Clinical Study to Assess the Long-term Safety, Tolerability, and Efficacy of Macitentan in Subjects With Eisenmenger Syndrome

Study at a glance

From the registry record, last updated by the sponsor on 16 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

Long Term, Single-arm, Open-label Extension Study of Protocol AC-055-305 to Assess the Safety, Tolerability and Efficacy of Macitentan in Subjects With Eisenmenger Syndrome

ClinicalTrials.gov ID

NCT01739400

Acronym / sponsor ID

MAESTRO-OL / AC-055-308

Conditions

Pulmonary Arterial Hypertension

Keywords

Eisenmenger Syndrome, exercise capacity

Phase

Phase 3

Study type

Interventional

Recruitment status

TerminatedReason stopped: Primary endpoint of parent study AC-055-305/MAESTRO (NCT01743001) not met.

Enrolment

217 participants (actual)

Lead sponsor

ActelionIndustry (pharma, biotech, devices)

Study start

10 Sep 2013 (actual)

Primary completion

12 Jan 2018 (actual)

Study completion

12 Jan 2018 (actual)

First posted

3 Dec 2012 (estimated)

Results

Posted 16 Apr 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose

Treatment

Allocation

Not applicable

Intervention model

Single group

Masking

None (open label)

Number of arms

1

Who may qualify

Ages

12 years and older

Sex

All

Healthy volunteers

Not accepted

Age groups

Child, Adult, Older adult

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12

   Time frame: From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.

   NOTE: The MAESTRO-OL study was exploratory in nature and no primary efficacy and safety endpoint were defined in the clinical protocol. This and the other exploratory efficacy outcome measures posted were selected to be reported as a primary endpoints. All efficacy analyses were considered exploratory. The analyses of the exploratory efficacy endpoints focused on the absolute values and on the change from DB baseline to Week 16 in the DB study and to Month 6 and Month 12 in the OL study. For missing 6MWD values in the OL study, the following imputation rules were applied: If the reason for missing data was death, a distance of zero (0) meters was imputed for all 6MWD visits from the date of death. For any other reasons, the last available value was carried forward.

2. Change in WHO Functional Class (FC) at Month 6 and 12

   Time frame: From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.

   Class I: no symptoms with exercise or at rest. No limitation of activity. Class II: No symptoms at rest but slight limitation with ordinary activities causing symptoms (e.g. short of breath with climbing stairs). Class III: may not have symptoms at rest but activities greatly limited by shortness of breath, fatigue, or near fainting. Class IV: symptoms at rest and inability to carry out any physical activity without symptoms. Patients in class IV manifest signs of right heart failure. For missing WHO FC values in the OL study, the following imputation rules were applied: If the reason for missing data was death, class IV was imputed for all WHO visits from the date of death. For any other reasons, the last available value was carried forward.

3. Change in Borg Dyspnea Score at Month 6 and 12

   Time frame: From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.

   The Borg dyspnea score rates the severity of dyspnea (difficult or labored breathing) on a scale from 0 ('Nothing at all') to 10 ('Very, very severe - maximal'). For missing Borg dyspnea index values in the OL study, the following imputation rules were applied: If the reason for missing data was death, a value of 10 was imputed for all Borg visits from the date of death. For any other reasons, the last available value was carried forward.

4. Change in Peripheral Oxygen Saturation (SpO2) at Rest at Month 6 and 12

   Time frame: From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.

   No imputation of missing data for SpO2 was applied. Oxygen saturation assessed by pulse oximetry: peripheral oxygen saturation (SpO2) at rest before the 6-minute walk test (6MWT)

Results and safety

Loading the posted results from ClinicalTrials.gov…

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2019.
• Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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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.

The record lists no contact. Use the official record to reach the sponsor.

51 sites in 19 countries: see every site and its status.