- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05731492
A Study of Macitentan in Children Below 2 Years of Age
February 26, 2024 updated by: Actelion
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.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
10
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Study Contact
- Phone Number: 844-434-4210
- Email: Participate-In-This-Study@its.jnj.com
Study Locations
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Freiburg im Breisgau, Germany, 79106
- Recruiting
- Universitatsklinikum Freiburg
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Goettingen, Germany, 37075
- Recruiting
- Universitatsmedizin Gottingen
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Warszawa, Poland, 04-730
- Recruiting
- Instytut Pomnik - Centrum Zdrowia Dziecka
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Wroclaw, Poland, 51-124
- Recruiting
- Wojewodzki Szpital Specjalistyczny We Wroclawiu
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 month to 2 years (Child)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Basic Science
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Open-label Core Treatment Period: Macitentan
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.
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Macitentan will be administered orally.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State
Time Frame: Predose (at Week 4)
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Trough concentration of macitentan and its active metabolite aprocitentan at week 4 in steady-state will be reported.
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Predose (at Week 4)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants with Adverse Events (AEs)
Time Frame: Up to 1.5 years
|
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.
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Up to 1.5 years
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Number of Participants with Serious Adverse Events (SAEs)
Time Frame: Up to 1.5 years
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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.
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Up to 1.5 years
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Number of Participants with AEs Leading to Premature Discontinuation of Macitentan
Time Frame: Up to 1.5 years
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Number of participants with AEs Leading to premature discontinuation of macitentan will be reported.
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Up to 1.5 years
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Number of Participants with Adverse Event of Special Interests (AESIs)
Time Frame: Up to 1.5 years
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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.
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Up to 1.5 years
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Number of Participants with Clinical Laboratory Abnormalities
Time Frame: Up to 1.5 years
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Number of participants with clinical laboratory abnormalities (serum, chemistry and hematology) will be reported.
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Up to 1.5 years
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Number of Participants with Change from Baseline in Clinical laboratory Values.
Time Frame: Up to 1.5 years
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Number of participants with change from baseline in clinical laboratory values will be reported.
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Up to 1.5 years
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Change from Baseline in Blood Pressure
Time Frame: Up to 1.5 years
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Change from baseline in blood pressure will be reported.
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Up to 1.5 years
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Change From Baseline in Heart Rate
Time Frame: Up to 1.5 years
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Change from baseline in heart rate will be reported.
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Up to 1.5 years
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Change From Baseline in Body Weight
Time Frame: Up to 1.5 years
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Change from baseline in body weight will be reported.
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Up to 1.5 years
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Change From Baseline in Length
Time Frame: Up to 1.5 years
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Change from baseline in length will be reported.
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Up to 1.5 years
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Change from Baseline in Height
Time Frame: Up to 1.5 years
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Change from baseline in height will be reported.
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Up to 1.5 years
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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
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Plasma concentrations of macitentan and its active metabolite (aprocitentan) after the first dose of macitentan for macitentan naive participants will be reported.
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At 2, 5, and 24 hours after the first dose of macitentan on Day 1
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Trough Concentrations of Macitentan and its Active Metabolite Aprocitentan at Week 8 in Steady-State Conditions
Time Frame: Predose (at Week 8)
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Trough concentrations of macitentan and its active metabolite aprocitentan at week 8 in steady-state conditions will be reported.
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Predose (at Week 8)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Actelion Pharmaceuticals Ltd Clinical Trial, Actelion
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Estimated)
March 14, 2024
Primary Completion (Estimated)
April 1, 2024
Study Completion (Estimated)
September 30, 2025
Study Registration Dates
First Submitted
February 9, 2023
First Submitted That Met QC Criteria
February 9, 2023
First Posted (Actual)
February 16, 2023
Study Record Updates
Last Update Posted (Estimated)
February 28, 2024
Last Update Submitted That Met QC Criteria
February 26, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Respiratory Tract Diseases
- Lung Diseases
- Hypertension
- Pulmonary Arterial Hypertension
- Hypertension, Pulmonary
- Molecular Mechanisms of Pharmacological Action
- Endothelin Receptor Antagonists
- Endothelin A Receptor Antagonists
- Endothelin B Receptor Antagonists
- Macitentan
Other Study ID Numbers
- CR109286
- 2022-002754-74 (EudraCT Number)
- 67896062PAH1013 (Other Identifier: Actelion Pharmaceuticals Ltd)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
YES
IPD Plan Description
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
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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