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Macitentan Use in an Idiopathic Pulmonary Fibrosis Clinical Study (MUSIC)

This study has been completed.
ClinicalTrials.gov Identifier:
First Posted: May 18, 2009
Last Update Posted: February 17, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):

The AC-055B201/MUSIC study is a Phase II study, comparing one dose of ACT-064922 (macitentan) 10 mg with placebo in patients with idiopathic pulmonary fibrosis (IPF). The main study objective is to demonstrate that macitentan positively affects the forced vital capacity (FVC) in comparison with placebo in patients with idiopathic pulmonary fibrosis (IPF).

The secondary objectives are to evaluate the effect of macitentan on the time to disease worsening or death in patients with IPF, and to evaluate the benefit/risk profile of macitentan in the treatment of patients with IPF.

Condition Intervention Phase
Idiopathic Pulmonary Fibrosis Drug: ACT-064992 (macitentan) Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double-blind, Randomized, Placebo-controlled, Multicenter, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Macitentan in Patients With Idiopathic Pulmonary Fibrosis

Resource links provided by NLM:

Further study details as provided by Actelion:

Primary Outcome Measures:
  • Forced Vital Capacity (FVC) at Baseline and End of Period 1 [ Time Frame: 12 months ]
    FVC was measured at baseline and at the end of Period 1. The same equipment and tester were used during the course of the study. The equipment was calibrated and the calibration documented prior to each patient's measurement. The person responsible for conducting the pulmonary function tests was required to comply with the study guidelines and the American Thoracic Society/European Respiratory Society joint criteria on lung function testing.

Secondary Outcome Measures:
  • Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study [ Time Frame: Up to end of study (Up to 24 months) ]

    Disease worsening was indicated by pulmonary function test/idiopathic pulmonary fibrosis worsening (PFT/IPF) or acute respiratory decompensation of IPF.

    PFT/IPF worsening was indicated by the occurrence of both of the following: confirmed by two tests at least 4 weeks apart, as defined by the occurrence of both of the following: decrease from baseline ≥ 10% in forced vital capacity and decrease from baseline ≥ 15% in corrected diffusing capacity of the lung for carbon monoxide.

    Acute respiratory decompensation of IPF was defined as an unexplained rapid deterioration (over a period of less than 4 weeks) of the patient's condition with increasing shortness of breath requiring oxygen supplementation ≥ 5 L/min to maintain a resting oxygen saturation ≥ 90% or arterial oxygen pressure ≥ 55 mmHg (sea level) or 50 mmHg (high altitude).

Enrollment: 178
Study Start Date: May 2009
Study Completion Date: August 2011
Primary Completion Date: June 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ACT-064922
ACT-064922 tablet (macitentan), 10 mg, once daily
Drug: ACT-064992 (macitentan)
ACT-064992 (macitentan) tablet, 10 mg, once daily
Other Name: macitentan
Placebo Comparator: Placebo
Matching placebo, once daily
Drug: Placebo
matching placebo, once daily

Detailed Description:
The study included two treatment periods: Period 1 (fixed duration) from randomization up to the primary endpoint evaluation (Month 12 or earlier in case of premature discontinuation of study drug) and Period 2 (variable duration) from the primary endpoint evaluation visit up to the end of study (EOS). EOS occurred when the last patient randomized and not prematurely discontinued completed Period 1.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Signed informed consent.
  2. Male or female patients of at least 18 years of age (females of child-bearing potential must use a reliable method of contraception).
  3. IPF diagnosis within 3 years prior to randomization, proven according to the American Thoracic Society/European Respiratory Society consensus conference criteria, with surgical lung biopsy.

Exclusion Criteria:

  1. Interstitial lung disease due to conditions other than IPF.
  2. Presence of extensive honeycombing on Baseline high-resolution computed tomography (HRCT) scan performed within 3 months prior to randomization.
  3. Severe concomitant illness limiting life expectancy (< 1 year).
  4. Severe restrictive lung disease: forced vital capacity (FVC) < 50% predicted, or FVC < 1.2 liter.
  5. Diffusing capacity of the lung for carbon monoxide (DLCO) < 30% predicted.
  6. Residual volume ≥ 120% predicted.
  7. Obstructive lung disease: forced expiratory volume in 1 second (FEV1)/FVC) < 0.70.
  8. Documented sustained improvement of the patient's IPF condition up to 12 months prior to randomization with or without IPF-specific therapy.
  9. Recent pulmonary or upper respiratory tract infection (up to 4 weeks prior to randomization).
  10. Acute or chronic impairment (other than dyspnea) limiting the ability to comply with study requirements (e.g., pulmonary function tests).
  11. Chronic heart failure with New York Heart Association class III/IV or known left ventricular ejection fraction < 25%.
  12. Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C.
  13. Estimated creatinine clearance < 30 mL/min.
  14. Aspartate aminotransferase (AST) and/or alanine aminotransferase > 1.5 x upper limit of normal.
  15. Hemoglobin < 75% of the lower limit of the normal range.
  16. Systolic blood pressure < 100 mmHg.
  17. Pregnant or breast-feeding.
  18. Current drug or alcohol dependence.
  19. Chronic treatment with the following drugs (within 4 weeks of randomization):

    • Oral corticosteroids (> 20 mg/day of prednisone or equivalent),
    • Immunosuppressive or cytotoxic drugs including cyclophosphamide and azathioprine,
    • Antifibrotic drugs including pirfenidone, D penicillamine, colchicine, tumor necrosis factor α blockers, imatinib and interferon γ,
    • Chronic use of N-acetylcysteine prescribed for IPF (> 600 mg/day).
    • Oral anticoagulants prescribed for IPF.
  20. Treatment with endothelin receptor antagonists within 4 weeks prior to randomization.
  21. Systemic treatment within 4 weeks prior to randomization with cyclosporine A or tacrolimus, everolimus, sirolimus (calcineurin or mammalian target of rapamycin (mTOR) inhibitors).
  22. Treatment with Cytochrome P450 3A inducers within 4 weeks prior to randomization.
  23. Known hypersensitivity to drugs of the same class as the study drug, or any of their excipients.
  24. Planned treatment, or treatment with another investigational drug within 4 weeks prior to randomization.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00903331

  Show 53 Study Locations
Sponsors and Collaborators
Study Chair: Loic Perchenet, Ph.D. Actelion
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT00903331     History of Changes
Other Study ID Numbers: AC-055B201
First Submitted: May 14, 2009
First Posted: May 18, 2009
Results First Submitted: October 29, 2013
Results First Posted: February 17, 2014
Last Update Posted: February 17, 2014
Last Verified: January 2014

Keywords provided by Actelion:
idiopathic pulmonary fibrosis

Additional relevant MeSH terms:
Pulmonary Fibrosis
Idiopathic Pulmonary Fibrosis
Idiopathic Interstitial Pneumonias
Pathologic Processes
Lung Diseases
Respiratory Tract Diseases
Lung Diseases, Interstitial
Endothelin A Receptor Antagonists
Endothelin Receptor Antagonists
Molecular Mechanisms of Pharmacological Action
Endothelin B Receptor Antagonists