Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12609000458235
Ethics application status
Approved
Date submitted
28/01/2009
Date registered
15/06/2009
Date last updated
29/08/2018
Type of registration
Retrospectively registered

Titles & IDs
Public title
Study to evaluate if macitentan is efficient and safe enough to be used for treatment of idiopathic pulmonary fibrosis.
Scientific 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.
Secondary ID [1] 259822 0
NCT00903331
Universal Trial Number (UTN)
Trial acronym
MUSIC: Macitentan USe in an Idiopathic pulmonary fibrosis Clinical study
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Idiopathic Pulmonary Fibrosis 4239 0
Condition category
Condition code
Respiratory 4363 4363 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Macitentan 10 mg, tablet. Taken orally, once daily, every morning throughout Period 1 (12 months) and Period 2 (variable, at least a further 12 months), until the last patient has completed 24 months therapy (Period 1 and 2 combined) unless the study medication is prematurely discontinued.
Intervention code [1] 3872 0
Treatment: Drugs
Comparator / control treatment
Placebo (sugar pill) alone. Tablet taken orally, once daily, every morning throughout Period 1 (12 months) and Period 2 (variable, at least a further 12 months), until the last patient has completed 24 months therapy (Period 1 and 2 combined) unless the study medication is prematurely discontinued.
Control group
Placebo

Outcomes
Primary outcome [1] 5524 0
Primary efficacy endpoint: Change from baseline to End-of-Period 1 in Forced Vital Capacity (FVC) as measured by Pulmonary Function Tests (PFTs).
A mean difference from placebo of at least 0.1 liter is to be detected and is considered clinically meaningful. Patients with an at least stable FVC have a statistically significant survival benefit over those with a decrease in FVC [Noble 2006]. Thus, change in FVC at 1 year provides a reasonable basis for evaluating efficacy of a new treatment.
Timepoint [1] 5524 0
End-of-Period 1: occurs at 12 months after subject randomisation or earlier in case of premature discontinuation of study medication during Period 1.
Secondary outcome [1] 9308 0
Secondary efficacy endpoint: Time to occurrence of disease worsening or death up to End-of-Study. Disease worsening is defined as worsening of Pulmonary Function Tests (PFTs) or acute respiratory decompensation of Idiopathic Pulmonary Fibrosis (IPF).
Timepoint [1] 9308 0
At End-of-Study, after the last patient randomised has completed at least 24 months therapy.

Eligibility
Key inclusion criteria
- Signed informed consent.
- IPF diagnosis within 3 years prior to randomization, proven according to American Thoracic Society/European Respiratory Society (ATS/ERS) consensus conference criteria, with surgical lung biopsy.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.
- Forced Vital Capacity (FVC) < 50% predicted, or FVC < 1.2 liter.
- Diffusing capacity of the lung for carbon monoxide (DLco) < 30% predicted.
- Residual volume at least 120% predicted.
- Forced expiratory volume of the lung in 1 second (FEV1)/FVC <0.70.
- Aspartate aminotransferase (AST) and/or Alanine aminotransferase (ALT) > 1.5 x Upper limit of Normal (ULN).
- Hemoglobin < 75% of the lower limit of the normal range.
- Systolic Blood Pressure (sBP) < 100 mmHg.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Allocation is done by a central randomisation system.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Stratified allocation (stratification by site). Simple randomisation by using a randomisation table created by a computer software (i.e., computerised sequence generation).
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Date of first participant enrolment
Anticipated
27/05/2009
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
156
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment postcode(s) [1] 1352 0
4032
Recruitment postcode(s) [2] 1353 0
6000
Recruitment postcode(s) [3] 1354 0
2010
Recruitment postcode(s) [4] 3866 0
3004
Recruitment outside Australia
Country [1] 1483 0
Germany
State/province [1] 1483 0
Country [2] 1484 0
France
State/province [2] 1484 0
Country [3] 1485 0
Italy
State/province [3] 1485 0
Country [4] 1486 0
Israel
State/province [4] 1486 0
Country [5] 1487 0
Canada
State/province [5] 1487 0
Country [6] 1488 0
United States of America
State/province [6] 1488 0
Country [7] 1489 0
Turkey
State/province [7] 1489 0
Country [8] 1490 0
South Africa
State/province [8] 1490 0
Country [9] 1491 0
Spain
State/province [9] 1491 0
Country [10] 1492 0
Slovenia
State/province [10] 1492 0
Country [11] 1493 0
Sweden
State/province [11] 1493 0

Funding & Sponsors
Funding source category [1] 4586 0
Commercial sector/Industry
Name [1] 4586 0
Actelion Pharmaceuticals Australia Pty Ltd
Country [1] 4586 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Actelion Pharmaceuticals Australia Pty Ltd
Address
Suite 6, 13 B Narabang Way,
Belrose NSW 2085, Australia
Country
Australia
Secondary sponsor category [1] 3897 0
None
Name [1] 3897 0
Address [1] 3897 0
Country [1] 3897 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 6374 0
Royal Perth Hospital Ethics Committee
Ethics committee address [1] 6374 0
Room 4112 Level 4, Kirkman House, 10 Murray St, Royal Perth Hospital
Perth WA 6000
Ethics committee country [1] 6374 0
Australia
Date submitted for ethics approval [1] 6374 0
04/02/2009
Approval date [1] 6374 0
26/03/2009
Ethics approval number [1] 6374 0
Ethics committee name [2] 6375 0
The Prince Charles Hospital Metro North Health Service District
Ethics committee address [2] 6375 0
Rode Road
CHERMSIDE QLD 4032
Ethics committee country [2] 6375 0
Australia
Date submitted for ethics approval [2] 6375 0
05/02/2009
Approval date [2] 6375 0
31/03/2009
Ethics approval number [2] 6375 0
HREC/09/QPCH/7
Ethics committee name [3] 6376 0
St Vincents Hospital Human Research Ethics Committee
Ethics committee address [3] 6376 0
Research Office Level 6 de Lacey Building St. Vincent's Hospital 390 Victoria Street DARLINGHURST NSW 2010 Australia
Ethics committee country [3] 6376 0
Australia
Date submitted for ethics approval [3] 6376 0
23/02/2009
Approval date [3] 6376 0
21/04/2009
Ethics approval number [3] 6376 0
HREC/09/SVH/20
Ethics committee name [4] 239253 0
Alfred Hospital Ethics Commitee
Ethics committee address [4] 239253 0
Alfred Hospital,
Commercial Rd
Melbourne VIC 3004
Ethics committee country [4] 239253 0
Australia
Date submitted for ethics approval [4] 239253 0
27/04/2009
Approval date [4] 239253 0
24/06/2009
Ethics approval number [4] 239253 0
148/09

Summary
Brief summary
Idiopathic pulmonary fibrosis (IPF) is a progressive, fatal lung disease with an unknown cause. Outside of Japan, no drug has been approved so far for the treatment of IPF.
The study medication that is tested in this research, macitentan, works by blocking the effect of a substance called endothelin, which has been detected in increased amounts in patients with IPF. By blocking the action of endothelin, macitentan may increase the breathing capacity, improve the quality of life of patients and reduce the progression of the disease.
The main purpose of this study is to find out if macitentan is efficient and safe enough to be used for treatment of IPF.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 35059 0
Address 35059 0
Country 35059 0
Phone 35059 0
Fax 35059 0
Email 35059 0
Contact person for public queries
Name 12406 0
Ms Tanya Robb
Address 12406 0
Regional Monitor
Actelion Pharmaceuticals Australia Pty Ltd
Suite 6, 13 B Narabang Way,
Belrose NSW 2085, Australia
Country 12406 0
Australia
Phone 12406 0
+612 94864600
Fax 12406 0
+612 99861344
Email 12406 0
[email protected]
Contact person for scientific queries
Name 3334 0
Dr Glen Pater
Address 3334 0
Regional Medical Director
Actelion Pharmaceuticals Australia Pty Ltd
Suite 6, 13 B Narabang Way,
Belrose NSW 2085, Australia
Country 3334 0
Australia
Phone 3334 0
+612 94864600
Fax 3334 0
+612 99861344
Email 3334 0
[email protected]

No information has been provided regarding IPD availability


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No Supporting Document Provided



Results publications and other study-related documents

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