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Pulmonary Arterial HypertensionPhase 2NCT04712669

A Study of Rodatristat Ethyl in Patients With Pulmonary Arterial Hypertension (Core OLE)

A Phase 2 study testing rodatristat ethyl 300 mg tablet BID and rodatristat ethyl 600 mg BID in people with Pulmonary Arterial Hypertension, run by Altavant Sciences GmbH at 64 sites in 17 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The purpose of this study is to assess the safety and efficacy of Rodatristat Ethyl in pulmonary arterial hypertension (PAH) patients. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Pulmonary Arterial Hypertension
What is being tested?
rodatristat ethyl 300 mg tablet BID and rodatristat ethyl 600 mg BID
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
108 participants
Where
64 sitesin 17 countries
Study dates
15 Mar 2021to 28 Aug 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 1. Male and female 18 years or older 2. Body Mass Index (BMI) >18kg/m2 to <=40kg/m2 3. Symptomatic PAH belonging to one of the following 2018 WHO Clinical Group 1 subtypes:
  • Idiopathic PAH b. Heritable PAH c. Drug- or toxin-induced d. PAH associated with:
  • Connective tissue disease
  • Congenital systemic to pulmonary shunt (atrial septal defect, ventricular septal defect, patent ductus arteriosus) repaired at least one year prior to Screening
  • Human immunodeficiency virus (HIV) infection - if diagnosed with HIV, must have stable disease status defined as follows:
  • stable treatment with HIV medications for at least 8 weeks prior to Screening
Show all 19Show fewer
  • no active opportunistic infection during the Screening Period
  • no hospitalizations due to HIV for at least 4 weeks prior to Screening
  • WHO FC II or III
  • Confirmed diagnosis of PAH and meet all the following hemodynamic criteria by means of a screening RHC completed prior to randomization:
  • mPAP of >20 mmHg
  • PVR ≥ 350 dyne•sec/cm5
  • Pulmonary capillary wedge pressure (PCWP) or left ventricular end diastolic pressure (LVEDP) of ≤ 12 mmHg if PVR ≥ 350 and < 500 dyne•sec/cm5, or PCWP/LVEDP ≤ 15 mmHg if PVR ≥ 500 dyne•sec/cm5
  • 6MWD of 100 to 550 meters at Screening
  • Currently on a stable treatment regimen with one or more treatments approved for PAH. Stable therapy is defined as receiving the same medication(s) for ≥ 12 weeks prior to the screening RHC and at a stable dose level for each for ≥ 8 weeks prior to the screening RHC (see Protocol Section 6.6.2 for approved PAH medications). Any instances where doses of a medication have been missed prior to RHC must be discussed with the Medical Monitor prior to performing the RHC.
  • Meet all of the following criteria determined by pulmonary function tests completed no more than 24 weeks prior to Screening (performed with or without bronchodilation):
  • Forced expiratory volume in one second (FEV1) ≥ 60% of predicted normal, and
  • Total lung capacity (TLC) ≥ 70% of predicted normal or FVC ≥ 70% predicted if TLC is not available; For subjects with CTD associated PAH, if TLC is ≥ 60% of predicted but < 70% of predicted of if FVC ≥ 60% or predicted but < 70% of predicted, high resolution computed tomography [HRCT] obtained within 6 months of screening may be utilized to demonstrate limited interstitial lung disease
  • If participating in an exercise program for pulmonary rehabilitation, the program must have been initiated ≥ 12 weeks prior to Screening, and patient must agree to maintain the current level of rehabilitation for the first 24 weeks of receiving IP. If not participating in an exercise training program for pulmonary rehabilitation, patient must agree not to enroll in an exercise training program for pulmonary rehabilitation during the Screening Period and the first 24 weeks of receiving IP.

Who cannot join

  • Women of childbearing potential who are pregnant, planning to become pregnant, or lactating or female/male patients unwilling to use effective contraception
  • WHO pulmonary hypertension (PH) Group 1 PAH associated with portal hypertension or schistosomiasis; PH due to left heart disease (WHO PH Group 2), lung diseases and/or hypoxia (WHO PH Group 3), chronic thromboembolic PH (WHO PH Group 4), or PH with unclear multifactorial mechanisms (WHO PH Group 5)
  • PH associated with significant venous or capillary involvement (PCWP > 15 mmHg), pulmonary capillary hemangiomatosis, portal hypertension, or unrepaired congenital heart defects (CHD)
  • Three or more of the following risk factors for left ventricular disease:
  • BMI > 30 kg/m2
  • Diagnosis of essential hypertension that is actively treated
Show all 18Show fewer
  • Diabetes mellitus
  • History of significant coronary artery disease (e.g., chronic stable angina, history of coronary intervention within the last 3 months, or a stenosis > 70% at coronary angiography)
  • Atrial fibrillation
  • Left atrial volume index > 41 mL/m2 [or left atrial diameter (LA) > 4 cm if LAVi unavailable]
  • Known genetic hypertrophic cardiomyopathy
  • Known cardiac sarcoidosis or amyloidosis
  • The patient has a history of, or currently has, a constrictive cardiomyopathy.
  • Known history of any left ventricular ejection fraction (LVEF) < 40% by echocardiogram within 3 years of randomization (Note: a transient decline in LVEF below 40% that occurred and recovered more than 6 months before the start of Screening and was associated with an acute intercurrent condition [e.g., atrial fibrillation] is allowed).
  • Hemodynamically significant valvular heart disease as determined by the Investigator, including:
  • greater than mild aortic and/or mitral stenosis and/or
  • severe mitral and/or aortic regurgitation (> Grade 3)
  • Severe arthritis, musculoskeletal problems, or morbid obesity that, in the opinion of the Investigator, is the cause of the patient's functional limitation and would affect the patient's ability to perform or complete the 6MWT.

Trial locations and contacts

64 sites in 17 countries.

64 sites in 17 countries

United States28 sites

  • University of New Mexico Heath Science Center

    Albuquerque, New Mexico, 87106

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • University of Colorado

    Aurora, Colorado, 80045

  • Tufts Medical Center

    Boston, Massachusetts, 02111

  • Brigham and Women's Hospital (BWH), Harvard Medical School

    Boston, Massachusetts, 02115

  • University of North Carolina Medical Center - Chapel Hill

    Chapel Hill, North Carolina, 27514

  • University of Cincinnati Physicians

    Cincinnati, Ohio, 45219

  • UT Southwestern

    Dallas, Texas, 75390

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Inova Fairfax Hospital

    Falls Church, Virginia, 22042-3307

  • Houston Methodist Hospital

    Houston, Texas, 77030

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224

  • The University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • University of California San Diego Health Sciences

    La Jolla, California, 92093

  • VA Greater LA Healthcare System/UCLA

    Los Angeles, California, 90073

  • Ronald Reagan UCLA Medical Center

    Los Angeles, California, 90095

  • Norton Pulmonary Specialists

    Louisville, Kentucky, 40202

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • NYU Langone Health

    New York, New York, 10016

  • Temple University Hospital

    Philadelphia, Pennsylvania, 19140

  • Arizona Pulmonary Specialists

    Phoenix, Arizona, 85012

  • Brown University - Rhode Island Hospital

    Providence, Rhode Island, 02903

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • University of Rochester

    Rochester, New York, 14623

  • UC Davis Medical Center

    Sacramento, California, 95816

  • Jeffrey S. Sager, MD Medical Corporation

    Santa Barbara, California, 93105

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • George Washington University Medical Center

    Washington D.C., District of Columbia, 20037

Other trials in United States

Canada4 sites

  • Peter Lougheed Centre

    Calgary, Alberta, T1Y6J4

  • London Health Sciences Centre - Victoria Hospital

    London, Ontario, N6A 5W9

  • Sir Mortimer B. Davis Jewish General Hospital

    Montreal, Quebec, H3T 1E2

  • University Health Network, Toronto General Hospital

    Toronto, Ontario, M5G 2N2

Other trials in Canada

France4 sites

  • Centre Hospitalier Universitaire (CHU) de Caen - Hopital Cote de Nacre

    Caen, Calvados, 14033

  • Chu De Bicetre

    Le Kremlin-Bicêtre, Val-de-Marne, 94275

  • Groupement Hospitalier Est

    Lyon, Rhône, 69677

  • CHU de Saint-Etienne - Hopital Nord

    Saint-Etienne, 42270

Other trials in France

Poland4 sites

  • Uniwersytecki Szpital Kliniczny w Bialymstoku

    Bialystok, 15-276

  • Wojewodzki Specjalistyczny Szpital im. dr Wl. Bieganskiego

    Lodz, Lódzkie, 91-347

  • Samodzielny Publiczny Szpital Kliniczny nr 4 w Lublinie

    Lublin, 20-954

  • Europejskie Centrum Zdrowia Otwock Szpital im Fryderyka Chopina

    Otwock, 05-400

Other trials in Poland

Italy3 sites

  • AOU S.Martino, IRCCS, IST-Istituto Nazionale Ricerca Sul Can

    Genova, 16132

  • IRCCS Policlinico San Matteo, Università degli studi di Pavi

    Pavia, 27100

  • Umberto I Policlinico di Roma, Università La Sapienza

    Rome, Roma, 00161

Other trials in Italy

Serbia3 sites

  • Clinical Center of Serbia

    Belgrade, 11000

  • Institute for Cardiovascular diseases of Vojvodina

    Kamenitz, Vojvodina, 21204

  • Institute for Pulmonary Diseases of Vojvodina

    Kamenitz, Vojvodina, 21204

Other trials in Serbia

Austria2 sites

  • Ordensklinikum Linz GmbH Elisabethinen

    Linz, Upper Austria, 4020

  • AKH- Wien, Medizinische Univsersität Wien

    Vienna, 1090

Other trials in Austria

Belgium2 sites

  • Hôpital Erasme

    Brussels, Brussels Capital, 1070

  • UZ Leuven - Campus Gasthuisberg - Pneumologie

    Leuven, Vlaams Brabant, 3000

Other trials in Belgium

Bosnia and Herzegovina2 sites

  • University Clinical Centre of the Republic of Srpska

    Banja Luka, 78 000

  • University Clinical Hospital Mostar

    Mostar, 88000

Other trials in Bosnia and Herzegovina

Czechia2 sites

  • Fakultni nemocnice Olomouc

    Olomouc, 779 00

  • Vseobecna fakultni nemocnice v Praze

    Prague, 128 08

Other trials in Czechia

Spain2 sites

  • Hospital Clinic de Barcelona

    Barcelona, 08036

  • Hospital Universitario 12 de Octubre

    Madrid, 28041

Other trials in Spain

Ukraine2 sites

  • Dnipropetrovsk Regional Clinical Diagnostic Center

    Dnipro, Dnipropetrovsk Oblast, 49070

  • Nats Naukovyi Tsentr Amn Ukrainy

    Kyiv, 03680

Other trials in Ukraine

United Kingdom2 sites

  • Royal Free London NHS Foundation Trust

    London, NW3 2QG

  • Royal Brompton Hospital

    London, SW3 6NP

Other trials in United Kingdom

Bulgaria1 site

  • University MHAT "Sv. Anna"

    Sofia, Sofia-Grad, 1750

Other trials in Bulgaria

Germany1 site

  • Universitätsklinikum Giessen und Marburg

    Giessen, 35392

Other trials in Germany

Latvia1 site

  • P.Stradina Clinical University Hospital

    Riga, LV-1002

Other trials in Latvia

Moldova1 site

  • Spitalul Clinic Republican

    Chisinau, MD2025

Other trials in Moldova

About this study

Official title: A Phase 2, Dose-Ranging, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Rodatristat Ethyl in Patients With Pulmonary Arterial Hypertension

The purpose of this study is to assess the safety and efficacy of Rodatristat Ethyl in pulmonary arterial hypertension (PAH) patients.

Read the detailed description

Rodatristat Ethyl is a peripherally restricted TPH inhibitor being studied as a potential treatment for PAH. This dose-ranging, randomized, double-blind, placebo-controlled, multicenter study will evaluate the effect of Rodatristat Ethyl from baseline on pulmonary vascular resistance as measured at right heart catheterization.

Patients will be enrolled into a main study with an option to enroll into an open label extension.

The study is expected to enroll patients in the USA, Canada and Europe.

Being tested
rodatristat ethyl 300 mg tablet BID, rodatristat ethyl 600 mg BID, Placebo
Keywords
PAH
Medical subject headings
Pulmonary Arterial Hypertension

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • rodatristat ethyl 300 mg tablet BIDDrug

    rodatristat ethyl 300 mg tablet + matching placebo tablet twice daily on top of standard of care

  • rodatristat ethyl 600 mg BIDDrug

    2 rodatristat ethyl 300 mg tablets twice daily on top of standard of care

  • PlaceboDrug

    2 matching placebo tablets on top of standard of care

Study groups in NCT04712669
Study groupRoleWhat they receive
Rodatristat Ethyl 300 mg BIDExperimentalMAIN study: Rodatristat ethyl 300 mg and placebo tablet BID + standard of care medication(s) taken for 24 weeks
Receives: Drug: rodatristat ethyl 300 mg tablet BID
Rodatristat Ethyl 600 mg BIDExperimentalMAIN study:Rodatristat ethyl two 300 mg tablets BID + standard of care medication(s) taken for 24 weeks
Receives: Drug: rodatristat ethyl 600 mg BID
PlaceboPlacebo comparatorMAIN study: Matching two placebo tablets BID+ standard of care medication(s) taken for 24 weeks
Receives: Drug: Placebo
Placebo-Rodatristat Ethyl 300 mgExperimentalSubjects whose actual treatment group is Placebo in the double-blind phase (Main Study) and received Rodatristat ethyl two 300 mg tablets BID in the open-label phase
Receives: Drug: rodatristat ethyl 300 mg tablet BID
Placebo-Rodatristat Ethyl 600 mgExperimentalSubjects whose actual treatment group is Placebo in the double-blind phase (Main Study) and received Rodatristat ethyl two 600 mg tablets BID in the open-label phase
Receives: Drug: rodatristat ethyl 600 mg BID
Rodatristat Ethyl 300 mg-Rodatristat Ethyl 300 mgExperimentalSubjects whose actual treatment group is Rodatristat ethyl two 300 mg in the double-blind phase (Main Study) and received Rodatristat ethyl two 300 mg tablets BID in the open-label phase
Receives: Drug: rodatristat ethyl 300 mg tablet BID
Rodatristat Ethyl 300 mg-Rodatristat Ethyl 600 mgExperimentalSubjects whose actual treatment group is Rodatristat ethyl two 300 mg in the double-blind phase (Main Study) and received Rodatristat ethyl two 600 mg tablets BID in the open-label phase
Receives: Drug: rodatristat ethyl 600 mg BID
Rodatristat Ethyl 600 mg-Rodatristat Ethyl 600 mgExperimentalSubjects whose actual treatment group is Rodatristat ethyl two 600 mg in the double-blind phase (Main Study) and received Rodatristat ethyl two 600 mg tablets BID in the open-label phase
Receives: Drug: rodatristat ethyl 600 mg BID

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percent Change From Baseline of Pulmonary Vascular Resistance (PVR) at Week 24

    Pulmonary vascular resistance (PVR) was measured by right heart catheterization (RHC)

    Measured: 24 Weeks

Secondary outcomes (3)

  1. Change From Baseline in World Health Organization (WHO) Functional Class (FC)

    PAH functional disease severity is classified in 4 classes (I to IV) according to World Health Organization (WHO).

    I:Patients with pulmonary hypertension (PH) but without resulting limitation of physical act.Ordinary physical act does not cause undue dyspnea,fatigue,chest pain,near syncope.

    II:Patients with PH with slight limitation of physical act.Ordinary physical act causes undue dyspnea or fatigue, chest pain,or near syncope.

    III:Patients with PH with marked limitation of physical act.Less than ordinary act causes undue dyspnea or fatigue,chest pain,or near syncope.

    IV:Patients with PH with inability to carry out any physical act without symptoms,manifest signs of right-heart failure.Dyspnea and/or fatigue may even be present at rest.Discomfort is increased by physical act

    -II, -I:Patients condition improved at WK24 compared with baseline II, I:Patients condition worsened at WK24 compared with baseline No change:Patients condition not changes at WK24 compare with baseline

    Measured: 24 Weeks

  2. Change From Baseline in Six-minute Walk Distance (6MWD)

    The six-minute walk distance (6MWD) is a simple, commonly used, standardized measure of functional exercise capacity and endurance. It is a commonly used measure of efficacy in PAH clinical studies.

    Measured: 24 Weeks

  3. Change From Baseline in N-terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP) Levels

    N-terminal prohormone of brain natriuretic peptide (NT-proBNP) is a strong predictor of disease progression and mortality in PAH patients. Current PAH treatment guidelines recommend measurement of NT-proBNP levels for both risk assessment and longitudinal follow up. NT-proBNP levels are also a good marker of response to treatment.

    Measured: 24 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Jan 2021
  2. Study start15 Mar 2021
  3. Primary completion5 Jun 2023
  4. Study completion28 Aug 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Following screening assessments, Patients will be enrolled into 1 of 3 treatment arms in a core double blind phase in 1:1:1 randomization. Subjects are allowed to participant into open label extension phase upon completion of double blind phase.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
108 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04712669
Sponsor's protocol ID
RVT-1201-2002 / ELEVATE 2
Status last verified
Jun 2025
First submitted
12 Jan 2021
Last updated
4 Jun 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Lachant DJ, Sahay S, Block R, Ossip D, Denning J, Lazarus HM, Parsley E, White RJ. Rodatristat ethyl decreases activity in pulmonary arterial hypertension: results from the ELEVATE-2 trial. Ann Am Thorac Soc. 2026 May 1;23(5):754-761. doi: 10.1093/annalsats/aaoaf066.

    PubMed 41973984

  • Sitbon O, Skride A, Feldman J, Sahay S, Shlobin OA, McLaughlin V, Ghofrani HA, Langleben D, Parsley E, D'Souza G, Marmon T, Kamau-Kelley W, Jones R, Grewal R, Wring S, Palacios M, Naik H, Denning J, Lazarus HM, Humbert M. Safety and efficacy of rodatristat ethyl for the treatment of pulmonary arterial hypertension (ELEVATE-2): a dose-ranging, randomised, multicentre, phase 2b trial. Lancet Respir Med. 2024 Nov;12(11):865-876. doi: 10.1016/S2213-2600(24)00226-1. Epub 2024 Sep 19.

    PubMed 39307144

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing rodatristat ethyl 300 mg tablet BID and rodatristat ethyl 600 mg BID in people with Pulmonary Arterial Hypertension, run by Altavant Sciences GmbH at 64 sites in 17 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 64 sites in United States, Canada, France, Poland, Italy and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change From Baseline of Pulmonary Vascular Resistance (PVR) at Week 24 (time frame: 24 Weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04712669, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2025; AzTrials last refreshed its copy on 26 Oct 2026.

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