sciences GmbH
Study Contact
Detailed Description
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.