Pulmonary Arterial HypertensionPhase 3NCT02112487
Extension of the Psychometric Validation Study ORCHESTRA in Patients With PAH
A Phase 3 study testing Macitentan in people with Pulmonary Arterial Hypertension, run by Actelion at 32 sites in 3 countries.
Trial summary
Prospective, multi-center, open-label, single-arm, Phase 3b extension study of macitentan in patients with PAH. To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- Macitentan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 88 participants
- Where
- 32 sitesin 3 countries
- Study dates
- 23 Jun 2014to 19 Sep 2018
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent prior to any study-mandated procedure.
- Patients with PAH who completed the ORCHESTRA study (AC-055-310) as scheduled
- Women of childbearing potential (as defined below) must:
- Have a negative serum pregnancy test at Visit 1 (i.e., Visit 4 of study AC 055 310) and agree to perform monthly serum pregnancy tests.
- Agree to use two reliable methods of contraception in parallel, from Visit 1 until 1 month after study drug discontinuation (see details below).
- A female is considered to have childbearing potential unless she meets at least one of the following criteria:
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- Previous bilateral salpingo and/or oophorectomy, or hysterectomy.
- Premature ovarian failure confirmed by a specialist.
- Pre-pubescence, XY genotype, Turner syndrome, uterine agenesis.
- Postmenopausal, defined as 12 consecutive months with no menses without an alternative medical cause.
- Of the two contraceptive methods that must be used, one must be from Group 1, and one must be from Group 2, defined as follows:
- Group 1: Oral, implantable, transdermal or injectable hormonal contraceptives, intrauterine devices, female sterilization (tubal ligation or non-surgical sterilization, e.g., permanent contraception with Essure procedure), or partner's sterilization (vasectomy). If a hormonal contraceptive is chosen from this group, it must be taken for at least one month prior to enrollment. Alternatively, if the Essure procedure is chosen as a contraceptive method, a hysterosalpingogram must have been performed to confirm correct location of the microinserts and tubal occlusion (as per manufacturer's recommendations).
- Group 2: Female or male condoms, diaphragm or cervical cap, any of them in combination with a spermicide.
- Sexual abstinence, rhythm methods, or contraception by the partner alone are not considered as acceptable methods of contraception for this study.
Who cannot join
- Patients who prematurely discontinued study drug in study AC-055-310.
- Females who are lactating or pregnant (positive Visit 1 pregnancy test) or plan to become pregnant during the study.
- AST and/or ALT more than 3 X ULN.
Trial locations and contacts
32 sites in 3 countries.
32 sites in 3 countries
Spain14 sites
Hospital General de Alicante
Alicante, 03010
Hospital Val Hebron
Barcelona, 08035
Hospital Clinic
Barcelona, 08036
Hospital de Cruces
Bilbao, 48903
Hospital Reina Sofia
Córdoba, 14004
Hospital Dr Negrin
Las Palmas de Gran Canaria, 35010
Hospital Universitario Insular Gran Canarias
Las Palmas de Gran Canaria, 35016
Hospital 12 Octubre
Madrid, 28041
Hospital La Paz
Madrid, 28046
Hospital Carlos Haya
Málaga, 29010
Hospital Son Espases
Palma de Mallorca, 7010
Hospital de Valdecilla
Santander, 39008
Hospital Virgen del Rocio
Seville, 41013
Hospita General U. Valencia
Valencia, 46014
France13 sites
Hôpital Louis Pradel
Bron, 69677
Hôpital Côte de Nacre
Caen, 14033
Hôpital Albert Michallon
Grenoble, 38700
CHU de Bicêtre
Le Kremlin-Bicêtre, 94270
CHRU Lille - Hôpital Cardiologique
Lille, 59037
Hôpital Arnaud de Villeneuve
Montpellier, 34295
Hôpitaux de Brabois
Nancy, 54511
Hôpital de Haut Levêque
Pessac, 33604
Hôpital Pontchaillou
Rennes, 35033
Hôpital Charles Nicolle
Rouen, 76031
Hôpital Nord
Saint-Etienne, 42227
Hôpital Civil
Strasbourg, 67091
Hôpital Larrey
Toulouse, 31059
Italy5 sites
Ospedale Sant'Orsola
Bologna, 40138
A.O.U.C. Careggi
Florence, 50124
UOC Immunologia Clinica B-PGRM Centro di Riferimento per la Sclerosi Sistemica
Rome, 00161
Centro Per La Diagnosi E La Cura Dell'Ipertensione Polmonare
Rome, 00186
Policlinico G.B. Rossi
Verona, 37134
About this study
Official title: An Extension of AC-055-310, a Multi-center, Open-label, Single-arm, Phase 3b Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the French, Italian and Spanish Versions of the PAH-SYMPACT™
Prospective, multi-center, open-label, single-arm, Phase 3b extension study of macitentan in patients with PAH.
To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study.
Read the detailed description
To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Macitentan
- Keywords
- pulmonary arterial hypertension, PAH-SYMPACT, PAH, psychometric instrument
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MacitentanDrug
Also called ACT-064992
10 mg once daily
| Study group | Role | What they receive |
|---|---|---|
| Macitentan | Experimental | 10 mg once daily Receives: Drug: Macitentan |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study.
- Treatment-emergent adverse events (AEs)
- AEs leading to premature discontinuation of study drug
- Treatment-emergent serious adverse events (SAEs)
- Proportion of patients with treatment-emergent ALT and/or AST abnormality (> 3, > 5, and > 8 x ULN) associated or not with total bilirubin > 2 x ULN.
- Proportion of patients with treatment-emergent hemoglobin abnormality (≦ 100 g/L, and ≦ 80 g/L)
Measured: Baseline to end of treatment visit (around 6 months on average)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2014 (estimated)
- Study start23 Jun 2014
- Primary completion19 Sep 2018
- Study completion19 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 88 participants (actual)
Sponsor and investigators
- Lead sponsor
- Actelion
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Loïc Perchenet, Study director, Actelion
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02112487
- Sponsor's protocol ID
- AC-055-311
- Status last verified
- Mar 2025
- First submitted
- 8 Apr 2014
- Last updated
- 30 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Extension of AC-055-310, a Multi-center, Open-label, Single-arm, Phase 3b Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the French, Italian and Spanish Versions of the PAH-SYMPACT™
- ClinicalTrials.gov ID
- NCT02112487
- Acronym / sponsor ID
- AC-055-311
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- pulmonary arterial hypertension, PAH-SYMPACT, PAH, psychometric instrument
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 88 participants (actual)
- Lead sponsor
- ActelionIndustry (pharma, biotech, devices)
- Study start
- 23 Jun 2014 (actual)
- Primary completion
- 19 Sep 2018 (actual)
- Study completion
- 19 Sep 2018 (actual)
- First posted
- 14 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Prospective, multi-center, open-label, single-arm, Phase 3b extension study of macitentan in patients with PAH.
To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study.
Detailed description
To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Macitentan | Experimental | Drug: Macitentan10 mg once daily |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Signed informed consent prior to any study-mandated procedure.
- Patients with PAH who completed the ORCHESTRA study (AC-055-310) as scheduled
- Women of childbearing potential (as defined below) must:
Key exclusion criteria (3 listed)
- Patients who prematurely discontinued study drug in study AC-055-310.
- Females who are lactating or pregnant (positive Visit 1 pregnancy test) or plan to become pregnant during the study.
- AST and/or ALT more than 3 X ULN.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MacitentanAlso: ACT-06499210 mg once daily | Drug | Macitentan |
Primary outcome
To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study.
Time frame: Baseline to end of treatment visit (around 6 months on average)
* Treatment-emergent adverse events (AEs) * AEs leading to premature discontinuation of study drug * Treatment-emergent serious adverse events (SAEs) * Proportion of patients with treatment-emergent ALT and/or AST abnormality (> 3, > 5, and > 8 x ULN) associated or not with total bilirubin > 2 x ULN. * Proportion of patients with treatment-emergent hemoglobin abnormality (≦ 100 g/L, and ≦ 80 g/L)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2025.
- Extension of the psychometric validation study ORCHESTRA in patients with PAHLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Loïc Perchenet study director
Actelion
32 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Macitentan in people with Pulmonary Arterial Hypertension, run by Actelion at 32 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in Spain, France, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the long-term safety of macitentan in patients with pulmonary arterial hypertension (PAH) beyond treatment in the AC-055-310 study. (time frame: Baseline to end of treatment visit (around 6 months on average)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2025; AzTrials last refreshed its copy on 7 Nov 2026.