Macitentan Use in an Idiopathic Pulmonary Fibrosis Clinical Study (MUSIC)
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| ClinicalTrials.gov Identifier: NCT00903331 |
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Recruitment Status :
Completed
First Posted : May 18, 2009
Results First Posted : February 17, 2014
Last Update Posted : February 17, 2014
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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 or disease | Intervention/treatment | Phase |
|---|---|---|
| Idiopathic Pulmonary Fibrosis | Drug: ACT-064992 (macitentan) Drug: Placebo | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 178 participants |
| 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 |
| Study Start Date : | May 2009 |
| Actual Primary Completion Date : | June 2011 |
| Actual Study Completion Date : | August 2011 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: ACT-064922
ACT-064922 tablet (macitentan), 10 mg, once daily
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Drug: ACT-064992 (macitentan)
ACT-064992 (macitentan) tablet, 10 mg, once daily
Other Name: macitentan |
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Placebo Comparator: Placebo
Matching placebo, once daily
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Drug: Placebo
matching placebo, once daily |
- 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.
- 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).
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Signed informed consent.
- Male or female patients of at least 18 years of age (females of child-bearing potential must use a reliable method of contraception).
- 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:
- Interstitial lung disease due to conditions other than IPF.
- Presence of extensive honeycombing on Baseline high-resolution computed tomography (HRCT) scan performed within 3 months prior to randomization.
- Severe concomitant illness limiting life expectancy (< 1 year).
- Severe restrictive lung disease: forced vital capacity (FVC) < 50% predicted, or FVC < 1.2 liter.
- Diffusing capacity of the lung for carbon monoxide (DLCO) < 30% predicted.
- Residual volume ≥ 120% predicted.
- Obstructive lung disease: forced expiratory volume in 1 second (FEV1)/FVC) < 0.70.
- Documented sustained improvement of the patient's IPF condition up to 12 months prior to randomization with or without IPF-specific therapy.
- Recent pulmonary or upper respiratory tract infection (up to 4 weeks prior to randomization).
- Acute or chronic impairment (other than dyspnea) limiting the ability to comply with study requirements (e.g., pulmonary function tests).
- Chronic heart failure with New York Heart Association class III/IV or known left ventricular ejection fraction < 25%.
- Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C.
- Estimated creatinine clearance < 30 mL/min.
- Aspartate aminotransferase (AST) and/or alanine aminotransferase > 1.5 x upper limit of normal.
- Hemoglobin < 75% of the lower limit of the normal range.
- Systolic blood pressure < 100 mmHg.
- Pregnant or breast-feeding.
- Current drug or alcohol dependence.
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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.
- Treatment with endothelin receptor antagonists within 4 weeks prior to randomization.
- Systemic treatment within 4 weeks prior to randomization with cyclosporine A or tacrolimus, everolimus, sirolimus (calcineurin or mammalian target of rapamycin (mTOR) inhibitors).
- Treatment with Cytochrome P450 3A inducers within 4 weeks prior to randomization.
- Known hypersensitivity to drugs of the same class as the study drug, or any of their excipients.
- Planned treatment, or treatment with another investigational drug within 4 weeks prior to randomization.
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
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| Study Chair: | Loic Perchenet, Ph.D. | Actelion |
| Responsible Party: | Actelion |
| ClinicalTrials.gov Identifier: | NCT00903331 |
| Other Study ID Numbers: |
AC-055B201 |
| First Posted: | May 18, 2009 Key Record Dates |
| Results First Posted: | February 17, 2014 |
| Last Update Posted: | February 17, 2014 |
| Last Verified: | January 2014 |
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idiopathic pulmonary fibrosis MUSIC |
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Pulmonary Fibrosis Idiopathic Pulmonary Fibrosis Fibrosis Pathologic Processes Lung Diseases, Interstitial Lung Diseases |
Respiratory Tract Diseases Macitentan Endothelin A Receptor Antagonists Endothelin Receptor Antagonists Molecular Mechanisms of Pharmacological Action Endothelin B Receptor Antagonists |