The Efficacy and Safety of Initial Triple Versus Initial Dual Oral Combination Therapy in Patients With Newly Diagnosed Pulmonary Arterial Hypertension

NCT ID: NCT02558231

Last Updated: 2025-03-30

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

247 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-05-01

Study Completion Date

2020-04-20

Brief Summary

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The objective of this clinical trial is to compare the efficacy and safety of an initial triple oral treatment regimen (macitentan, tadalafil, selexipag) versus an initial dual oral treatment regimen (macitentan, tadalafil, placebo) in newly diagnosed, treatment-naïve patients with pulmonary arterial hypertension.

Detailed Description

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Conditions

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Pulmonary Arterial Hypertension

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Triple oral combination treatment

Macitentan, tadalafil, and selexipag

Group Type EXPERIMENTAL

Macitentan

Intervention Type DRUG

Used open-label in both arms, 10 mg tablet, 1 tablet u.i.d.

Tadalafil

Intervention Type DRUG

Used open-label in both arms, 20 mg tablet, 1-2 tablets u.i.d.

Selexipag

Intervention Type DRUG

Used double-blind in the triple oral treatment arm, 200 microgram tablet, 1-8 tablets b.i.d.

Dual oral combination treatment

Macitentan, tadalafil, and placebo

Group Type PLACEBO_COMPARATOR

Macitentan

Intervention Type DRUG

Used open-label in both arms, 10 mg tablet, 1 tablet u.i.d.

Tadalafil

Intervention Type DRUG

Used open-label in both arms, 20 mg tablet, 1-2 tablets u.i.d.

Interventions

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Macitentan

Used open-label in both arms, 10 mg tablet, 1 tablet u.i.d.

Intervention Type DRUG

Tadalafil

Used open-label in both arms, 20 mg tablet, 1-2 tablets u.i.d.

Intervention Type DRUG

Selexipag

Used double-blind in the triple oral treatment arm, 200 microgram tablet, 1-8 tablets b.i.d.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Signed informed consent prior to any study-mandated procedure.
2. Male or female ≥ 18 and ≤ 75 years of age at screening.
3. Initial PAH diagnosis \< 6 months prior to enrollment.
4. RHC performed between Day -28 and Day 1, meeting all the following criteria:

* Mean pulmonary artery pressure (mPAP) ≥ 25 mmHg.
* Pulmonary artery wedge pressure or left ventricular end-diastolic pressure ≤ 15 mmHg.
* PVR ≥ 480 dyn•sec/cm5 (≥ 6 Wood Units).
* Negative vasoreactivity test mandatory in idiopathic, heritable, and drug/toxin induced PAH (at this or a previous RHC).
5. Symptomatic PAH belonging to one of the following subgroups:

* Idiopathic.
* Heritable.
* Drug or toxin induced.
* Associated with one of the following: connective tissue disease; HIV infection; congenital heart disease.
6. 6-minute walk distance (6MWD) ≥ 50 m at screening.
7. Women of childbearing potential must not be pregnant, must perform regular pregnancy tests, and use reliable contraception.

Exclusion Criteria

1. Any PAH-specific drug therapy at any time.
2. Cardio pulmonary rehabilitation program based on exercise (planned, or started ≤ 12 weeks prior to Day 1).
3. Body mass index (BMI) \> 40 kg/m2 at screening.
4. Presence of three or more of the following risk factors for heart failure with preserved ejection fraction at screening:

* BMI \> 30 kg/m2.
* Diabetes mellitus of any type.
* Essential hypertension.
* Coronary artery disease, i.e., any of the following:

* History of stable angina or
* More than 50% stenosis in a coronary artery (by coronary angiography) or
* History of myocardial infarction or
* History of or planned coronary artery bypass grafting and/or coronary artery stenting.
5. Acute myocardial infarction ≤ 12 weeks prior to screening.
6. Stroke ≤ 12 weeks prior to screening.
7. Known permanent atrial fibrillation.
8. SBP \< 90 mmHg at screening or Day 1.
9. Ongoing or planned treatment with organic nitrates and/or doxazosin.
10. Presence of one or more of the following signs of relevant lung disease at any time up to screening:

* Diffusing capacity of the lung for carbon monoxide (DLCO) \< 40% of predicted (eligible only if no or mild interstitial lung disease on computed tomography).
* Forced vital capacity (FVC) \< 60% of predicted.
* Forced expiratory volume in one second (FEV1) \< 60% of predicted.
11. Known or suspected pulmonary veno-occlusive disease (PVOD).
12. Documented severe hepatic impairment (with or without cirrhosis) according to National Cancer Institute organ dysfunction working group criteria, defined as total bilirubin \> 3 × upper limit of the normal range (ULN) accompanied by aspartate aminotransferase (AST) \> ULN (assessed by central laboratory at screening); and/or Child-Pugh Class C.
13. Serum AST and/or alanine aminotransferase (ALT) \> 3 × ULN (assessed by central laboratory at screening).
14. Severe renal impairment (estimated creatinine clearance ≤ 30 mL/min/1.73 m2) assessed by central laboratory at screening.
15. Ongoing or planned dialysis.
16. Hemoglobin \< 100 g/L assessed by central laboratory at screening.
17. Known or suspected uncontrolled thyroid disease (hypo- or hyperthyroidism).
18. Loss of vision in one or both eyes because of non-arteritic ischemic optic neuropathy (NAION).
19. Treatment with strong inducers of cytochrome P450 3A4 (CYP3A4; e.g., carbamazepine, rifampin, rifampicin, rifabutin, rifapentin, phenobarbital, phenytoin, and St. John's wort) ≤ 28 days prior to Day 1.
20. Treatment with strong inhibitors of CYP3A4 (e.g., ketoconazole, itraconazole, voriconazole, clarithromycin, telithromycin, nefazodone, ritonavir, and saquinavir) and/or strong inhibitors of CYP2C8 (e.g., gemfibrozil) ≤ 28 days prior to Day 1.
21. Treatment with another investigational drug (planned, or taken ≤ 12 weeks prior to Day 1).
22. Hypersensitivity to any of the 3 study treatments or any excipient of their formulations.
23. Pregnancy, breastfeeding, or intention to become pregnant during the study.
24. Concomitant life-threatening disease with a life expectancy \< 12 months.
25. Alcohol abuse.
26. Any factor or condition likely to affect protocol compliance of the subject, as judged by the investigator.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Actelion

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Arizona Pulmonary Specialists, LTD

Phoenix, Arizona, United States

Site Status

UCSD Health Sciences

La Jolla, California, United States

Site Status

UCLA Medical Center

Los Angeles, California, United States

Site Status

Mayo Clinic Jacksonville

Jacksonville, Florida, United States

Site Status

Cleveland Clinic Florida

Weston, Florida, United States

Site Status

Piedmont Pulmonary and Critical Care Research

Atlanta, Georgia, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

University of Iowa Hospitals & Clinics

Iowa City, Iowa, United States

Site Status

Kentuckiana Pulmonary Associates

Louisville, Kentucky, United States

Site Status

LSU Health Sciences Center

New Orleans, Louisiana, United States

Site Status

Johns Hopkins School of Medicine

Baltimore, Maryland, United States

Site Status

Tufts Medical Center

Boston, Massachusetts, United States

Site Status

Boston University Medical Center

Boston, Massachusetts, United States

Site Status

Washington University School of Medicine

Saint Louis, Michigan, United States

Site Status

University of New Mexico Hospital

Albuquerque, New Mexico, United States

Site Status

The Christ Hospital

Cincinnati, Ohio, United States

Site Status

Allegheny General Hospital of Research

Pittsburgh, Pennsylvania, United States

Site Status

UPMC Presbyterian

Pittsburgh, Pennsylvania, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Houston Methodist Hospital

Houston, Texas, United States

Site Status

Royal Prince Albert Hospital

Camperdown, New South Wales, Australia

Site Status

St. Vincents Hospital Sydney

Darlinghurst, New South Wales, Australia

Site Status

LKH -Universität Klinkum Graz

Graz, , Austria

Site Status

Krankenhaus der Elisabethinen Linz

Linz, , Austria

Site Status

AKH Wien

Vienna, , Austria

Site Status

Hôpital Erasme

Brussels, , Belgium

Site Status

UZ Leuven - Campus Gasthuisberg

Leuven, , Belgium

Site Status

Vancouver General Hospital

Vancouver, British Columbia, Canada

Site Status

London Health Sciences Centre - Victoria Hospital

London, Ontario, Canada

Site Status

University of Toronto

Toronto, Ontario, Canada

Site Status

Jewish General Hospital

Montreal, Quebec, Canada

Site Status

Institut Universitaire de Cardiologie et de Pneumologie de Québec

Québec, Quebec, Canada

Site Status

University of Calgary

Calgary, , Canada

Site Status

University of Ottawa Heart Institute

Ottawa, , Canada

Site Status

Aarhus University Hospital Skejby

Aarhus, , Denmark

Site Status

Rigshospitalet Copenhagen

Copenhagen, , Denmark

Site Status

CHU de Bicêtre

Le Kremlin-Bicêtre, , France

Site Status

Universitätsklinikum Köln

Cologne, , Germany

Site Status

Unversitätsklinikum Carl Gustav Carus

Dresden, , Germany

Site Status

Universitätsklinikum Giessen

Giessen, , Germany

Site Status

Universitätsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Universitätsklinikum Heidelberg

Heidelberg, , Germany

Site Status

Universitätsklinikum Regensburg

Regensburg, , Germany

Site Status

Mater Misericordiae University Hospital

Dublin, , Ireland

Site Status

Ospedale Sant'Orsola

Bologna, , Italy

Site Status

VUmc Amsterdam

Amsterdam, , Netherlands

Site Status

Maastricht University Medical Center

Maastricht, , Netherlands

Site Status

Hospital Clinic de Barcelona

Barcelona, , Spain

Site Status

Hospital 12 de Octubre

Madrid, , Spain

Site Status

Skånes universitetssjukhus Lund

Lund, , Sweden

Site Status

Norrlands universitetssjukhus

Umeå, , Sweden

Site Status

Kardiologkliniken

Uppsala, , Sweden

Site Status

Universiätsspital Zürich

Zurich, , Switzerland

Site Status

Golden Jubilee National Hospital

Clydebank, , United Kingdom

Site Status

The Royal Free Hospital

London, , United Kingdom

Site Status

Royal Brompton Hospital

London, , United Kingdom

Site Status

Hammersmith Hospital

London, , United Kingdom

Site Status

Countries

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United States Australia Austria Belgium Canada Denmark France Germany Ireland Italy Netherlands Spain Sweden Switzerland United Kingdom

References

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Chin KM, Sitbon O, Doelberg M, Feldman J, Gibbs JSR, Grunig E, Hoeper MM, Martin N, Mathai SC, McLaughlin VV, Perchenet L, Poch D, Saggar R, Simonneau G, Galie N. Three- Versus Two-Drug Therapy for Patients With Newly Diagnosed Pulmonary Arterial Hypertension. J Am Coll Cardiol. 2021 Oct 5;78(14):1393-1403. doi: 10.1016/j.jacc.2021.07.057.

Reference Type DERIVED
PMID: 34593120 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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AC-065A308

Identifier Type: -

Identifier Source: org_study_id

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