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

NCT03153137 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: AC-055H3012016-003320-23
• Study Type: interventional
• Target: 142
• Locations: 12 countries
• Sites: 32

Brief Summary

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

Conditions

Congenital Heart Disease

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Peak Oxygen Uptake/Consumption (VO2) Up to Week 16

  Change from baseline in peak VO2 up to Week 16 was reported.

  Baseline up to Week 16

Secondary Outcomes (26)

• Change From Baseline in Peak VO2 Up to Week 52

  Baseline up to Week 52

• Change From Baseline in Mean Count Per Minute of Daily Physical Activity Measured by Accelerometer (PA-Ac) Up to Week 16

  Baseline up to Week 16

• Number of Participants With Treatment-emergent Serious Adverse Events (SAEs)

  Up to 56 weeks

• Number of Participants With Treatment-emergent Adverse Events (AEs)

  Up to 56 weeks

• Number of Participants With AEs Leading to Premature Discontinuation of Study Treatment

  Up to 56 weeks

Study Arms (2)

Macitentan

EXPERIMENTAL

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

Drug: Macitentan 10 mg

Placebo

PLACEBO COMPARATOR

film-coated tablet; oral use

Drug: Placebo

Interventions

Macitentan 10 mg DRUG

film-coated tablet; oral use

Macitentan

Placebo DRUG

film-coated tablet; oral use

Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Actelion: lead

Study Sites (32)

University of Alabama at Birmingham

Birmingham, Alabama, 35233-1935, United States

Location

UCLA

Los Angeles, California, 90095, United States

Location

Massachusetts General Hospital Heart Center

Boston, Massachusetts, 02114, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Texas Children's Hospital

Houston, Texas, 77030-2303, United States

Location

Providence Medical Research Providence Health Care

Spokane, Washington, 99202, United States

Location

Royal Adelaide Hospital

Adelaide, 5000, Australia

Location

Royal Prince Alfred Hospital

Camperdown, 2050, Australia

Location

The Prince Charles Hospital, Adult Congenital Heart Disease Unit

Chermside, 4032, Australia

Location

Royal Children's Hospital

Parkville, 3052, Australia

Location

Queensland CHILDREN'S HOSPITAL

South Brisbane, 4101, Australia

Location

Westmead Hospital

Westmead, 2145, Australia

Location

CHU de Quebec Universite Laval

Québec, Quebec, G1V 4G2, Canada

Location

Beijing Anzhen Hospital

Beijing, 100029, China

Location

Beijing Fuwai Hospital

Beijing, 100037, China

Location

Shanghai Childrens Medical Center

Shanghai, 200127, China

Location

Wuhan Asia Heart Hospital

Wuhan, 430022, China

Location

Fakultni nemocnice v Motole

Prague, 150 06, Czechia

Location

Rigshospitalet Kardiologisk Klinisk

Copenhagen, 2100, Denmark

Location

CHU Arnaud de Villeneuve

Montpellier, 34295, France

Location

Hôpital Necker - Enfants Malades

Paris, 75015, France

Location

Hôpital Cardiologique Du Haut-Lévêque

Pessac, 33604, France

Location

Deutsches Herzzentrum Berlinklinik Für Angeborene Herzfehler

Berlin, 13353, Germany

Location

Deutsches Herzzentrum München

München, 80636, Germany

Location

Auckland City Hospital

Auckland, 1640, New Zealand

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80 952, Poland

Location

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, 30-663, Poland

Location

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

Krakow, 31 202, Poland

Location

Wojewodzki Szpital Specjalistyczny We Wroclawiu

Wroclaw, 51 124, Poland

Location

National Taiwan University Hospital

Taipei, 100, Taiwan

Location

Queen Elizabeth Hospital

Birmingham, B15 2TH, United Kingdom

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

MeSH Terms

Conditions

Heart Defects, Congenital

Interventions

macitentan

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Senior Clinical Leader
• Organization: Actelion Pharmaceuticals Ltd

ClinicalTrialDisclosure@its.jnj.com

844-434-4210

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2017
• First Posted: May 15, 2017
• Study Start: August 14, 2017
• Primary Completion: June 30, 2021
• Study Completion: July 26, 2021
• Last Updated: March 30, 2025
• Results First Posted: September 13, 2022

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (1); CN (4); CA (1); AU (6); US (6); DK (1); DE (2); TW (1); GB (2); PL (4); NZ (1); FR (3)