Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02932410
Recruitment Status : Recruiting
First Posted : October 13, 2016
Last Update Posted : June 11, 2020
Sponsor:
Information provided by (Responsible Party):
Actelion

Tracking Information
First Submitted Date  ICMJE October 12, 2016
First Posted Date  ICMJE October 13, 2016
Last Update Posted Date June 11, 2020
Actual Study Start Date  ICMJE November 28, 2017
Estimated Primary Completion Date July 14, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 29, 2017)
Time to the first CEC-confirmed disease progression event [ Time Frame: Between randomization and end-of-study (EOS)/study closure; up to 6 years ]
Time to the first of the following CEC-confirmed disease progression events: • Death (all causes) • Atrial septostomy or Potts' anastomosis, or registration on lung transplant list • Hospitalization due to worsening PAH • Clinical worsening of PAH
Original Primary Outcome Measures  ICMJE
 (submitted: October 12, 2016)
Time to the first CEC-confirmed disease progression event [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
Time to the first of the following CEC-confirmed disease progression events: • Death (all causes) • Atrial septostomy or Potts' anastomosis, or registration on lung transplant list • Hospitalization due to worsening PAH • Clinical worsening of PAH
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 29, 2017)
  • Time to first CEC-confirmed hospitalization for PAH [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
    Time to first CEC-confirmed hospitalization for PAH occurring between randomization and EOS
  • Time to CEC-confirmed death due to PAH [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
    Time to CEC-confirmed death due to PAH occurring between randomization and EOS
  • Time to death (all causes) [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
    Time to death (all causes) occurring between randomization and Study Closure
Original Secondary Outcome Measures  ICMJE
 (submitted: October 12, 2016)
  • Time to first CEC-confirmed hospitalization for PAH occurring between randomization and EOS [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
  • Time to CEC-confirmed death due to PAH occurring between randomization and EOS [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
  • Time to death (all causes) occurring between randomization and Study Closure [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH)
Official Title  ICMJE A Multicenter, Open-label, Randomized, Event-driven Study to Assess Efficacy, Safety and Pharmacokinetics of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension
Brief Summary This is a prospective, multicenter, open-label, randomized, controlled, parallel group, group-sequential, event-driven Phase 3 study to evaluate efficacy, safety and pharmacokinetics (PK) of macitentan in children.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pulmonary Arterial Hypertension
Intervention  ICMJE
  • Drug: Macitentan
    Dispersible tablet; Oral use
    Other Name: ACT-064992
  • Other: Standard-of-care
    Standard-of-care as per site's clinical practice which may comprise treatment with PAH non-specific treatment and/or up to two PAH-specific medications excluding macitentan and i.v./s.c. prostanoids.
Study Arms  ICMJE
  • Experimental: Macitentan
    Macitentan is administered once daily via oral route
    Intervention: Drug: Macitentan
  • Standard-of-care
    Standard-of-care as per site's clinical practice which may comprise treatment with PAH non-specific treatment and/or up to two PAH-specific medications excluding macitentan and i.v./s.c. prostanoids.
    Intervention: Other: Standard-of-care
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 12, 2016)
300
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 14, 2022
Estimated Primary Completion Date July 14, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  1. Signed informed consent by the parent(s) or legally designated representative and assent from developmentally capable children prior to initiation of any study-mandated procedure.
  2. Males or females between ≥ 2 years and < 18 years of age.
  3. Subjects with body weight ≥ 10 kg at randomization.
  4. Pulmonary arterial hypertension (PAH) diagnosis, confirmed by historical right heart catheterization (mPAP ≥ 25 mmHg, and PAWP ≤ 15 mmHg, and PVRi > 3 WUxm2).
  5. PAH belonging to the Nice 2013 Updated Classification Group 1 (including subjects with Down syndrome) and of following etiologies:

    • idiopathic PAH
    • heritable PAH
    • PAH associated with congenital heart disease (CHD)
    • Drug or toxin induced PAH
    • PAH associated with HIV
    • PAH associated with connective tissue diseases (PAH-aCTD)
  6. WHO Functional class I to III.
  7. Females of childbearing potential must have a negative pregnancy test at Screening and at Baseline, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) up to the end of study (EOS).

Key Exclusion Criteria:

  1. Subjects with PAH due to portal hypertension, schistosomiasis, or with pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis, and persistent pulmonary hypertension of the newborn.
  2. Subjects with PAH associated with Eisenmenger syndrome, or with moderate to large left-to-right shunts.
  3. Subjects receiving a combination of > 2 PAH-specific treatments at randomization.
  4. Treatment with i.v. or s.c. prostanoids within 4 weeks before randomization, unless given for vasoreactivity testing.
  5. Hemoglobin or hematocrit <75% of the lower limit of normal range
  6. Serum AST and/or ALT > 3 times the upper limit of normal range'
  7. Pregnancy (including family planning) or breastfeeding.
  8. Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Study Contact 844-434-4210 JNJ.CT@sylogent.com
Listed Location Countries  ICMJE Australia,   Austria,   Canada,   China,   Colombia,   Finland,   France,   Hungary,   Israel,   Korea, Republic of,   Malaysia,   Mexico,   Philippines,   Poland,   Portugal,   Russian Federation,   South Africa,   Spain,   Thailand,   Ukraine,   United States,   Vietnam
Removed Location Countries Argentina,   Bulgaria
 
Administrative Information
NCT Number  ICMJE NCT02932410
Other Study ID Numbers  ICMJE AC-055-312
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Actelion
Study Sponsor  ICMJE Actelion
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Actelion
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP