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

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ClinicalTrials.gov Identifier: NCT02932410
Recruitment Status : Recruiting
First Posted : October 13, 2016
Last Update Posted : June 7, 2018
Sponsor:
Information provided by (Responsible Party):
Actelion

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.

Condition or disease Intervention/treatment Phase
Pulmonary Arterial Hypertension Drug: Macitentan Other: Standard-of-care Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 300 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : November 28, 2017
Estimated Primary Completion Date : July 14, 2022
Estimated Study Completion Date : July 14, 2022


Arm Intervention/treatment
Experimental: Macitentan
Macitentan is administered once daily via oral route
Drug: Macitentan
Dispersible tablet; Oral use
Other Name: ACT-064992

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.
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.




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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

  2. 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

  3. 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



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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02932410


  Show 90 Study Locations
Sponsors and Collaborators
Actelion

Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT02932410     History of Changes
Other Study ID Numbers: AC-055-312
First Posted: October 13, 2016    Key Record Dates
Last Update Posted: June 7, 2018
Last Verified: June 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Hypertension
Familial Primary Pulmonary Hypertension
Vascular Diseases
Cardiovascular Diseases
Hypertension, Pulmonary
Lung Diseases
Respiratory Tract Diseases
Macitentan
Endothelin A Receptor Antagonists
Endothelin Receptor Antagonists
Molecular Mechanisms of Pharmacological Action
Endothelin B Receptor Antagonists