Arterial Hypertension, PulmonaryPhase 1NCT05731492

A Study of Macitentan in Children Below 2 Years of Age

A Phase 1 study testing Macitentan in people with Arterial Hypertension, Pulmonary, run by Actelion at 4 sites in 2 countries.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial summary

The purpose of this study is to learn what happens to macitentan and its active metabolite (aprocitentan) in the body of children aged between 1 month and 2 years. Read more about this study

Who is it for?: Ages 1 month to 2 years
Conditions the study is for: Arterial Hypertension, Pulmonary
What is being tested?: Macitentan
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: 4 sitesin 2 countries
Study dates: 14 Mar 2024to 1 Apr 2024
Phase: Phase 1
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 1 month to 2 years
All sexes
No healthy volunteers
Child

Who may be able to join

Pulmonary arterial hypertension (PAH): 1) including participants with Down syndrome. Diagnosis must have been confirmed by (historical, any time before screening) right heart catheterization mean pulmonary arterial pressure (mPAP) greater than or equal to (>=) 25 millimeter of mercury (mmHg), pulmonary arterial wedge pressure (PAWP) less than or equal to (=<)15 mmHg, pulmonary vascular resistance index greater than (>) 3 Wood units * meter square (m^2) where in the absence of pulmonary vein obstruction and/or significant lung disease PAWP can be replaced left atrium pressure or left ventricular end diastolic pressure (in the absence of mitral stenosis) assessed by heart catheterization. a) Idiopathic PAH, or b) Heritable PAH, or c) PAH associated with congenital heart disease: i) Eisenmenger syndrome (Qp/Qs less than (<) 1.5 and saturation of peripheral oxygen ≤ 90 percent (%) measured by pulse oximetry at room air), or ii) Inoperable open left-to-right shunts (with a Pulmonary vascular resistance [PVR] > 8 WU and Qp/Qs <2), or iii) Co-incidental shunt (that is, not explaining hemodynamically the presence of PAH), or iv) Post-operative PAH (persisting/recurring/developing ≥ 6 months after repair of shunt), or d) Drug or toxin induced PAH, or e) PAH associated with Human immunodeficiency viruses (HIV)
World Health Organization Functional Class (WHO FC) I, II, or III
PAH-specific treatment-naive participants or participants on PAH specific monotherapy or combination of 2 therapies. Use of macitentan before or during screening is allowed
Body weight of greater than or equal to (>=) 3.5 kilogram (kg)
Parent(s) (preferably both if available or as per local requirements) or participant's legally designated representative must sign an informed consent form (ICF) indicating that they understand the purpose of, and procedures required for, the study and is/are willing to allow the child to participate in the study

Who cannot join

PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis
Persistent pulmonary hypertension of the newborn
The following congenital cardiac abnormalities: a) Cyanotic congenital cardiac lesions such as transposition of the great arteries, truncus arteriosus, pulmonary atresia with ventricular septal defect, unless operatively repaired and with no residual shunt. b) Univentricular heart and/or participants with Fontan-palliation
Pulmonary hypertension due to lung disease
Known diagnosis of bronchopulmonary dysplasia

Trial locations and contacts

4 sites in 2 countries.

Country

City, postcode or hospital

4 sites in 2 countries

Germany2 sites

Universitatsklinikum Freiburg
Freiburg im Breisgau, 79106
Universitatsmedizin Gottingen
Göttingen, 37075

Other trials in Germany

Poland2 sites

Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04 730
Wojewodzki Szpital Specjalistyczny We Wroclawiu
Wroclaw, 51 124

Other trials in Poland

About this study

Official title: A Multicenter, Open-label, Single-arm Study to Assess the Pharmacokinetics and Safety of Macitentan in Children Aged 1 Month to <2 Years With Pulmonary Arterial Hypertension

The purpose of this study is to learn what happens to macitentan and its active metabolite (aprocitentan) in the body of children aged between 1 month and 2 years.

Conditions: Arterial Hypertension, Pulmonary
Being tested: Macitentan
Medical subject headings: Pulmonary Arterial Hypertension

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

MacitentanDrug
Also called JNJ-67896062, ACT-064922
Macitentan will be administered orally.

Study groups in NCT05731492
Study group	Role	What they receive
Open-label Core Treatment Period: Macitentan	Experimental	Participants will receive macitentan as a monotherapy or add-on to an existing therapy daily for 24 weeks during core treatment period. Optional treatment extension period of up to 1 year for those participants who completed the core treatment period. Receives: Drug: Macitentan

What is being measured

The primary outcome is the main question the study is designed to answer.

Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State
Trough concentration of macitentan and its active metabolite aprocitentan at week 4 in steady-state will be reported.
Measured: Predose (at Week 4)

Secondary outcomes (13)

Number of Participants with Adverse Events (AEs)
An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non-investigational) product, whether or not related to that medicinal (investigational or non-investigational) product.
Measured: Up to 1.5 years
Number of Participants with Serious Adverse Events (SAEs)
A SAE is any AE that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect and is a suspected transmission of any infectious agent via a medicinal product, is medically important.
Measured: Up to 1.5 years
Number of Participants with AEs Leading to Premature Discontinuation of Macitentan
Number of participants with AEs Leading to premature discontinuation of macitentan will be reported.
Measured: Up to 1.5 years
Number of Participants with Adverse Event of Special Interests (AESIs)
Number of participants with AESI will be reported. AEs considered to be of special interest are as hypotension, edema/fluid retention, hemoglobin decrease/anemia, liver events.
Measured: Up to 1.5 years
Number of Participants with Clinical Laboratory Abnormalities
Number of participants with clinical laboratory abnormalities (serum, chemistry and hematology) will be reported.
Measured: Up to 1.5 years
Number of Participants with Change from Baseline in Clinical laboratory Values.
Number of participants with change from baseline in clinical laboratory values will be reported.
Measured: Up to 1.5 years
Change from Baseline in Blood Pressure
Change from baseline in blood pressure will be reported.
Measured: Up to 1.5 years
Change From Baseline in Heart Rate
Change from baseline in heart rate will be reported.
Measured: Up to 1.5 years
Change From Baseline in Body Weight
Change from baseline in body weight will be reported.
Measured: Up to 1.5 years
Change From Baseline in Length
Change from baseline in length will be reported.
Measured: Up to 1.5 years
Change from Baseline in Height
Change from baseline in height will be reported.
Measured: Up to 1.5 years
Plasma Concentration of Macitentan and its Active Metabolite (Aprocitentan) for Macitentan Naive Participants
Plasma concentrations of macitentan and its active metabolite (aprocitentan) after the first dose of macitentan for macitentan naive participants will be reported.
Measured: At 2, 5, and 24 hours after the first dose of macitentan on Day 1
Trough Concentrations of Macitentan and its Active Metabolite Aprocitentan at Week 8 in Steady-State Conditions
Trough concentrations of macitentan and its active metabolite aprocitentan at week 8 in steady-state conditions will be reported.
Measured: Predose (at Week 8)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

14 Mar 20241 Apr 2024

First posted16 Feb 2023
Study start14 Mar 2024 (estimated)
Primary completion1 Apr 2024 (estimated)
Study completion1 Apr 2024 (estimated)

Why it stopped: Sponsor Decision

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Arterial Hypertension, PulmonaryStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Basic science
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05731492
Sponsor's protocol ID: CR109286
Other IDs: 2022-002754-74, 67896062PAH1013
Status last verified: Mar 2025
First submitted: 9 Feb 2023
Last updated: 30 Mar 2025
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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An Open-Label Extension Study to Evaluate Inhaled Treprostinil SMI in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
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Study of Treprostinil Administered Via Soft Mist Inhaler (SMI) in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease
Active, not recruitingPhase 1
A Pilot Study of Microbiota Transplant With Antibiotic Preconditioning and Fiber Supplementation in Pulmonary Arterial Hypertension (GUT RESTORE-PAH)
Pulmonary Arterial Hypertension
RecruitingObservational study
Natural History Study of Biomarkers in Pulmonary Arterial Hypertension
Pulmonary Disease, Pulmonary Hypertension
Active, not recruitingPhase 1
Study of Intravenous ZMA001 in Healthy Subjects
Pulmonary Arterial Hypertension PAH

All Arterial Hypertension, Pulmonary trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter, Open-label, Single-arm Study to Assess the Pharmacokinetics and Safety of Macitentan in Children Aged 1 Month to <2 Years With Pulmonary Arterial Hypertension
ClinicalTrials.gov ID: NCT05731492
Acronym / sponsor ID: CR109286
Conditions: Arterial Hypertension, Pulmonary
Phase: Phase 1
Study type: Interventional
Recruitment status: WithdrawnReason stopped: Sponsor Decision
Enrolment: 0 participants (actual)
Lead sponsor: ActelionIndustry (pharma, biotech, devices)
Study start: 14 Mar 2024 (estimated)
Primary completion: 1 Apr 2024 (estimated)
Study completion: 1 Apr 2024 (estimated)
First posted: 16 Feb 2023 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of this study is to learn what happens to macitentan and its active metabolite (aprocitentan) in the body of children aged between 1 month and 2 years.

Study design

Primary purpose: Basic science
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Open-label Core Treatment Period: Macitentan	Experimental	Drug: MacitentanParticipants will receive macitentan as a monotherapy or add-on to an existing therapy daily for 24 weeks during core treatment period. Optional treatment extension period of up to 1 year for those participants who completed the core treatment period.

Who may qualify

Ages: 1 month to 2 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (5 listed)

Pulmonary arterial hypertension (PAH): 1) including participants with Down syndrome. Diagnosis must have been confirmed by (historical, any time before screening) right heart catheterization mean pulmonary arterial pressure (mPAP) greater than or equal to (>=) 25 millimeter of mercury (mmHg), pulmonary arterial wedge pressure (PAWP) less than or equal to (=<)15 mmHg, pulmonary vascular resistance index greater than (>) 3 Wood units * meter square (m^2) where in the absence of pulmonary vein obstruction and/or significant lung disease PAWP can be replaced left atrium pressure or left ventricular end diastolic pressure (in the absence of mitral stenosis) assessed by heart catheterization. a) Idiopathic PAH, or b) Heritable PAH, or c) PAH associated with congenital heart disease: i) Eisenmenger syndrome (Qp/Qs less than (<) 1.5 and saturation of peripheral oxygen ≤ 90 percent (%) measured by pulse oximetry at room air), or ii) Inoperable open left-to-right shunts (with a Pulmonary vascular resistance [PVR] > 8 WU and Qp/Qs <2), or iii) Co-incidental shunt (that is, not explaining hemodynamically the presence of PAH), or iv) Post-operative PAH (persisting/recurring/developing ≥ 6 months after repair of shunt), or d) Drug or toxin induced PAH, or e) PAH associated with Human immunodeficiency viruses (HIV)
World Health Organization Functional Class (WHO FC) I, II, or III
PAH-specific treatment-naive participants or participants on PAH specific monotherapy or combination of 2 therapies. Use of macitentan before or during screening is allowed
Body weight of greater than or equal to (>=) 3.5 kilogram (kg)
Parent(s) (preferably both if available or as per local requirements) or participant's legally designated representative must sign an informed consent form (ICF) indicating that they understand the purpose of, and procedures required for, the study and is/are willing to allow the child to participate in the study

Key exclusion criteria (5 listed)

PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis
Persistent pulmonary hypertension of the newborn
The following congenital cardiac abnormalities: a) Cyanotic congenital cardiac lesions such as transposition of the great arteries, truncus arteriosus, pulmonary atresia with ventricular septal defect, unless operatively repaired and with no residual shunt. b) Univentricular heart and/or participants with Fontan-palliation
Pulmonary hypertension due to lung disease
Known diagnosis of bronchopulmonary dysplasia

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
MacitentanAlso: JNJ-67896062, ACT-064922Macitentan will be administered orally.	Drug	Open-label Core Treatment Period: Macitentan

Primary outcome

Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State
Time frame: Predose (at Week 4)
Trough concentration of macitentan and its active metabolite aprocitentan at week 4 in steady-state will be reported.

13 secondary outcomes

Number of Participants with Adverse Events (AEs)
Time frame: Up to 1.5 years
Number of Participants with Serious Adverse Events (SAEs)
Time frame: Up to 1.5 years
Number of Participants with AEs Leading to Premature Discontinuation of Macitentan
Time frame: Up to 1.5 years
Number of Participants with Adverse Event of Special Interests (AESIs)
Time frame: Up to 1.5 years
Number of Participants with Clinical Laboratory Abnormalities
Time frame: Up to 1.5 years
Number of Participants with Change from Baseline in Clinical laboratory Values.
Time frame: Up to 1.5 years
Change from Baseline in Blood Pressure
Time frame: Up to 1.5 years
Change From Baseline in Heart Rate
Time frame: Up to 1.5 years
Change From Baseline in Body Weight
Time frame: Up to 1.5 years
Change From Baseline in Length
Time frame: Up to 1.5 years
Change from Baseline in Height
Time frame: Up to 1.5 years
Plasma Concentration of Macitentan and its Active Metabolite (Aprocitentan) for Macitentan Naive Participants
Time frame: At 2, 5, and 24 hours after the first dose of macitentan on Day 1
Trough Concentrations of Macitentan and its Active Metabolite Aprocitentan at Week 8 in Steady-State Conditions
Time frame: Predose (at Week 8)

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2025.
Individual participant dataSharing planned: Yes. The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Related trials for Arterial Hypertension, Pulmonary

Not yet recruitingPhase 2/3
An Open-Label Extension Study to Evaluate Inhaled Treprostinil SMI in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease
Not yet recruitingPhase 2
Study of Treprostinil Administered Via Soft Mist Inhaler (SMI) in Participants With Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease (PH-COPD)
Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease
Active, not recruitingPhase 1
A Pilot Study of Microbiota Transplant With Antibiotic Preconditioning and Fiber Supplementation in Pulmonary Arterial Hypertension (GUT RESTORE-PAH)
Pulmonary Arterial Hypertension
RecruitingObservational study
Natural History Study of Biomarkers in Pulmonary Arterial Hypertension
Pulmonary Disease, Pulmonary Hypertension
Active, not recruitingPhase 1
Study of Intravenous ZMA001 in Healthy Subjects
Pulmonary Arterial Hypertension PAH

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Actelion Pharmaceuticals Ltd Clinical Trial study director

Actelion

4 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Macitentan in people with Arterial Hypertension, Pulmonary, run by Actelion at 4 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Germany, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State (time frame: Predose (at Week 4)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05731492.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2025.