A clinical study to investigate the long term safety of the drug macitentan in patients with pulmonary arterial hypertension and who were previously treated with macitentan in clinical studies.

• Register: EUCTR
• Last refreshed on: 28 February 2019
• Main ID: EUCTR2017-003934-10-FR
• Date of registration: 19/01/2018
• Prospective Registration: Yes
• Primary sponsor: Actelion Pharmaceuticals Ltd
• Public title: A clinical study to investigate the long term safety of the drug macitentan in patients with pulmonary arterial hypertension and who were previously treated with macitentan in clinical studies.
• Scientific title: mUlticenter, single-arM, open-laBel, long-teRm safety study with macitEntan in patients with puLmonary arterial hypertension previousLy treated with mAcitentan in clinical studies - UMBRELLA
• Date of first enrolment: 19/03/2018
• Target sample size: 94
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-003934-10
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

France

Contacts

• Name: Clinical trial disclosure desk
• Affiliation: Actelion Pharmaceuticals Ltd

• Name: Clinical trial disclosure desk
• Affiliation: Actelion Pharmaceuticals Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
1. Signed informed consent to take part in the study before any studymandated
procedure.
2. Subjects currently treated with macitentan in a clinical study that is
about to come to an end in France.
3. Women of childbearing potential are able to take part in the study if
the following applies:
a. Urine pregnancy test is negative at Enrollment.
b. Agreement to perform monthly urine or serum pregnancy tests during
the study and up to at least 30 days after the study treatment
discontinuation.
c. Agreement to adhere to the planned contraception scheme from
Enrollment up to at least 30 days after study treatment discontinuation.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 59
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 35

Exclusion criteria:
1. Hemoglobin < 80 g/L.
2. Serum aspartate (AST) and/or alanine (ALT) aminotransferases more
than three times the upper limit of normal range.
3. Known and documented history of severe drug-induced hepatic
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impairment (with or without cirrhosis).
4. Pregnant, planning to be become pregnant, or breastfeeding.
5. Known hypersensitivity to macitentan, its excipients, or drugs of the
same class.
6. Planned or current treatment with another investigational treatment
up to 3 months prior to Enrollment.
7. Any known factor or disease that might interfere with treatment
compliance, study conduct, or interpretation of the results, such as drug
or alcohol dependence or psychiatric disease.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Pulmonary arterial hypertension (PAH)
MedDRA version: 20.0 Level: PT Classification code 10064911 Term: Pulmonary arterial hypertension System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders

Intervention(s)

Trade Name: Opsumit
Product Name: Macitentan
Product Code: ACT-064992
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: MACITENTAN
CAS Number: 441798-33-0
Current Sponsor code: ACT-064992
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

Main Objective: The main objective of the study is to evaluate the long-term safety of macitentan 10 mg in subjects with pulmonary arterial hypertension.

Secondary Objective: No secondary objectives were defined for this study.

Timepoint(s) of evaluation of this end point: 1. for AEs during treatment phase at months 6, 12, 18, 24, 30, 36 and at
End-of-Treatment (EoT) visit, End-of-Study (EoS) visit and any
unscheduled visits
2. for SAEs during treatment phase at months 6, 12, 18, 24, 30, 36 and
at EoT visit, at EoS visit and any unscheduled visits
3. pregnancy tests performed at Screening and Enrollment (Day1) and
then monthly during treatment phase and at EoT visit

Primary end point(s): The long-term safety of macitentan will be evaluated by the following
endpoints:
1. Treatment-emergent adverse events (AEs) leading to premature
discontinuation of study treatment.
2. Treatment-emergent serious adverse events (SAEs).
3. Pregnancies with maternal exposure to macitentan.

Secondary Outcome(s)

Secondary end point(s): not applicable

Timepoint(s) of evaluation of this end point: not applicable

Secondary ID(s)

AC-055-314

Source(s) of Monetary Support

Actelion Pharmaceuticals Ltd.

Ethics review

Status: Approved
Approval date:
Contact: