MAESTRO (Macitentan in Eisenmenger Syndrome To Restore Exercise Capacity) - Full Text View

Purpose

Clinical study to assess the efficacy, safety, and tolerability of macitentan in subjects with Eisenmenger Syndrome.

Condition	Intervention	Phase
Eisenmenger Syndrome	Drug: Macitentan 10 mg, oral tablet, to be taken once daily. Drug: Matching placebo oral tablet, to be taken once daily.	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment
Official Title:	A Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group, Phase 3 Study to Evaluate the Effects of Macitentan on Exercise Capacity in Subjects With Eisenmenger Syndrome.

Resource links provided by NLM:

MedlinePlus related topics: Exercise and Physical Fitness

U.S. FDA Resources

Further study details as provided by Actelion:

Primary Outcome Measures:

Change from baseline to Week 16 in exercise capacity, as measured by 6MWD [ Time Frame: Baseline to Week 16 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change from baseline to Week 16 in WHO functional class [ Time Frame: Baseline to Week 16 ] [ Designated as safety issue: No ]
Change from baseline to Week 16 in dyspnea (assessed by the Borg dyspnea index) [ Time Frame: Baseline to Week 16 ] [ Designated as safety issue: No ]
Change from baseline to Week 16 in quality of life (assessed by the SF-36 questionnaire) [ Time Frame: Baseline to Week 16 ] [ Designated as safety issue: No ]

Estimated Enrollment:	220
Study Start Date:	December 2012
Estimated Study Completion Date:	June 2014
Estimated Primary Completion Date:	May 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Macitentan Macitentan 10 mg, oral tablet, to be taken once daily.	Drug: Macitentan 10 mg, oral tablet, to be taken once daily. Macitentan 10 mg, oral tablet, to be taken once daily. Other Name: Macitentan, ACT-064992
Placebo Comparator: Placebo Matching placebo oral tablet, to be taken once daily.	Drug: Matching placebo oral tablet, to be taken once daily. Matching placebo oral tablet, to be taken once daily. Other Name: Placebo

Eligibility

Ages Eligible for Study:	12 Years to 70 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Subjects:
- not participating in the hemodynamic sub-study: males or females ≥ 12 and ≤ 70 years of age.
- participating in the hemodynamic sub-study: males or females ≥ 18 and ≤ 70 years of age.
Subjects with confirmed ES (European Society of Cardiology [ESC] and the European Respiratory Society [ERS] guidelines):
- Established by echocardiography as:
  - Isolated ASD > 2 cm in diameter,
  - or isolated VSD > 1 cm in diameter,
  - or presence of both ASD and VSD (with either VSD ≤ 1 cm in diameter and ASD > 2 cm in diameter, or VSD > 1 cm in diameter and ASD ≤ 2 cm in diameter, or VSD > 1 cm in diameter and ASD > 2 cm in diameter),
  - and right to left shunt or bi-directional shunt with prevalent right to left direction.
- Unoperated, or previously palliated surgically for defects mentioned above (incomplete closure).
- Resting peripheral arterial oxygen saturation (SaO2) ≤ 90% and >70% (pulse oximetry, room air).
Cardiac catheterization measurements must show the following:
- mPAP > 25 mmHg,
- PCWP or LAP or LVED ≤ 15 mmHg,
- PVR ≥ 800 dyn∙s/cm5 or ≥ 10 Wood units.
Subjects with WHO functional class ≥ II.
Subjects able to perform the 6MWT with a minimum distance of 50 m and a maximum distance of 450 m.

Exclusion Criteria:

PAH not fulfilling the criteria of ES as described in inclusion criteria (e.g., complex cardiac defects, single ventricle, and patent ductus arteriosus).
Known moderate-to-severe restrictive (i.e., TLC < 60% of predicted value) or obstructive lung disease (i.e., FEV1 < 80 % of predicted, with FEV1 / FVC < 70%).
Down Syndrome.
Treatment with PDE-5 inhibitors or prostanoids within 1 month prior to Randomization.
Treatment with ERAs within 1 month prior to Randomization.
Subjects who initiated diuretics within 1 week prior to Randomization or subjects whose diuretic treatment has not been stable within 1 week prior to Randomization.
Subjects being considered for an organ transplant.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01743001

Show 84 Study Locations

Sponsors and Collaborators

Actelion

More Information

No publications provided

Responsible Party:	Actelion
ClinicalTrials.gov Identifier:	NCT01743001 History of Changes
Other Study ID Numbers:	AC-055-305
Study First Received:	November 29, 2012
Last Updated:	May 16, 2013
Health Authority:	Austria: Austrian Medicines and Medical Devices Agency Belgium: Federal Agency for Medicinal Products and Health Products Bulgaria: Bulgarian Drug Agency Canada: Health Canada Chile: Instituto de Salud Publica de Chile China: Food and Drug Administration France: L’Agence nationale de sécurité du médicament et des produits de santé Germany: Federal Institute for Drugs and Medical Devices Hungary: National Institute of Pharmacy India: Drugs Controller General of India Israel: Ministry of Health Italy: The Italian Medicines Agency Malaysia: Ministry of Health Mexico: Federal Commission for Protection Against Health Risks Netherlands: The Central Committee on Research Involving Human Subjects (CCMO) Philippines: Department of Health Philippines : Food and Drug Administration Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Portugal: National Pharmacy and Medicines Institute Russia: Ministry of Health of the Russian Federation Serbia and Montenegro: Agency for Drugs and Medicinal Devices South Africa: Medicines Control Council Spain: Agencia Española de Medicamentos y Productos Sanitarios Taiwan: Department of Health Turkey: Ministry of Health United Kingdom: Medicines and Healthcare Products Regulatory Agency United States: Food and Drug Administration Vietnam: Ministry of Health

Keywords provided by Actelion:

Eisenmenger Syndrome

Additional relevant MeSH terms:

Eisenmenger Complex
Heart Defects, Congenital
Cardiovascular Abnormalities

Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities

ClinicalTrials.gov processed this record on May 16, 2013