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RESEARCH

Clinical Trials in Canada

In the past couple of decades, pulmonary hypertension medical research has evolved immensely throughout the world. Medical professionals are delving deeper into the science and uncovering new information every day. And while this has taken place internationally, we are incredibly fortunate to have leading research and clinical trials taking place right here in Canada.

How can I participate in pulmonary hypertension research?

A clinical trial is a research study that evaluates the safety and effects of one or more treatments on actual patients.

Patient participation in research is essential to making scientific advancements in the diagnosis and treatment of pulmonary hypertension. There are unique challenges associated with rare disease clinical trials, partly because patients are fewer and scattered around the globe. As a result, studies for rare disease research must find ways to adapt their designs without compromising the quality of the research.

Numerous early-stage and clinical-stage research studies are currently taking place in research centres throughout Canada. Check out the list below for more information on studies that are currently recruiting participants. For additional information, speak to your pulmonary hypertension team or visit ClinicalTrials.gov.

If you are in Quebec, you can learn more about clinical trials and how to access them at Clinical Trials Québec.

Disclaimer: This information is updated on a rolling basis. PHA Canada is not responsible for errors or omissions.

Thanks to advancements in research, many pulmonary hypertension treatments are available in Canada. However, they aren’t always available through public funding in every province and territory. This means people in some parts of the country don’t have equal access to the vital treatments they need. Visit our advocacy section to learn more and join us in representing a united pulmonary hypertension community.

A Phase 2, Open-Label Extension Study to Investigate the Safety and Efficacy Of PF-07868489 Administered to Adult Participants with Pulmonary Arterial Hypertension

Pfizer

Population:

PAH

Start Date:

November 10, 2025

End Date:

July 18, 2028

II

Phase:

STUDY SUMMARY

The purpose of this study is to learn about the long-term safety, tolerability and effects of the study medicine PF-07868489 for the possible treatment of PAH. This is an open-label study in which both the healthcare providers and the study participants are aware of the medicine being given. This study is an extension study with study medicine PF-07868489, allowing patients from an earlier clinical trial to continue participating to assess long-term benefits and safety of the medicine.

Sotatercept in Pulmonary Arterial Hypertension

University of Alberta

Population:

PAH

Start Date:

October 6, 2025

End Date:

January 1, 2030

IV

Phase:

STUDY SUMMARY

The goal of this clinical trial is to determine whether sotatercept is effective in improving diffusing capacity in patients with pulmonary arterial hypertension.

A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR)

AllRock Bio, Inc.

Population:

PAH & PH-ILD

Start Date:

October 1, 2025

End Date:

March 1, 2029

II

Phase:

STUDY SUMMARY

This study evaluates the effect of ROC-101 in adults with either Pulmonary Arterial Hypertension (PAH) or Pulmonary Hypertension Associated with Interstitial Lung Disease (ILD-PH). Each eligible participant will receive standard of care (SOC) plus ROC-101 for a 24-week treatment period, followed by a long-term extension period of the study through the end of the program or marketing approval/authorization.

Feasibility Trial for a Right Ventricular Failure Platform Trial (CRAVE)

University of Alberta

Population:

PH + RV dysfunction

Start Date:

February 1, 2025

End Date:

December 31, 2026

II

Phase:

STUDY SUMMARY

The primary objective of the CRAVE feasibility trial is to assess the feasibility of conducting a larger CRAVE platform trial by performing a randomized trial of 30 participants with pulmonary hypertension and right ventricular dysfunction, comparing empagliflozin or ranolazine plus standard of care to standard of care alone.

LEVosimendan to Improve Exercise Limitation in Patients With PH-HFpEF (LEVEL)

Tenax Therapeutics, Inc.

Population:

Group 2 PH

Start Date:

January 10, 2024

End Date:

October 1, 2028

III

Phase:

STUDY SUMMARY

This study will evaluate the efficacy of oral levosimendan compared with placebo in subjects with pulmonary hypertension due to heart failure with preserved ejection fraction (PH-HFpEF) as measured by the change in 6-Minute Walk Distance.

Functional Pulmonary Capillary Surface Area in BPA for CTEPH

Jewish General Hospital

Population:

CTEPH

Start Date:

January 30, 2023

End Date:

January 30, 2028

-

Phase:

STUDY SUMMARY

Many patients with chronic thromboembolic pulmonary hypertension (CTEPH) are treated with balloon pulmonary angioplasty [BPA] which mechanically opens the narrow pulmonary arteries. It is unclear how much downstream functional pulmonary capillary surface area [FCSA] is recovered during BPA. We plan to measure FCSAIn CTEPH patients, before and after a session of BPA.

PET/MR Pre- and Post Radiotherapy for Cardiopulmonary Dysfunction Evaluation

University Health Network

Population:

Radiotherapy induced PH

Start Date:

June 1, 2021

End Date:

June 1, 2025

-

Phase:

STUDY SUMMARY

This study aims to demonstrate correlation of combined PET/MR and plasma metabolomics markers in patients at risk of developing cardiopulmonary disfunction after radiotherapy.

A Long-term Follow-up Study of Sotatercept for PAH Treatment (SOTERIA)

Acceleron Pharma, Inc.

Population:

PAH

Start Date:

May 12, 2021

End Date:

December 7, 2028

III

Phase:

STUDY SUMMARY

The primary objective of this open-label, long-term follow-up study is to evaluate the long-term safety and tolerability of sotatercept when added to background pulmonary arterial hypertension (PAH) therapy in adult participants with PAH who have completed prior sotatercept studies. The secondary objective is to evaluate continued efficacy in adult participants with PAH who have completed prior sotatercept studies.

Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)

Duke University

Population:

PAH

Start Date:

March 5, 2020

End Date:

September 1, 2027

Observational

Phase:

STUDY SUMMARY

The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to evaluate the pharmacokinetics of understudied drugs currently being administered to children per standard of care as prescribed by their treating provider.

Reducing Right Ventricular Failure in Pulmonary Arterial Hypertension (RELIEVE-PAH)

V-Wave Ltd

Population:

PAH

Start Date:

February 14, 2020

End Date:

December 31, 2026

-

Phase:

STUDY SUMMARY

The objectives of the RELIEVE-PAH study are to obtain first-in-human experience with the study device in patients with severe pulmonary arterial hypertension, including evidence of initial safety, device performance and possible signals of clinical effectiveness.

Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry

University of Colorado

Population:

Pediatric PH

Start Date:

October 1, 2014

End Date:

December 1, 2031

Observational

Phase:

STUDY SUMMARY

Patients are being asked to be in this research study because medical researchers hope that by gathering information about a large number of children with pulmonary hypertension over time, their understanding of the disease process will increase and lead to better treatment. Investigators believe that pulmonary hypertension in children is different than pulmonary hypertension in adults and this study will help us understand those differences.

A Study Evaluating the Long-Term Efficacy and Safety of Ralinepag in Subjects With PAH Via an Open-Label Extension

United Therapeutics

Population:

PAH

Start Date:

September 23, 2019

End Date:

December 31, 2026

III

Phase:

STUDY SUMMARY

Study ROR-PH-303, ADVANCE EXTENSION, is an open-label extension study for participants with WHO Group 1 pulmonary arterial hypertension who have participated in another Phase 2 or Phase 3 study of ralinepag.

A Clinical Study of Sotatercept in People With Pulmonary Arterial Hypertension (MK-7962-031/LIGHTRAY EXTENSION)

Merck Sharp & Dohme LLC

Population:

PAH

Start Date:

May 12, 2025

End Date:

June 16, 2028

II

Phase:

STUDY SUMMARY

This is an extension study, which means people who took part in MK-7962-024 (LIGHTRAY) may be able to join this study. In this extension study, people will get weight-banded doses of sotatercept. The main goal of this study is to learn about the safety of weight-banded doses of sotatercept and if people tolerate it over a longer period of time.

Extension Study of Sotatercept in People With Pulmonary Hypertension (HARMONIZE)

Merck Sharp & Dohme LLC

Population:

Group 2 PH

Start Date:

April 16, 2025

End Date:

March 30, 2029

II

Phase:

STUDY SUMMARY

This is an extension study, which means people who took part in a certain study on sotatercept for combined postcapillary and precapillary pulmonary hypertension (Cpc-PH) may be able to join this study. This study focuses on Cpc-PH that is caused by heart failure with preserved ejection fraction (HFpEF). Researchers want to know if the study treatment, sotatercept, can treat people with Cpc-PH caused by HFpEF. The main goal of this extension study is to learn about the long-term safety of sotatercept and if people tolerate it over a longer period of time.

Open-label Extension Study of Seralutinib in Adult Subjects With PAH (PROSERA-EXT)

GB002, Inc., a subsidiary of Gossamer Bio

Population:

PAH

Start Date:

September 3, 2024

End Date:

December 1, 2026

III

Phase:

STUDY SUMMARY

This open-label extension study will evaluate the long-term safety, tolerability and efficacy of orally inhaled seralutinib in subjects who have completed a previous seralutinib study

TrEatment Approach in the Multimodal Era Registry (TEAM)

International CTEPH Association

Population:

CTEPH

Start Date:

April 12, 2023

End Date:

April 1, 2028

Observational

Phase:

STUDY SUMMARY

The goal of this observational patient registry is to learn how expert centers treat patients with chronic thromboembolic pulmonary hypertension (CTEPH).

A Study of Sotatercept for the Treatment of Combined Postcapillary and Precapillary Pulmonary Hypertension Due to HFpEF (CADENCE)

Acceleron Pharma, Inc.

Population:

Group 2 PH

Start Date:

December 29, 2021

End Date:

March 30, 2026

II

Phase:

STUDY SUMMARY

This is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of sotatercept versus placebo in adults with combined postcapillary and precapillary pulmonary hypertension (Cpc-PH) due to heart failure with preserved ejection fraction (HFpEF). Efficacy is measured by change from baseline in pulmonary vascular resistance (PVR, primary endpoint) and 6-minute walk distance (6MWD, key secondary endpoint).

Hemodynamic Testing Strategies to Assess Pulmonary Hypertension in Heart Failure

Mount Sinai Hospital

Population:

Group 2 PH

Start Date:

October 19, 2021

End Date:

June 1, 2025

Observational

Phase:

STUDY SUMMARY

The goal of this observational study is to learn about heart function and high lung blood pressure in patients with heart failure. The main question it aims to answer is whether we can improve the ability to predict patients who will have complications after they receive treatment including heart transplant and left ventricular assist devices.

Outcome Study Assessing a 75 mg Dose of Macitentan in Patients With Pulmonary Arterial Hypertension (UNISUS)

Actelion

Population:

PAH

Start Date:

June 30, 2020

End Date:

February 20, 2029

III

Phase:

STUDY SUMMARY

The purpose of this study is to demonstrate superiority of macitentan 75 milligrams in prolonging the time to the first clinical events committee-adjudicated morbidity or mortality event in participants with symptomatic pulmonary arterial hypertension compared to macitentan 10 mg.

A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension (SALTO)

Actelion

Population:

Pediatric PH

Start Date:

January 16, 2020

End Date:

October 1, 2027

III

Phase:

STUDY SUMMARY

The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with pulmonary arterial hypertension (PAH) in comparison to placebo.

A Clinical Study to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension
Conditions

Actelion

Population:

Pediatric PAH

Start Date:

July 23, 2018

End Date:

December 31, 2026

II

Phase:

STUDY SUMMARY

The purpose of this study to confirm the selexipag starting dose(s), selected based on pharmacokinetic (PK) extrapolation from adults, that leads to similar exposure as adults doses in children from greater than or equal to 2 to less than 18 years of age with pulmonary arterial hypertension (PAH), by investigating the PK of selexipag and its active metabolite ACT-333679 in this population.

A Clinical Study of Sotatercept (MK-7962) in People With Pulmonary Arterial Hypertension (LIGHTRAY)

Merck Sharp & Dohme LLC

Population:

PAH

Start Date:

November 14, 2024

End Date:

November 20, 2025

II

Phase:

STUDY SUMMARY

The goal of the study is to learn what happens to different doses of sotatercept in a person's body over time when it is given using weight-banded doses compared to weight-based doses. There may be differences in how the medicine works with the new dosing method (weight-banded dosing) being studied in this trial. The second goal is to lear about the safety of sotatercept and if people tolerate it.

Efficacy and Safety of Seralutinib in Adult Subjects With PAH (PROSERA)

Gossamer Bio

Population:

PAH

Start Date:

December 28, 2023

End Date:

December 22, 2025

III

Phase:

STUDY SUMMARY

The PROSERA Study is a Phase 3 clinical research study designed to help researchers understand whether an inhaled investigational product called seralutinib may be an effective and safe future treatment for adults with pulmonary arterial hypertension. The primary objective of the study is to determine the effect of seralutinib on improving exercise capacity in subjects with WHO Group 1 PAH who are FC II or III. The secondary objective for this trial is to determine time to clinical worsening.

A Study of AZD3427 in Participants With Heart Failure and Pulmonary Hypertension Group 2 (Re-PHIRE)

AstraZeneca

Population:

Group 2 PH

Start Date:

April 24, 2023

End Date:

August 25, 2025

II

Phase:

STUDY SUMMARY

This study is intended to assess the ability of AZD3427 to reduce pulmonary vascular resistance (PVR) after 24 weeks of treatment in participants with heart failure (HF) and pulmonary hypertension (PH) Group 2

Mono vs. Dual Therapy for Pediatric Pulmonary Arterial Hypertension

Johns Hopkins University

Population:

Pediatric PAH

Start Date:

August 1, 2022

End Date:

December 31, 2025

III

Phase:

STUDY SUMMARY

The investigators' central hypothesis is that early combination therapy with two pulmonary-arterial-hypertension-specific oral therapies that have been shown to be well tolerated in the pediatric population, sildenafil and bosentan, will result in better World Health Organization functional class at 12 months after initiation of PAH treatment than therapy with sildenafil alone.

A Study of the Efficacy and Safety of MK-5475 in Participants With Pulmonary Arterial Hypertension (INSIGNIA-PAH)

Merck Sharp & Dohme LLC

Population:

PAH

Start Date:

May 19, 2021

End Date:

July 2, 2026

II, III

Phase:

STUDY SUMMARY

This was a two-part (Phase 2/3) study of MK-5475, an inhaled soluble guanylate cyclase stimulator, in participants with pulmonary arterial hypertension. Phase 2 assessed three different doses of MK-5475 compared to placebo. Phase 3’s purpose was to confirm the efficacy, safety, and tolerability of MK-5475 at the selected dose compared to placebo during a 12 week base period followed by an extension period of up to 5 years.

A Study of Rodatristat Ethyl in Patients With Pulmonary Arterial Hypertension (ELEVATE 2)

Altavant Sciences GmbH

Population:

PAH

Start Date:

March 15, 2021

End Date:

August 28, 2023

II

Phase:

STUDY SUMMARY

The purpose of this study was to assess the safety and efficacy of rodatristat ethyl in pulmonary arterial hypertension (PAH) patients. Rodatristat ethyl is a peripherally restricted tryptophan hydroxylase inhibitor being studied as a potential treatment for PAH.

A Study of Sotatercept for the Treatment of Pulmonary Arterial Hypertension (STELLAR)

Acceleron Pharma, Inc.

Population:

PAH

Start Date:

January 25, 2021

End Date:

December 6, 2022

III

Phase:

STUDY SUMMARY

The objectives of this study were to evaluate the efficacy and safety of sotatercept treatment (plus background PAH therapy) versus placebo (plus background PAH therapy) at 24 weeks in adults with pulmonary arterial hypertension.

GB002 in Adult Subjects With Pulmonary Arterial Hypertension

GB002, Inc., a subsidiary of Gossamer Bio

Population:

PAH

Start Date:

November 12, 2020

End Date:

November 1, 2022

II

Phase:

STUDY SUMMARY

The primary objective for this trial was to determine the effect of GB002 (seralutinib) on improving pulmonary hemodynamics in subjects with World Health Organization Group 1 PAH who are Functional Class II and III. The secondary objective for this trial was to determine the effect of seralutinib on improving exercise capacity in this population.

Home-based Exercise Training in Patients With Pulmonary Arterial Hypertension: Effect on Skeletal Muscular Function and Metabolism

Laval University

Population:

PAH

Start Date:

March 1, 2020

End Date:

March 1, 2021

-

Phase:

STUDY SUMMARY

This study evaluated the impact of a 12 week home-based rehabilitation program on peripheral muscle function and metabolism, focusing on lipid infiltration, oxidative metabolism and epigenetic factors that can be involved in metabolic syndrome, in patients with pulmonary arterial hypertension.

Apabetalone for Pulmonary Arterial Hypertension: a Pilot Study (APPRoAcH-p)

Steeve Provencher

Population:

PAH

Start Date:

August 22, 2019

End Date:

December 13, 2021

I

Phase:

STUDY SUMMARY

Extended the investigator's preclinical findings on the role of epigenetics and DNA damage and Bromodomain-Containing Protein 4 (BRD4) inhibition as a therapy for a devastating disease, pulmonary arterial hypertension.

Clinical Study to Compare the Efficacy and Safety of Macitentan and Tadalafil Monotherapies With the Corresponding Fixed-dose Combination Therapy in Subjects With Pulmonary Arterial Hypertension (A DUE)

Actelion

Population:

PAH

Start Date:

July 29, 2019

End Date:

September 27, 2024

III

Phase:

STUDY SUMMARY

This study aimed to demonstrate that the fixed-dose combination (FDC) of macitentan and tadalafil is more effective than therapy with 10 mg of macitentan alone or 40 mg of tadalafil alone. This phase 3 study evaluated the efficacy and safety at 16 weeks of an FDC (macitentan 10 mg and tadalafil 40 mg) against these two pulmonary-arterial-hypertension-approved therapies given as monotherapy to further confirm the added value of the FDC.

Skeletal Muscle Mitochondrial Abnormalities and the Metabolic Syndrome in PAH

Laval University

Population:

PAH

Start Date:

June 1, 2019

End Date:

April 15, 2023

Observational

Phase:

STUDY SUMMARY

The investigators looked at whether or not metabolic syndrome seen in pulmonary arterial hypertension patients impairs mitochondrial functions through an IRS1/PPARg/PGC1-dependent mechanism, which would ultimately decrease skeletal muscle function and perfusion, and thus overall exercise capacity.

Dyspnea in Chronic Thromboembolic Pulmonary Hypertension

Queen's University

Population:

CTEPH

Start Date:

November 1, 2018

End Date:

January 1, 2023

Observational

Phase:

STUDY SUMMARY

Using new sophisticated technology, the investigators will determine the root causes of perceived breathing difficulty. The investigators will test the idea that breathlessness is fundamentally the result of increased drive to breathe from control centers in the brain. The investigators will measure drive to breathe by measuring the electrical activity descending from the brain to the main muscle of breathing - the diaphragm.

A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients (ADVANCE OUTCOMES)

United Therapeutics

Population:

PAH

Start Date:

August 30, 2018

End Date:

December 31, 2025

III

Phase:

STUDY SUMMARY

Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization Group 1 PAH.

Inflammatory Biomarkers of Pediatric Pulmonary Hypertension

The Hospital for Sick Children

Population:

Pediatric PH

Start Date:

June 20, 2018

End Date:

December 1, 2023

Observational

Phase:

STUDY SUMMARY

To investigate if the inflammatory protein, high mobility group box 1 (HMGB1), along with other inflammatory mediators, is elevated in pediatric patients with congenital heart disease (CHD) and pulmonary hypertension as compared to those with CHD alone, or with healthy controls.

Spironolactone Therapy in Chronic Stable Right Heart Failure Trial (STAR-HF)

Ottawa Heart Institute Research Corporation

Population:

Right heart failure

Start Date:

April 1, 2018

End Date:

May 1, 2024

IV

Phase:

STUDY SUMMARY

The purpose of this study was to evaluate the safety, tolerability and mechanistic effects of spironolactone, an aldosterone receptor antagonist, on sympathetic nervous system activity and right heart function and remodeling in patients with chronic right heart failure.

A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (TOMORROW)

Actelion

Population:

Pediatric PAH

Start Date:

October 24, 2017

End Date:

November 27, 2025

III

Phase:

STUDY SUMMARY

This is a prospective, multicenter, open-label, randomized, controlled, parallel Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of macitentan in children with pulmonary arterial hypertension (PAH).

Novel Echocardiographic Methods for Early Identification of Neonates at Risk for Chronic Pulmonary Hypertension

Mount Sinai Hospital

Population:

Newborn PH

Start Date:

August 31, 2017

End Date:

April 1, 2024

Observational

Phase:

STUDY SUMMARY

The primary objective of this study was to develop new, sensitive, quantitative echocardiographic diagnostic criteria which will allow for the identification of extreme preterm neonates suffering from significantly high pressure in their pulmonary blood vessels, early in postnatal course, when the disease is likely to be most amenable to preventative/curative treatment.

Upfront Combination Pulmonary Arterial Hypertension Therapy

University of Calgary

Population:

PAH

Start Date:

April 26, 2016

End Date:

January 31, 2021

IV

Phase:

STUDY SUMMARY

To evaluate the safety and efficacy of first-line combination therapy using riociguat with ambrisentan in patients with Pulmonary Arterial Hypertension (PAH).

Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP-2)

Association for Pediatric Pulmonary Hypertension

Population:

Pediatric PH

Start Date:

August 1, 2015

End Date:

December 31, 2022

Observational

Phase:

STUDY SUMMARY

The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood.

An Open-Label, Long-Term Study of Oral Treprostinil in Subjects With Pulmonary Arterial Hypertension

United Therapeutics

Population:

PAH

Start Date:

May 1, 2012

End Date:

August 12, 2021

III

Phase:

STUDY SUMMARY

This study was an international, multi-center, open-label study designed to provide oral treprostinil (UT-15C) to eligible subjects with pulmonary arterial hypertension who had completed the TDE-PH-310 study. The purpose of this study is to assess the long-term safety of UT-15C and to assess the effects of long-term treatment with UT-15C on exercise capacity.

ACT-293987 in Pulmonary Arterial Hypertension

Actelion

Population:

PAH

Start Date:

July 7, 2010

End Date:

August 26, 2021

III

Phase:

STUDY SUMMARY

Long-term, single-arm, multicenter, open-label extension, Phase 3 study, to evaluate the safety and tolerability of ACT-293987 in patients with PAH who participated in the double-blind study AC-065A302 (GRIPHON)

A Study to Test the Effects of Riociguat in Patients With Pulmonary Hypertension Associated With Left Ventricular Systolic Dysfunction Conditions

Bayer

Population:

Group 2 PH

Start Date:

April 14, 2010

End Date:

July 23, 2025

II

Phase:

STUDY SUMMARY

The aim of this study was to assess whether increasing oral doses of riociguat are safe and improve the well-being, symptoms and outcome in patients with pulmonary hypertension associated with left ventricular systolic dysfunction.

A Study of HS135 for the Treatment of Pulmonary Arterial Hypertension in Adults

35Pharma Inc.

Population:

PAH

Start:

October 23, 2024

End:

March 31, 2025

I

Phase:

STUDY SUMMARY

A phase 1B open-label, multiple ascending dose study assessing the pharmacokinetics, safety, pharmacodynamics, and efficacy of HS135 added to background pulmonary arterial hypertension therapy.

Study of Sotatercept in Newly Diagnosed Intermediate- and High-Risk PAH Participants (HYPERION)

Acceleron Pharma, Inc.

Population:

PAH

Start:

March 18, 2022

End:

April 11, 2025

III

Phase:

STUDY SUMMARY

The objective of this study was to evaluate the effects of sotatercept treatment (plus background pulmonary arterial hypertension (PAH) therapy) versus placebo (plus background PAH therapy) on time to clinical worsening in participants who are newly diagnosed with pulmonary arterial hypertension and are at intermediate or high risk of disease progression. The trial was stopped early so all patients in the trial, including those in the placebo arm, can benefit from sotatercept. Patients in the trial will be able to receive sotatercept through the ongoing SOTERIA extension study.

A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (IMPAHCT)

Aerovate Therapeutics

Population:

PAH

Start:

December 2, 2021

End:

August 9, 2024

II, III

Phase:

STUDY SUMMARY

The IMPAHCT study evaluated the safety and efficacy of AV-101 (dry powder inhaled imatinib) in patients with pulmonary arterial hypertension (PAH). The study was stopped early because it demonstrated that while inhaled imatinib was well tolerated, it did not prove to be efficacious at any of the doses of AV-101 evaluated in the study. The follow-up long-term extension study (IMPAHCT-FUL) was also cancelled.

A Study of Sotatercept in Participants With PAH WHO FC III or FC IV at High Risk of Mortality (ZENITH)

Acceleron Pharma, Inc.

Population:

PAH

Start:

December 1, 2021

End:

November 25, 2024

III

Phase:

STUDY SUMMARY

The ZENITH study examined whether adding sotatercept to people's ongoing therapies reduced the risk of death, lung transplantation, or hospitalizations for pulmonary arterial hypertension (PA) in PAH patients in functional class III or IV at high risk of mortality. Because of its "overwhelming efficacy," the trial was stopped early so all patients in the trial, including those in the placebo arm, can benefit from sotatercept. Patients in the trial will be able to receive sotatercept through the ongoing SOTERIA extension study.

A Study of Real-world Cohort of Pulmonary Arterial Hypertension Participants (CARE PAH)

Actelion

Population:

PAH

Start:

October 14, 2021

End:

December 20, 2022

IV

Phase:

STUDY SUMMARY

The purpose of this study was to describe the time to all-cause death and time to death due to pulmonary arterial hypertension (PAH) or first hospitalization due to PAH in the overall study population and within each cohort. The study was stopped early.

A Study in Participants With Sarcoidosis-associated Pulmonary Hypertension (SAPH) to Assess the Efficacy and Safety of Oral Selexipag (SPHINX)

Actelion

Population:

Sarcoidosis-associated PH

Start:

February 26, 2021

End:

April 19, 2023

II

Phase:

STUDY SUMMARY

The aim of the study was to investigate whether selexipag could be helpful to treat patients with a form of PH called sarcoidosis-associated pulmonary hypertension (SAPH). The study stopped early due to a lower-than-expected recruitment rate.

A Study to Evaluate Efficacy and Safety of Macitentan 75 mg in Inoperable or Persistent/Recurrent Chronic Thromboembolic Pulmonary Hypertension

Actelion

Population:

CTEPH

Start:

July 7, 2020

End:

December 21, 2023

III

Phase:

STUDY SUMMARY

The purpose of the study was to evaluate the effect of macitentan 75 mg versus placebo on exercise capacity at Week 28 in participants with chronic thromboembolic pulmonary hypertension (CTEPH). The study was stopped for futility following a pre-planned interim analysis.

Olaparib for PAH: a Multicenter Clinical Trial (OPTION)

Laval University

Population:

PAH

Start:

November 20, 2019

End:

October 31, 2024

I, II

Phase:

STUDY SUMMARY

The primary objective of this Phase 1B study was to confirm the safety of using olaparib in PAH patients, and precise the sample size of a future Phase 2 trial. In addition to safety, efficacy signals would thus be assessed. The study stopped early due to limited recruitment and the end of funding.

A Study to Find Out if Selexipag is Effective and Safe in Patients With CTEPH When the Disease is Inoperable or Persistent/Recurrent After Surgery and/or Interventional Treatment

Actelion

Population:

CTEPH

Start:

January 23, 2019

End:

June 7, 2022

III

Phase:

STUDY SUMMARY

Due to the similarities between pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH) and the observed efficacy of other PAH medicines in CTEPH, it is believed that selexipag could benefit to patients with CTEPH. This study aimed to assess the efficacy and safety of selexipag in subjects with inoperable or persistent/recurrent CTEPH. It was stopped early because it did not show efficacy on the primary endpoint at a planned interim analysis.

Study of Angiogenic Cell Therapy for Progressive Pulmonary Hypertension (SAPPHIRE)

Northern Therapeutics

Population:

PAH

Start:

September 28, 2017

End:

November 1, 2023

II, III

Phase:

STUDY SUMMARY

The SAPPHIRE clinical trial sought to establish the efficacy and safety of repeated monthly dosing of autologous EPCs transfected with human eNOS (heNOS) in patients with symptomatic severe pulmonary arterial hypertension on available PAH-targeted medical therapy. The study was stopped early because of lower than anticipated enrollment.

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