A Study of Macitentan in Children Below 2 Years of Age

NCT05731492 | Withdrawn Phase 1 DMC FDA Drug

• 3 other identifiers: CR1092862022-002754-7467896062PAH1013
• Study Type: interventional
• Target: N/A
• Locations: 2 countries
• Sites: 4

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.

Conditions

Arterial Hypertension, Pulmonary

Outcome Measures

Primary Outcomes (1)

• 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.

  Predose (at Week 4)

Secondary Outcomes (13)

• Number of Participants with Adverse Events (AEs)

  Up to 1.5 years

• Number of Participants with Serious Adverse Events (SAEs)

  Up to 1.5 years

• Number of Participants with AEs Leading to Premature Discontinuation of Macitentan

  Up to 1.5 years

• Number of Participants with Adverse Event of Special Interests (AESIs)

  Up to 1.5 years

• Number of Participants with Clinical Laboratory Abnormalities

  Up to 1.5 years

Study Arms (1)

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.

Drug: Macitentan

Interventions

Macitentan DRUG

Macitentan will be administered orally.

Also known as: JNJ-67896062, ACT-064922

Open-label Core Treatment Period: Macitentan

Eligibility Criteria

• Age: 1 Month - 2 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Actelion: lead

Study Sites (4)

Universitatsklinikum Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Universitatsmedizin Gottingen

Göttingen, 37075, Germany

Location

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, 04 730, Poland

Location

Wojewodzki Szpital Specjalistyczny We Wroclawiu

Wroclaw, 51 124, Poland

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

macitentan

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Study Officials

• Actelion Pharmaceuticals Ltd Clinical Trial

  Actelion

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 9, 2023
• First Posted: February 16, 2023
• Study Start: March 14, 2024
• Primary Completion: April 1, 2024
• Study Completion: April 1, 2024
• Last Updated: March 30, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share

Locations

PL (2); DE (2)