Macitentan Use in an Idiopathic Pulmonary Fibrosis Clinical Study - Full Text View

Purpose

The AC-055B201/MUSIC study is a Phase II study, comparing one dose of macitentan (10 mg) vs 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 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 Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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:

Genetics Home Reference related topics: idiopathic pulmonary fibrosis

MedlinePlus related topics: Pulmonary Fibrosis

U.S. FDA Resources

Further study details as provided by Actelion:

Primary Outcome Measures:

To demonstrate that macitentan positively affects FVC compared with placebo in patients with IPF. [ Time Frame: Baseline - 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To evaluate the effect of macitentan on the time to disease worsening or death in patients with IPF and to evaluate the safety and tolerability of macitentan in this patient population. [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]

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: 1 ACT-064922 tablet, 10 mg, once daily	Drug: ACT-064992 (macitentan) tablet, 10 mg, once daily Other Name: macitentan
Placebo Comparator: 2 Matching placebo, one daily	Drug: Placebo matching placebo, once daily

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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 ATS/ERS 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: FVC < 50% predicted, or FVC < 1.2 liter.
DLCO < 30% predicted.
Residual volume ≥ 120% predicted.
Obstructive lung disease: 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., PFTs).
Chronic heart failure with NYHA 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.
AST and/or ALT > 1.5 x ULN.
Hemoglobin < 75% of the lower limit of the normal range.
Systolic blood pressure < 100 mmHg.
Pregnant or breast-feeding.
Current drug or alcohol dependence.
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, TNFα blocker, imatinib and interferon γ,
- Chronic use of N-acetylcysteine prescribed for IPF (> 600 mg/day).
- Oral anticoagulants prescribed for IPF.
Treatment with ERAs within 4 weeks prior to randomization.
Systemic treatment within 4 weeks prior to randomization with cyclosporine A or tacrolimus, everolimus, sirolimus (calcineurin or mTOR inhibitors).
Treatment with CYP3A 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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00903331

Show 53 Study Locations

Sponsors and Collaborators

Actelion

Investigators

Study Chair:

Loic Perchenet, Ph.D.

Actelion

More Information

No publications provided

Responsible Party:	Actelion
ClinicalTrials.gov Identifier:	NCT00903331 History of Changes
Other Study ID Numbers:	AC-055B201
Study First Received:	May 14, 2009
Last Updated:	September 27, 2012
Health Authority:	Canada: Ethics Review Committee Canada: Health Canada United States: Food and Drug Administration France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) France: Committee of Protection of Individuals SUD-EST IV (Lyon) Slovenia: Agency for Medicinal Products - Ministry of Health Slovenia: Ethics Committee Australia: Department of Health and Ageing Therapeutic Goods Administration Australia: Human Research Ethics Committee Australia: District Research Governance Germany: Ethics Commission Germany: Federal Institute for Drugs and Medical Devices South Africa: Human Research Ethics Committee South Africa: Medicines Control Council Turkey: Ethics Committee Turkey: Ministry of Health Israel: Ethics Commission Israel: Israeli Health Ministry Pharmaceutical Administration Italy: Ethics Committee Spain: Comité Ético de Investigación Clínica Spain: Spanish Agency of Medicines Sweden: Medical Products Agency Sweden: Regional Ethical Review Board

Keywords provided by Actelion:

idiopathic pulmonary fibrosis
MUSIC

Additional relevant MeSH terms:

Fibrosis
Pulmonary Fibrosis
Idiopathic Pulmonary Fibrosis
Pathologic Processes

Lung Diseases
Respiratory Tract Diseases
Idiopathic Interstitial Pneumonias
Lung Diseases, Interstitial

ClinicalTrials.gov processed this record on May 16, 2013

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