Macitentan for Pulmonary Hypertension
(TOMORROW Trial)

Recruiting in Palo Alto (17 mi)

+85 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Actelion

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing macitentan, a drug that helps lower lung blood pressure, in children with pulmonary arterial hypertension. Macitentan has shown significant effectiveness in improving the condition of patients with pulmonary arterial hypertension.

Research Team

Eligibility Criteria

This trial is for children under 18 with pulmonary arterial hypertension (PAH), including those with Down Syndrome. They must have a confirmed PAH diagnosis and be in WHO Functional class I to III. Females of childbearing potential need negative pregnancy tests and must use contraception. Participants can't join if they have certain conditions like severe renal insufficiency, are on multiple PAH treatments, or have severe liver impairment.

Inclusion Criteria

I am under 18 years old.

I have been diagnosed with PAH confirmed by a heart catheter test.

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

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Exclusion Criteria

I haven't taken IV or SC prostanoids in the last 4 weeks, except for a specific test.

I am currently on more than two treatments for pulmonary arterial hypertension.

I have PAH linked to Eisenmenger syndrome or significant heart defects.

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Treatment Details

Interventions

Macitentan (Endothelin Receptor Antagonist)

Trial OverviewThe study is testing the effects of Macitentan compared to standard care in children with PAH. It's an open-label Phase 3 trial where participants are randomly assigned to either receive Macitentan or continue with their usual treatment while researchers track how well the drug works and its safety.

Participant Groups

2Treatment groups

Experimental Treatment

Group I: Standard-of-careExperimental Treatment1 Intervention

Standard-of-care as per site's clinical practice which may comprise treatment with pulmonary arterial hypertension (PAH) non-specific treatment and/or up to two PAH-specific medications excluding macitentan and intravenous/subcutaneous (IV/SC) prostanoids.

Group II: MacitentanExperimental Treatment1 Intervention

Macitentan is administered once daily via oral route. Children less than (\<) 2 years old (y.o.) will be assigned as a cohort to the macitentan group without randomization. The dose will be adjusted to the participant's age (for those \< 2 y.o.) or to the participant's body weight (for those greater than or equal to (\>=) 2 y.o.). single-arm extension period (SAEP) will start at end of core period (EOCP) visit and ends at end of study (EOS) visit.