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Endothelin Receptor Antagonist
Macitentan for Pulmonary Hypertension (TOMORROW Trial)
Phase 3
Recruiting
Research Sponsored by Actelion
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Males or females between greater than or equal to (>=) 1 month and less than (<) 18 years of age
Males or females between 1 month and less than 18 years of age
Timeline
Screening 3 weeks
Treatment Varies
Follow Up between randomization/visit 2 and eocp; up to 7 years
Awards & highlights
TOMORROW Trial Summary
This trial will test the effects of macitentan in children with pulmonary arterial hypertension. The trial will see if the medication is safe and effective in this population.
Who is the study for?
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.Check my eligibility
What is being tested?
The 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.See study design
What are the potential side effects?
While not specified here, common side effects of Macitentan may include symptoms like headache, cold-like symptoms (nasopharyngitis), anemia, bronchitis, urinary tract infections, and inflammation of the airways (bronchitis).
TOMORROW Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am under 18 years old.
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I am under 18 years old.
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I have been diagnosed with PAH confirmed by a heart catheter test.
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My PAH is classified under Group 1 and may be related to a specific cause like heart disease or HIV.
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My body weight is at least 3.5 kg.
TOMORROW Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ between randomization/visit 2 and eocp; up to 7 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~between randomization/visit 2 and eocp; up to 7 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Plasma Exchange
Participants Less than (<) 2 Years: Observed Steady-state Trough Plasma Concentration of Macitentan and its Active Metabolite ACT-132577 at Week 4
Secondary outcome measures
Change from Baseline to Week 24 in Left Ventricular Eccentricity Index (LVEI) Measured by Echocardiography
Change from Baseline to Week 24 in N-terminal Pro-brain Natriuretic Peptide (NT-proBNP)
Change from Baseline to Week 24 in Pediatric Quality of Life Inventory version 4.0 (PedsQL 4.0) Generic Core Scales Short Form (SF-15)
+7 moreSide effects data
From 2019 Phase 4 trial • 89 Patients • NCT0231067222%
Oedema Peripheral
21%
Headache
14%
Dizziness
11%
Upper Respiratory Tract Infection
11%
Haemoglobin Decreased
11%
Cough
10%
Myalgia
9%
Diarrhoea
9%
Nasal Congestion
8%
Back Pain
8%
Pyrexia
7%
Pain in Extremity
7%
Nasopharyngitis
7%
Nausea
6%
Flushing
3%
Pneumonia
2%
Pulmonary Embolism
2%
Pulmonary Arterial Hypertension
2%
Sepsis
2%
Acute Myocardial Infarction
1%
Cholangitis Infective
1%
Cytomegalovirus Infection
1%
Herpes Zoster
1%
Hypersensitivity
1%
Therapeutic Response Decreased
1%
Escherichia Bacteraemia
1%
Epistaxis
1%
Urinary Tract Infection
1%
Hypoglycaemia
1%
Rheumatoid Arthritis
1%
Pancreatic Carcinoma
1%
Systemic Lupus Erythematosus
1%
Syncope
1%
Acute Kidney Injury
1%
Dysfunctional Uterine Bleeding
1%
Anaemia
1%
Haemolytic Anaemia
1%
Iron Deficiency Anaemia
1%
Angina Pectoris
1%
Atrial Fibrillation
1%
Cardiac Arrest
1%
Coronary Artery Disease
1%
Right Ventricular Failure
1%
Ventricular Hypokinesia
1%
Rectal Haemorrhage
1%
Dysmenorrhoea
1%
Menorrhagia
1%
Pelvic Haemorrhage
1%
Dyspnoea
100%
80%
60%
40%
20%
0%
Study treatment Arm
Macitentan 10 mg
TOMORROW Trial Design
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Macitentan
2020
Completed Phase 4
~1920
Find a Location
Who is running the clinical trial?
ActelionLead Sponsor
191 Previous Clinical Trials
35,393 Total Patients Enrolled
66 Trials studying Pulmonary Arterial Hypertension
15,685 Patients Enrolled for Pulmonary Arterial Hypertension
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am under 18 years old.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.My liver is not working well (severe issues).Your hemoglobin or hematocrit levels are very low.Your blood test shows that your AST and/or ALT levels are more than three times the normal range.I have been diagnosed with PAH confirmed by a heart catheter test.I have low blood pressure that makes starting PAH treatment risky.I have been diagnosed with PAH confirmed by a heart catheter test.I am under 18 years old.My kidneys are not working well (severe renal insufficiency).My PAH is classified under Group 1 and may be related to a specific cause like heart disease or HIV.I have been diagnosed with bronchopulmonary dysplasia.I am not pregnant, will test monthly, and will use birth control until the study ends.My PAH is caused by portal hypertension, schistosomiasis, or specific lung diseases.My body weight is at least 3.5 kg.
Research Study Groups:
This trial has the following groups:- Group 1: Macitentan
- Group 2: Standard-of-care
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Please let me know how many hospitals are currently testing this new treatment.
"There are a total of 18 sites running this trial currently. To help reduce burdening enrollees with travel, the sites are located in major cities such as Boston, Indianapolis and Aurora."
Answered by AI
What is the novel aspect of this research?
"There are 8 ongoing trials for Macitentan in 88 cities and 46 countries. The first study began in 2017 and completed Phase 3 in the same year. This was a 300-patient trial sponsored by Actelion. There have been 34 completed trials since then."
Answered by AI
Might I be a candidate to participate in this research project?
"Eligible patients for this trial must have pulmonary arterial hypertension and be between the ages of 1 month to 17 years old. This study plans to enroll around 300 individuals."
Answered by AI
Are there similar therapies to compare this drug to?
"8 clinical trials are currently testing the efficacy of Macitentan. Of these 8 studies, 1 is in Phase 3. It's worth mentioning that while several studies are taking place in Durham, North carolina, there are 818 total locations running Macitentan trials."
Answered by AI
Is there a risk of long-term side effects from taking Macitentan?
"There is some clinical data supporting the efficacy of Macitentan, and as it is a Phase 3 trial, it received a score of 3 for safety."
Answered by AI
Which goals does this test hope to achieve?
"The primary objective of this study, as measured over Week 12, is to establish the observed steady-state trough plasma concentration of macitentan and its active metabolite ACT-132577 in participants aged 2 years or younger. Secondary objectives include determining the percentage of participants with WHO functional class I or II versus III or IV, measuring the time to CEC-confirmed death due to PAH, and assessing the change from baseline to Week 24 in PedsQL 4.0 Generic Core Scales Short Form (SF-15) scores."
Answered by AI
Do elderly individuals qualify for this experiment?
"This clinical trial is enrolling patients that are infants up to seventeen years old. Out of the 756 total studies, 58 are for patients under eighteen and 698 are for patients over the age of sixty-five."
Answered by AI
Who else is applying?
What state do they live in?
Nevada
How old are they?
18 - 65
What site did they apply to?
Children's Heart Center
What portion of applicants met pre-screening criteria?
Met criteria
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