Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 25 of 317 for:    "Pulmonary Fibrosis, Idiopathic"

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

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.
ClinicalTrials.gov Identifier: NCT00903331
Recruitment Status : Completed
First Posted : May 18, 2009
Results First Posted : February 17, 2014
Last Update Posted : February 17, 2014
Sponsor:
Information provided by (Responsible Party):
Actelion

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Idiopathic Pulmonary Fibrosis
Interventions Drug: ACT-064992 (macitentan)
Drug: Placebo
Enrollment 178
Recruitment Details The study was conducted at 48 centers in Australia, Canada, France, Germany, Israel, Italy, Slovenia, South Africa, Spain, Sweden, Turkey, and the USA.
Pre-assignment Details The study included a screening period of up to 28 days followed by a double-blind treatment phase that was further divided into two periods. 300 patients were screened and 178 randomized in a 2:1 ratio to study treatment with ACT-064922 or placebo
Arm/Group Title Placebo ACT-064922
Hide Arm/Group Description

Matching placebo, once daily

Placebo : matching placebo, once daily

ACT-064922 tablet, 10 mg, once daily

ACT-064992 (macitentan) : tablet, 10 mg, once daily

Period Title: Overall Study
Started 59 119
Completed 54 101
Not Completed 5 18
Reason Not Completed
Death             4             8
Withdrawal of consent             1             8
Lung transplant             0             2
Arm/Group Title Placebo ACT-064922 Total
Hide Arm/Group Description

Matching placebo, once daily

Placebo : matching placebo, once daily

ACT-064922 tablet, 10 mg, once daily

ACT-064992 (macitentan) : tablet, 10 mg, once daily

Total of all reporting groups
Overall Number of Baseline Participants 59 119 178
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 59 participants 119 participants 178 participants
64.5  (6.32) 65.1  (7.85) 64.9  (7.37)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 59 participants 119 participants 178 participants
Female
22
  37.3%
35
  29.4%
57
  32.0%
Male
37
  62.7%
84
  70.6%
121
  68.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 59 participants 119 participants 178 participants
Australia 9 18 27
Canada 5 10 15
France 10 16 26
Germany 5 8 13
Israel 2 5 7
Italy 1 7 8
Slovenia 1 1 2
South Africa 1 1 2
Spain 4 7 11
Sweden 0 1 1
Turkey 3 8 11
United States 18 37 55
1.Primary Outcome
Title Forced Vital Capacity (FVC) at Baseline and End of Period 1
Hide Description 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.
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized patients
Arm/Group Title Placebo ACT-064922
Hide Arm/Group Description:

Matching placebo, once daily

Placebo : matching placebo, once daily

ACT-064922 tablet, 10 mg, once daily

ACT-064992 (macitentan) : tablet, 10 mg, once daily

Overall Number of Participants Analyzed 59 119
Median (95% Confidence Interval)
Unit of Measure: litres
Baseline
2.74
(2.49 to 3.04)
2.83
(2.65 to 3.06)
End of Period 1
2.40
(2.10 to 2.70)
2.57
(2.28 to 2.79)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, ACT-064922
Comments The null hypothesis was that there was no difference between ACT-064922 and placebo for the change in FVC from baseline to the end of Period 1. The aim was to detect a placebo-corrected change in FVC of ≥ 0.1 L (Standard Deviation = 0.2 L) at a two-sided 0.05 type 1 error level and 80% power.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9631
Comments [Not Specified]
Method Wilcoxon Rank Sum
Comments [Not Specified]
Method of Estimation Estimation Parameter Median Difference (Net)
Estimated Value 0.00
Confidence Interval (2-Sided) 95%
-0.09 to 0.08
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study
Hide Description

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).

Time Frame Up to end of study (Up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized patients
Arm/Group Title Placebo ACT-064922
Hide Arm/Group Description:

Matching placebo, once daily

Placebo : matching placebo, once daily

ACT-064922 tablet, 10 mg, once daily

ACT-064992 (macitentan) : tablet, 10 mg, once daily

Overall Number of Participants Analyzed 59 119
Measure Type: Number
Unit of Measure: participants
Patients at Risk of Event at Month 4 59 112
Patients at Risk of Event at Month 8 57 103
Patients at Risk of Event at Month 12 44 81
Patients at Risk of Event at Month 16 22 43
Patients at Risk of Event at Month 20 8 14
Patients at Risk of Event at Month 24 2 1
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, ACT-064922
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7056
Comments [Not Specified]
Method Log Rank
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.118
Confidence Interval (2-Sided) 95%
0.626 to 1.996
Estimation Comments [Not Specified]
Time Frame Up to 28 days after treatment discontinuation, approximately 2 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo ACT-064922
Hide Arm/Group Description

Matching placebo, once daily

Placebo : matching placebo, once daily

ACT-064922 tablet, 10 mg, once daily

ACT-064992 (macitentan) : tablet, 10 mg, once daily

All-Cause Mortality
Placebo ACT-064922
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo ACT-064922
Affected / at Risk (%) Affected / at Risk (%)
Total   20/59 (33.90%)   37/119 (31.09%) 
Blood and lymphatic system disorders     
THROMBOCYTOPENIA  1  0/59 (0.00%)  2/119 (1.68%) 
Cardiac disorders     
ANGINA PECTORIS  1  1/59 (1.69%)  1/119 (0.84%) 
ACUTE MYOCARDIAL INFARCTION  1  0/59 (0.00%)  1/119 (0.84%) 
ARTERIOSPASM CORONARY  1  0/59 (0.00%)  1/119 (0.84%) 
ATRIAL FLUTTER  1  0/59 (0.00%)  1/119 (0.84%) 
CORONARY ARTERY DISEASE  1  0/59 (0.00%)  1/119 (0.84%) 
DIASTOLIC DYSFUNCTION  1  0/59 (0.00%)  1/119 (0.84%) 
ANGINA UNSTABLE  1  1/59 (1.69%)  0/119 (0.00%) 
CARDIAC ARREST  1  1/59 (1.69%)  0/119 (0.00%) 
SINUS BRADYCARDIA  1  1/59 (1.69%)  0/119 (0.00%) 
Gastrointestinal disorders     
GASTRIC MUCOSAL HYPERTROPHY  1  0/59 (0.00%)  1/119 (0.84%) 
HIATUS HERNIA  1  0/59 (0.00%)  1/119 (0.84%) 
SMALL INTESTINAL OBSTRUCTION  1  0/59 (0.00%)  1/119 (0.84%) 
UMBILICAL HERNIA  1  0/59 (0.00%)  1/119 (0.84%) 
VOMITING  1  1/59 (1.69%)  0/119 (0.00%) 
General disorders     
CHEST PAIN  1  0/59 (0.00%)  1/119 (0.84%) 
DEVICE MALFUNCTION  1  0/59 (0.00%)  1/119 (0.84%) 
HERNIA OBSTRUCTIVE  1  0/59 (0.00%)  1/119 (0.84%) 
PYREXIA  1  0/59 (0.00%)  1/119 (0.84%) 
Hepatobiliary disorders     
CHOLELITHIASIS  1  1/59 (1.69%)  1/119 (0.84%) 
Immune system disorders     
ALLERGY TO ARTHROPOD BITE  1  0/59 (0.00%)  1/119 (0.84%) 
Infections and infestations     
PNEUMONIA  1  2/59 (3.39%)  6/119 (5.04%) 
LOWER RESPIRATORY TRACT INFECTION  1  2/59 (3.39%)  1/119 (0.84%) 
COMMUNITY ACQUIRED INFECTION  1  0/59 (0.00%)  1/119 (0.84%) 
LOWER RESPIRATORY TRACT INFECTION BACTERIAL  1  0/59 (0.00%)  1/119 (0.84%) 
Injury, poisoning and procedural complications     
CYSTITIS RADIATION  1  0/59 (0.00%)  1/119 (0.84%) 
INCISIONAL HERNIA  1  0/59 (0.00%)  1/119 (0.84%) 
LACERATION  1  0/59 (0.00%)  1/119 (0.84%) 
POST PROCEDURAL HAEMORRHAGE  1  0/59 (0.00%)  1/119 (0.84%) 
JOINT DISLOCATION  1  1/59 (1.69%)  0/119 (0.00%) 
SNAKE BITE  1  1/59 (1.69%)  0/119 (0.00%) 
TRAUMATIC BRAIN INJURY  1  1/59 (1.69%)  0/119 (0.00%) 
Metabolism and nutrition disorders     
FLUID RETENTION  1  0/59 (0.00%)  1/119 (0.84%) 
Musculoskeletal and connective tissue disorders     
PAIN IN EXTREMITY  1  0/59 (0.00%)  1/119 (0.84%) 
BACK PAIN  1  1/59 (1.69%)  0/119 (0.00%) 
OSTEONECROSIS  1  1/59 (1.69%)  0/119 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
HEAD AND NECK CANCER  1  0/59 (0.00%)  1/119 (0.84%) 
MALIGNANT MEDIASTINAL NEOPLASM  1  0/59 (0.00%)  1/119 (0.84%) 
MYELODYSPLASTIC SYNDROME  1  0/59 (0.00%)  1/119 (0.84%) 
RECTAL CANCER  1  0/59 (0.00%)  1/119 (0.84%) 
SQUAMOUS CELL CARCINOMA  1  0/59 (0.00%)  1/119 (0.84%) 
LUNG NEOPLASM  1  1/59 (1.69%)  0/119 (0.00%) 
LUNG SQUAMOUS CELL CARCINOMA STAGE UNSPECIFIED  1  1/59 (1.69%)  0/119 (0.00%) 
Nervous system disorders     
CEREBROVASCULAR ACCIDENT  1  0/59 (0.00%)  1/119 (0.84%) 
TRANSIENT ISCHAEMIC ATTACK  1  0/59 (0.00%)  1/119 (0.84%) 
DIZZINESS  1  1/59 (1.69%)  0/119 (0.00%) 
PARAESTHESIA  1  1/59 (1.69%)  0/119 (0.00%) 
Renal and urinary disorders     
RENAL FAILURE ACUTE  1  0/59 (0.00%)  1/119 (0.84%) 
Respiratory, thoracic and mediastinal disorders     
IDIOPATHIC PULMONARY FIBROSIS  1  6/59 (10.17%)  10/119 (8.40%) 
RESPIRATORY FAILURE  1  2/59 (3.39%)  4/119 (3.36%) 
HYPOXIA  1  2/59 (3.39%)  3/119 (2.52%) 
ACUTE RESPIRATORY FAILURE  1  1/59 (1.69%)  2/119 (1.68%) 
PULMONARY EMBOLISM  1  2/59 (3.39%)  1/119 (0.84%) 
ACUTE RESPIRATORY DISTRESS SYNDROME  1  0/59 (0.00%)  1/119 (0.84%) 
PNEUMONIA ASPIRATION  1  0/59 (0.00%)  1/119 (0.84%) 
HAEMOPTYSIS  1  1/59 (1.69%)  0/119 (0.00%) 
PLEURAL EFFUSION  1  1/59 (1.69%)  0/119 (0.00%) 
PULMONARY ARTERIAL HYPERTENSION  1  1/59 (1.69%)  0/119 (0.00%) 
PULMONARY HYPERTENSION  1  1/59 (1.69%)  0/119 (0.00%) 
Skin and subcutaneous tissue disorders     
SKIN HAEMORRHAGE  1  0/59 (0.00%)  1/119 (0.84%) 
Surgical and medical procedures     
INTESTINAL OPERATION  1  0/59 (0.00%)  1/119 (0.84%) 
MALIGNANT TUMOUR EXCISION  1  0/59 (0.00%)  1/119 (0.84%) 
SKIN LESION EXCISION  1  0/59 (0.00%)  1/119 (0.84%) 
HIP ARTHROPLASTY  1  2/59 (3.39%)  0/119 (0.00%) 
Vascular disorders     
AORTIC ANEURYSM  1  1/59 (1.69%)  1/119 (0.84%) 
HYPOTENSION  1  0/59 (0.00%)  1/119 (0.84%) 
HYPERTENSION  1  1/59 (1.69%)  0/119 (0.00%) 
WEGENER'S GRANULOMATOSIS  1  1/59 (1.69%)  0/119 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 14.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo ACT-064922
Affected / at Risk (%) Affected / at Risk (%)
Total   57/59 (96.61%)   114/119 (95.80%) 
Blood and lymphatic system disorders     
ANAEMIA  1  0/59 (0.00%)  13/119 (10.92%) 
Cardiac disorders     
ANGINA PECTORIS  1  3/59 (5.08%)  3/119 (2.52%) 
MITRAL VALVE INCOMPETENCE  1  3/59 (5.08%)  2/119 (1.68%) 
Ear and labyrinth disorders     
VERTIGO  1  3/59 (5.08%)  1/119 (0.84%) 
Gastrointestinal disorders     
NAUSEA  1  2/59 (3.39%)  9/119 (7.56%) 
DIARRHOEA  1  5/59 (8.47%)  8/119 (6.72%) 
CONSTIPATION  1  3/59 (5.08%)  5/119 (4.20%) 
GASTROOESOPHAGEAL REFLUX DISEASE  1  4/59 (6.78%)  1/119 (0.84%) 
General disorders     
OEDEMA PERIPHERAL  1  4/59 (6.78%)  14/119 (11.76%) 
CHEST PAIN  1  3/59 (5.08%)  7/119 (5.88%) 
FATIGUE  1  2/59 (3.39%)  6/119 (5.04%) 
PYREXIA  1  5/59 (8.47%)  3/119 (2.52%) 
ASTHENIA  1  4/59 (6.78%)  1/119 (0.84%) 
Infections and infestations     
UPPER RESPIRATORY TRACT INFECTION  1  12/59 (20.34%)  20/119 (16.81%) 
BRONCHITIS  1  9/59 (15.25%)  16/119 (13.45%) 
LOWER RESPIRATORY TRACT INFECTION  1  5/59 (8.47%)  7/119 (5.88%) 
Investigations     
PULMONARY FUNCTION TEST DECREASED  1  5/59 (8.47%)  9/119 (7.56%) 
ALANINE AMINOTRANSFERASE INCREASED  1  4/59 (6.78%)  9/119 (7.56%) 
ASPARTATE AMINOTRANSFERASE INCREASED  1  4/59 (6.78%)  8/119 (6.72%) 
WEIGHT DECREASED  1  2/59 (3.39%)  6/119 (5.04%) 
Metabolism and nutrition disorders     
DIABETES MELLITUS  1  3/59 (5.08%)  2/119 (1.68%) 
Musculoskeletal and connective tissue disorders     
ARTHRALGIA  1  2/59 (3.39%)  6/119 (5.04%) 
BACK PAIN  1  6/59 (10.17%)  3/119 (2.52%) 
NECK PAIN  1  4/59 (6.78%)  2/119 (1.68%) 
Nervous system disorders     
DIZZINESS  1  5/59 (8.47%)  11/119 (9.24%) 
HEADACHE  1  8/59 (13.56%)  7/119 (5.88%) 
Psychiatric disorders     
INSOMNIA  1  3/59 (5.08%)  8/119 (6.72%) 
Respiratory, thoracic and mediastinal disorders     
DYSPNOEA  1  9/59 (15.25%)  24/119 (20.17%) 
COUGH  1  21/59 (35.59%)  22/119 (18.49%) 
IDIOPATHIC PULMONARY FIBROSIS  1  10/59 (16.95%)  17/119 (14.29%) 
Skin and subcutaneous tissue disorders     
RASH  1  3/59 (5.08%)  4/119 (3.36%) 
Vascular disorders     
HYPERTENSION  1  5/59 (8.47%)  4/119 (3.36%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 14.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Parisa Danaietash
Organization: Actelion Pharmaceuticals Ltd
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