A Study of Macitentan in Children Below 2 Years of Age

• Register: ClinicalTrials.gov
• Last refreshed on: 7 April 2025
• Main ID: NCT05731492
• Date of registration: 09/02/2023
• Prospective Registration: Yes
• Primary sponsor: Actelion
• Public title: A Study of Macitentan in Children Below 2 Years of Age
• Scientific 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
• Date of first enrolment: March 14, 2024
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/ct2/show/NCT05731492
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Basic Science. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Germany, Poland

Contacts

• Name: Actelion Pharmaceuticals Ltd Clinical Trial
• Affiliation: Actelion

Key inclusion & exclusion 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


Age minimum: 1 Month
Age maximum: 2 Years
Gender: All

Health Condition(s) or Problem(s) studied

Arterial Hypertension, Pulmonary

Intervention(s)

Drug: Macitentan

Primary Outcome(s)

Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State [Time Frame: Predose (at Week 4)]

Secondary Outcome(s)

Number of Participants with Adverse Events (AEs) [Time Frame: Up to 1.5 years]

Change from Baseline in Blood Pressure [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]

Change From Baseline in Heart Rate [Time Frame: Up to 1.5 years]

Change From Baseline in Length [Time Frame: Up to 1.5 years]

Trough Concentrations of Macitentan and its Active Metabolite Aprocitentan at Week 8 in Steady-State Conditions [Time Frame: Predose (at Week 8)]

Number of Participants with Adverse Event of Special Interests (AESIs) [Time Frame: Up to 1.5 years]

Number of Participants with Serious Adverse Events (SAEs) [Time Frame: Up to 1.5 years]

Change From Baseline in Body Weight [Time Frame: Up to 1.5 years]

Change from Baseline in Height [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]

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]

Secondary ID(s)

67896062PAH1013

2022-002754-74

CR109286

Source(s) of Monetary Support

Please refer to primary and secondary sponsors