A Clinical Study to Investigate the Long-term Safety of the Drug Macitentan in Patients With Pulmonary Hypertension Who Were Previously Treated With Macitentan in Clinical Studies. (UMBRELLA)

April 25, 2025 updated by: Actelion

mUlticenter, Single-arM, Open-laBel, Long-teRm Safety Study With macitEntan in Patients With puLmonary Arterial Hypertension previousLy Treated With mAcitentan in Clinical Studies

The aim of the trial is to study the long-term safety of macitentan and to provide continued treatment with macitentan to patients with pulmonary arterial hypertension (PAH) and Chronic thromboembolic pulmonary hypertension (CTEPH) who were previously treated with macitentan in clinical studies.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The purpose of this study is to provide continued treatment with macitentan to subjects with PAH or CTEPH who participated in "parent studies" and to continue to accrue long-term safety data. The design of this study is widely used in clinical programs to give participants in a clinical study access to an effective study treatment beyond completion of the parent study. This is considered the best option to collect long-term safety and tolerability information of macitentan 10 mg and survival status of participants with PAH and CTEPH. "Parent study/studies" refer to a number of clinical studies with macitentan that are conducted in different clinical classification of PAH and CTEPH (NCT00667823, NCT02112487, NCT02310672, NCT02968901, NCT02558231, NCT02382016, NCT02060721) and may be completed before the participants have access to commercial macitentan in their country of residence. The "parent studies" are fully or partially running in countries where no access to commercial macitentan is expected in the near future.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
151

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belarus
      • Minsk, Belarus, 220036
        • The Republican Scientific-Practical Center ''Cardiology''
  • Belgium
      • Leuven, Belgium, 3000
        • UZ Leuven
  • France
      • Besancon, France, 25030
        • CHRU Besancon Hopital Jean Minjoz
      • Bordeaux (Pessac), France, 33604
        • CHU de Bordeaux - Hospital Haut-Leveque
      • Brest Cedex 2, France, 29609
        • CHU de la Cavale Blanche
      • Bron Cedex, France, 69677
        • GH est - Hôpital Cardiovasculaire et Pneumologie Louis Pradel
      • Caen Cedex, France
        • Hôpital Côte De Nacre
      • Dijon Cedex, France, 21079
        • CHU Dijon
      • Grenoble, France, 38700
        • CHU Grenoble
      • Le Kremlin Bicetre cedex, France, 94275
        • Hopital Bicetre Aphp Hopitaux Universitaires Paris Sud
      • Lille Cedex, France, 59037
        • Hôpital Cardiologique - Chru Lille
      • Limoges Cedex, France, 87042
        • CHU de Limoges
      • Marseille cedex 5, France, 13005
        • CHU de la Timone
      • Montpellier, France, 34295
        • CHU de Montpellier - Arnaud de Villeneuve
      • Nantes Cedex 1, France, 44093
        • CHU Nantes - Hopital Nord Laënnec
      • Nice, France, 06001
        • Centre Hospitalier Universitaire - de Nice - Hopital Pasteur
      • Paris Cedex 15, France, 75908
        • Hopital Europeen Georges Pompidou
      • Reims, France, 51100
        • CHU de Reims
      • Rennes, France, 35033
        • Chu Rennes Hopital Pontchaillou
      • Rouen, France, 76031
        • CHU Rouen Hopital Charles Nicolle
      • St Priest en Jarez Cedex, France, 42277
        • CHU Saint Etienne Hopital Nord
      • Strasbourg, France, 67091
        • Nouvel Hôpital Civil
      • Toulouse Cedex 9, France
        • Hopital Larrey CHU de Toulouse
      • Vandoeuvre les Nancy Cedex, France, 54511
        • CHU de Nancy - Hopital de Brabois
  • Poland
      • Lublin, Poland, 20 718
        • Wojewodzki Szpital Specjalistyczny im Stefana Kardynala Wyszynskiego SPZOZ
      • Wroclaw, Poland, 51 124
        • Wojewodzki Szpital Specjalistyczny Oddzial Kardiologiczny
  • Russian Federation
      • Kemerovo, Russian Federation, 650002
        • Cardiovascular Pathology Research Institute of Siberian Branch of RAMS
      • Moscow, Russian Federation, 121552
        • National Medical Research Center of Cardiology of MoH of Russian Federation
      • Novosibirsk, Russian Federation, 630055
        • Federal State Budgetary Institution Of Ministry Of Health Of Russian Federation
      • Saint-Petersburg, Russian Federation, 197341
        • Federal North-West Medical Research Centre
      • Tomsk, Russian Federation, 634012
        • Federal State Budget Scientific Institution
  • Turkey
      • Capa_Istanbul, Turkey, 34093
        • Istanbul University Istanbul Medical Faculty
  • Ukraine
      • Dnipro, Ukraine, 49059
        • CE 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiosurgery'
      • Lviv, Ukraine, 79000
        • Lviv Regional Clinical Hospital

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Signed informed consent to take part in the study before any study mandated procedure.
  2. Participants from one of the parent studies and: a) the sponsor has decided to terminate the parent study in that country and b) the participant has completed the end of treatment (EOT) Visit of the parent study
  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; and c) Agreement to adhere to the planned contraception scheme from Enrollment up to at least 30 days after study treatment discontinuation

Exclusion Criteria:

  1. Hemoglobin less than 80 gram per liter (g/L)
  2. Serum Aspartate aminotransferase (AST) and/or alanine aminotransferases (ALT) more than three times the upper limit of normal range
  3. Known and documented history of severe hepatic impairment that is Child-Pugh Class C.
  4. Pregnant, planning to 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 may interfere with treatment compliance, study conduct, or interpretation of the results, such as drug or alcohol dependence or psychiatric disease
  8. Treatment with a strong CYP3A4 inhibitor (e.g., ketoconazole, itraconazole, voriconazole, clarithromycin, telithromycin, nefazodone, ritonavir, and saquinavir)

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: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Open-label macitentan 10 mg
10 mg macitentan film coated tablet, administered orally once daily
Drug: macitentan
macitentan 10 mg, film-coated tablet, oral use
Other Names:
  • ACT-064992
  • JNJ-67896062

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Incident Rate of Treatment-emergent Adverse Event
Time Frame: From Day 1 to End of study (EoS) visit (an average of 3 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 study 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. A treatment-emergent AE is any AE temporally associated with the use of study treatment.
From Day 1 to End of study (EoS) visit (an average of 3 years)
Incident rate of treatment-emergent adverse events (AEs) leading to premature discontinuation of study treatment
Time Frame: From Day 1 to EoS visit (an average of 3 years)
Any AE will be recorded that 1) is (temporally) associated with the use of study treatment whether or not considered by the investigator as related to study treatment and 2) leads to premature discontinuation of study medication.
From Day 1 to EoS visit (an average of 3 years)
Incident rate of treatment-emergent serious adverse events (SAEs)
Time Frame: From Day 1 to EoS visit (an average of 3 years)
Any SAE as defined by the ICH guidelines will be recorded. Any hepatic AE that leads to discontinuation of study treatment will be defined as SAE.
From Day 1 to EoS visit (an average of 3 years)
Number of pregnancies with maternal exposure to macitentan
Time Frame: From Day 1 to EoS visit (an average of 3 years)
Pregnancies with maternal exposure to macitentan will be recorded.
From Day 1 to EoS visit (an average of 3 years)

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change of WHO functional class to each scheduled time point
Time Frame: From Day 1 to EoT visit (an average of 3 years)
The proportion of patients who worsened, remain unchanged or improved from baseline to each scheduled time-point in WHO function will be calculated, where baseline is the initial baseline from the "parent study" (or the double-blind core study preceding the "parent study").
From Day 1 to EoT visit (an average of 3 years)
Assessment of survival status at End-of-Study (EoS)
Time Frame: From Day 1 to EoS visit (an average of 3 years)
Time to death of all causes up to EoS from the date of randomization or enrollment in the "parent study" (or the double-blind core study preceding the "parent study") will be estimated.
From Day 1 to EoS visit (an average of 3 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Actelion

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Marek Sochor, 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 (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 5, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 27, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 27, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 30, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 30, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 5, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 27, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 25, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AC-055-314
  • 2017-003934-10 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

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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