Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects

NCT ID: NCT03153137

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

142 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-08-14

Study Completion Date

2021-07-26

Brief Summary

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The primary objective is to assess the effect of macitentan 10 mg as compared to placebo on exercise capacity through cardiopulmonary exercise testing.

Detailed Description

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Conditions

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Congenital Heart Disease

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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Macitentan

Macitentan 10 mg per day; film-coated tablet; oral use

Group Type EXPERIMENTAL

Macitentan 10 mg

Intervention Type DRUG

film-coated tablet; oral use

Placebo

film-coated tablet; oral use

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

film-coated tablet; oral use

Interventions

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Macitentan 10 mg

film-coated tablet; oral use

Intervention Type DRUG

Placebo

film-coated tablet; oral use

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Written informed consent/assent from the subject and/or a legal representative prior to initiation of any study-mandated procedures
* Fontan-palliated subjects with either intra-atrial lateral tunnel total cavopulmonary connection (LT-TCPC), or extra cardiac tunnel TCPC (EC-TCPC) surgery \> 1 year before Screening. Either LT- or EC-TCPC can be primary or secondary to atrio-pulmonary connection
* New York Heart Association (NYHA) functional class (FC) II or III (assessed by the investigator using the Specific Activity Scale
* Women of childbearing potential must have a negative serum pregnancy test use reliable contraception

Exclusion Criteria

* Pattern of Fontan circulation severity
* Deterioration of the Fontan-palliated condition.
* Limitations to Cardiopulmonary exercise testing (CPET)
* Peak VO2 \< 15 mL/kg/min.
* Any known factor or disease that may interfere with treatment compliance or full participation in the study
Minimum Eligible Age

12 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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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

UCLA

Los Angeles, California, United States

Site Status

Massachusetts General Hospital Heart Center

Boston, Massachusetts, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

Providence Medical Research Providence Health Care

Spokane, Washington, United States

Site Status

Royal Adelaide Hospital

Adelaide, , Australia

Site Status

Royal Prince Alfred Hospital

Camperdown, , Australia

Site Status

The Prince Charles Hospital, Adult Congenital Heart Disease Unit

Chermside, , Australia

Site Status

Royal Children's Hospital

Parkville, , Australia

Site Status

Queensland CHILDREN'S HOSPITAL

South Brisbane, , Australia

Site Status

Westmead Hospital

Westmead, , Australia

Site Status

CHU de Quebec Universite Laval

Québec, Quebec, Canada

Site Status

Beijing Anzhen Hospital

Beijing, , China

Site Status

Beijing Fuwai Hospital

Beijing, , China

Site Status

Shanghai Childrens Medical Center

Shanghai, , China

Site Status

Wuhan Asia Heart Hospital

Wuhan, , China

Site Status

Fakultni nemocnice v Motole

Prague, , Czechia

Site Status

Rigshospitalet Kardiologisk Klinisk

Copenhagen, , Denmark

Site Status

CHU Arnaud de Villeneuve

Montpellier, , France

Site Status

Hôpital Necker - Enfants Malades

Paris, , France

Site Status

Hôpital Cardiologique Du Haut-Lévêque

Pessac, , France

Site Status

Deutsches Herzzentrum Berlinklinik Für Angeborene Herzfehler

Berlin, , Germany

Site Status

Deutsches Herzzentrum München

München, , Germany

Site Status

Auckland City Hospital

Auckland, , New Zealand

Site Status

Uniwersyteckie Centrum Kliniczne

Gdansk, , Poland

Site Status

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, , Poland

Site Status

Krakowski Szpital Specjalityczny im Jana Pawla II Oddzial Kliniczny Chorob Serca i Naczyn

Krakow, , Poland

Site Status

Wojewodzki Szpital Specjalistyczny We Wroclawiu

Wroclaw, , Poland

Site Status

National Taiwan University Hospital

Taipei, , Taiwan

Site Status

Queen Elizabeth Hospital

Birmingham, , United Kingdom

Site Status

Birmingham Children's Hospital

Birmingham, , United Kingdom

Site Status

Countries

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United States Australia Canada China Czechia Denmark France Germany New Zealand Poland Taiwan United Kingdom

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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2016-003320-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AC-055H301

Identifier Type: -

Identifier Source: org_study_id

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