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A Study of Macitentan in Children Below 2 Years of Age

Primary Purpose

Arterial Hypertension, Pulmonary

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Macitentan
Sponsored by
Actelion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional basic science trial for Arterial Hypertension, Pulmonary

Eligibility Criteria

1 Month - 2 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Universitatsklinikum FreiburgRecruiting
  • Universitätsmedizin GöttingenRecruiting
  • Instytut Pomnik - Centrum Zdrowia DzieckaRecruiting
  • Wojewodzki Szpital Specjalistyczny We WroclawiuRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open-label Core Treatment Period: Macitentan

Arm Description

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.
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.
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.
Number of Participants with Clinical Laboratory Abnormalities
Number of participants with clinical laboratory abnormalities (serum, chemistry and hematology) will be reported.
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.
Change from Baseline in Blood Pressure
Change from baseline in blood pressure will be reported.
Change From Baseline in Heart Rate
Change from baseline in heart rate will be reported.
Change From Baseline in Body Weight
Change from baseline in body weight will be reported.
Change From Baseline in Length
Change from baseline in length will be reported.
Change from Baseline in Height
Change from baseline in height will be reported.
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.
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.

Full Information

First Posted
February 9, 2023
Last Updated
October 9, 2023
Sponsor
Actelion
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1. Study Identification

Unique Protocol Identification Number
NCT05731492
Brief Title
A Study of Macitentan in Children Below 2 Years of Age
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 2, 2023 (Anticipated)
Primary Completion Date
April 1, 2024 (Anticipated)
Study Completion Date
September 30, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Actelion

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Arterial Hypertension, Pulmonary

7. Study Design

Primary Purpose
Basic Science
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Open-label Core Treatment Period: Macitentan
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Macitentan
Other Intervention Name(s)
JNJ-67896062, ACT-064922
Intervention Description
Macitentan will be administered orally.
Primary Outcome Measure Information:
Title
Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State
Description
Trough concentration of macitentan and its active metabolite aprocitentan at week 4 in steady-state will be reported.
Time Frame
Predose (at Week 4)
Secondary Outcome Measure Information:
Title
Number of Participants with Adverse Events (AEs)
Description
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.
Time Frame
Up to 1.5 years
Title
Number of Participants with Serious Adverse Events (SAEs)
Description
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.
Time Frame
Up to 1.5 years
Title
Number of Participants with AEs Leading to Premature Discontinuation of Macitentan
Description
Number of participants with AEs Leading to premature discontinuation of macitentan will be reported.
Time Frame
Up to 1.5 years
Title
Number of Participants with Adverse Event of Special Interests (AESIs)
Description
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.
Time Frame
Up to 1.5 years
Title
Number of Participants with Clinical Laboratory Abnormalities
Description
Number of participants with clinical laboratory abnormalities (serum, chemistry and hematology) will be reported.
Time Frame
Up to 1.5 years
Title
Number of Participants with Change from Baseline in Clinical laboratory Values.
Description
Number of participants with change from baseline in clinical laboratory values will be reported.
Time Frame
Up to 1.5 years
Title
Change from Baseline in Blood Pressure
Description
Change from baseline in blood pressure will be reported.
Time Frame
Up to 1.5 years
Title
Change From Baseline in Heart Rate
Description
Change from baseline in heart rate will be reported.
Time Frame
Up to 1.5 years
Title
Change From Baseline in Body Weight
Description
Change from baseline in body weight will be reported.
Time Frame
Up to 1.5 years
Title
Change From Baseline in Length
Description
Change from baseline in length will be reported.
Time Frame
Up to 1.5 years
Title
Change from Baseline in Height
Description
Change from baseline in height will be reported.
Time Frame
Up to 1.5 years
Title
Plasma Concentration of Macitentan and its Active Metabolite (Aprocitentan) for Macitentan Naive Participants
Description
Plasma concentrations of macitentan and its active metabolite (aprocitentan) after the first dose of macitentan for macitentan naive participants will be reported.
Time Frame
At 2, 5, and 24 hours after the first dose of macitentan on Day 1
Title
Trough Concentrations of Macitentan and its Active Metabolite Aprocitentan at Week 8 in Steady-State Conditions
Description
Trough concentrations of macitentan and its active metabolite aprocitentan at week 8 in steady-state conditions will be reported.
Time Frame
Predose (at Week 8)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Study Contact
Phone
844-434-4210
Email
Participate-In-This-Study@its.jnj.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Actelion Pharmaceuticals Ltd Clinical Trial
Organizational Affiliation
Actelion
Official's Role
Study Director
Facility Information:
Facility Name
Universitatsklinikum Freiburg
City
Freiburg im Breisgau
ZIP/Postal Code
79106
Country
Germany
Individual Site Status
Recruiting
Facility Name
Universitätsmedizin Göttingen
City
Goettingen
ZIP/Postal Code
37075
Country
Germany
Individual Site Status
Recruiting
Facility Name
Instytut Pomnik - Centrum Zdrowia Dziecka
City
Warszawa
ZIP/Postal Code
04-730
Country
Poland
Individual Site Status
Recruiting
Facility Name
Wojewodzki Szpital Specjalistyczny We Wroclawiu
City
Wroclaw
ZIP/Postal Code
51-124
Country
Poland
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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
IPD Sharing URL
https://www.janssen.com/clinical-trials/transparency

Learn more about this trial

A Study of Macitentan in Children Below 2 Years of Age

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