Trial Outcomes & Findings for Macitentan Use in an Idiopathic Pulmonary Fibrosis Clinical Study (NCT NCT00903331)
NCT ID: NCT00903331
Last Updated: 2014-02-17
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
178 participants
Primary outcome timeframe
12 months
Results posted on
2014-02-17
Participant Flow
The study was conducted at 48 centers in Australia, Canada, France, Germany, Israel, Italy, Slovenia, South Africa, Spain, Sweden, Turkey, and the USA.
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
Participant milestones
| Measure |
Placebo
Matching placebo, once daily
Placebo : matching placebo, once daily
|
ACT-064922
ACT-064922 tablet, 10 mg, once daily
ACT-064992 (macitentan) : tablet, 10 mg, once daily
|
|---|---|---|
|
Overall Study
STARTED
|
59
|
119
|
|
Overall Study
COMPLETED
|
54
|
101
|
|
Overall Study
NOT COMPLETED
|
5
|
18
|
Reasons for withdrawal
| Measure |
Placebo
Matching placebo, once daily
Placebo : matching placebo, once daily
|
ACT-064922
ACT-064922 tablet, 10 mg, once daily
ACT-064992 (macitentan) : tablet, 10 mg, once daily
|
|---|---|---|
|
Overall Study
Death
|
4
|
8
|
|
Overall Study
Withdrawal of consent
|
1
|
8
|
|
Overall Study
Lung transplant
|
0
|
2
|
Baseline Characteristics
Macitentan Use in an Idiopathic Pulmonary Fibrosis Clinical Study
Baseline characteristics by cohort
| Measure |
Placebo
n=59 Participants
Matching placebo, once daily
Placebo : matching placebo, once daily
|
ACT-064922
n=119 Participants
ACT-064922 tablet, 10 mg, once daily
ACT-064992 (macitentan) : tablet, 10 mg, once daily
|
Total
n=178 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
64.5 years
STANDARD_DEVIATION 6.32 • n=5 Participants
|
65.1 years
STANDARD_DEVIATION 7.85 • n=7 Participants
|
64.9 years
STANDARD_DEVIATION 7.37 • n=5 Participants
|
|
Sex: Female, Male
Female
|
22 Participants
n=5 Participants
|
35 Participants
n=7 Participants
|
57 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
37 Participants
n=5 Participants
|
84 Participants
n=7 Participants
|
121 Participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
9 participants
n=5 Participants
|
18 participants
n=7 Participants
|
27 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
5 participants
n=5 Participants
|
10 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Region of Enrollment
France
|
10 participants
n=5 Participants
|
16 participants
n=7 Participants
|
26 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
5 participants
n=5 Participants
|
8 participants
n=7 Participants
|
13 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
2 participants
n=5 Participants
|
5 participants
n=7 Participants
|
7 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
1 participants
n=5 Participants
|
7 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Region of Enrollment
Slovenia
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
South Africa
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
4 participants
n=5 Participants
|
7 participants
n=7 Participants
|
11 participants
n=5 Participants
|
|
Region of Enrollment
Sweden
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Turkey
|
3 participants
n=5 Participants
|
8 participants
n=7 Participants
|
11 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
18 participants
n=5 Participants
|
37 participants
n=7 Participants
|
55 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: All randomized patients
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.
Outcome measures
| Measure |
Placebo
n=59 Participants
Matching placebo, once daily
Placebo : matching placebo, once daily
|
ACT-064922
n=119 Participants
ACT-064922 tablet, 10 mg, once daily
ACT-064992 (macitentan) : tablet, 10 mg, once daily
|
|---|---|---|
|
Forced Vital Capacity (FVC) at Baseline and End of Period 1
Baseline
|
2.74 litres
95% Confidence Interval 0.776 • Interval 2.49 to 3.04
|
2.83 litres
95% Confidence Interval 0.834 • Interval 2.65 to 3.06
|
|
Forced Vital Capacity (FVC) at Baseline and End of Period 1
End of Period 1
|
2.40 litres
95% Confidence Interval 0.918 • Interval 2.1 to 2.7
|
2.57 litres
95% Confidence Interval 1.012 • Interval 2.28 to 2.79
|
SECONDARY outcome
Timeframe: Up to end of study (Up to 24 months)Population: All randomized patients
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).
Outcome measures
| Measure |
Placebo
n=59 Participants
Matching placebo, once daily
Placebo : matching placebo, once daily
|
ACT-064922
n=119 Participants
ACT-064922 tablet, 10 mg, once daily
ACT-064992 (macitentan) : tablet, 10 mg, once daily
|
|---|---|---|
|
Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study
Patients at Risk of Event at Month 4
|
59 participants
|
112 participants
|
|
Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study
Patients at Risk of Event at Month 8
|
57 participants
|
103 participants
|
|
Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study
Patients at Risk of Event at Month 12
|
44 participants
|
81 participants
|
|
Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study
Patients at Risk of Event at Month 16
|
22 participants
|
43 participants
|
|
Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study
Patients at Risk of Event at Month 20
|
8 participants
|
14 participants
|
|
Number of Patients at Risk of Event of Disease Worsening or Death up to the End of Study
Patients at Risk of Event at Month 24
|
2 participants
|
1 participants
|
Adverse Events
Placebo
Serious events: 20 serious events
Other events: 57 other events
Deaths: 0 deaths
ACT-064922
Serious events: 37 serious events
Other events: 114 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=59 participants at risk
Matching placebo, once daily
Placebo : matching placebo, once daily
|
ACT-064922
n=119 participants at risk
ACT-064922 tablet, 10 mg, once daily
ACT-064992 (macitentan) : tablet, 10 mg, once daily
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
IDIOPATHIC PULMONARY FIBROSIS
|
10.2%
6/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
8.4%
10/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
RESPIRATORY FAILURE
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
3.4%
4/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
HYPOXIA
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
2.5%
3/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
ACUTE RESPIRATORY FAILURE
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
1.7%
2/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY EMBOLISM
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
ACUTE RESPIRATORY DISTRESS SYNDROME
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
PNEUMONIA ASPIRATION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
HAEMOPTYSIS
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
PLEURAL EFFUSION
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY ARTERIAL HYPERTENSION
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY HYPERTENSION
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Infections and infestations
PNEUMONIA
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
5.0%
6/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Infections and infestations
LOWER RESPIRATORY TRACT INFECTION
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Infections and infestations
COMMUNITY ACQUIRED INFECTION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Infections and infestations
LOWER RESPIRATORY TRACT INFECTION BACTERIAL
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
ANGINA PECTORIS
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
ACUTE MYOCARDIAL INFARCTION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
ARTERIOSPASM CORONARY
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
ATRIAL FLUTTER
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
CORONARY ARTERY DISEASE
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
DIASTOLIC DYSFUNCTION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
ANGINA UNSTABLE
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
CARDIAC ARREST
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
SINUS BRADYCARDIA
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
GASTRIC MUCOSAL HYPERTROPHY
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
HIATUS HERNIA
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
SMALL INTESTINAL OBSTRUCTION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
UMBILICAL HERNIA
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
VOMITING
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
CHEST PAIN
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
DEVICE MALFUNCTION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
HERNIA OBSTRUCTIVE
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
PYREXIA
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Injury, poisoning and procedural complications
CYSTITIS RADIATION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Injury, poisoning and procedural complications
INCISIONAL HERNIA
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Injury, poisoning and procedural complications
LACERATION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Injury, poisoning and procedural complications
POST PROCEDURAL HAEMORRHAGE
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Injury, poisoning and procedural complications
JOINT DISLOCATION
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Injury, poisoning and procedural complications
SNAKE BITE
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Injury, poisoning and procedural complications
TRAUMATIC BRAIN INJURY
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
HEAD AND NECK CANCER
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MALIGNANT MEDIASTINAL NEOPLASM
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MYELODYSPLASTIC SYNDROME
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
RECTAL CANCER
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
SQUAMOUS CELL CARCINOMA
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
LUNG NEOPLASM
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
LUNG SQUAMOUS CELL CARCINOMA STAGE UNSPECIFIED
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Blood and lymphatic system disorders
THROMBOCYTOPENIA
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
1.7%
2/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Nervous system disorders
CEREBROVASCULAR ACCIDENT
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Nervous system disorders
TRANSIENT ISCHAEMIC ATTACK
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Nervous system disorders
DIZZINESS
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Nervous system disorders
PARAESTHESIA
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Surgical and medical procedures
INTESTINAL OPERATION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Surgical and medical procedures
MALIGNANT TUMOUR EXCISION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Surgical and medical procedures
SKIN LESION EXCISION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Surgical and medical procedures
HIP ARTHROPLASTY
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Vascular disorders
AORTIC ANEURYSM
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Vascular disorders
HYPOTENSION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Vascular disorders
HYPERTENSION
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Vascular disorders
WEGENER'S GRANULOMATOSIS
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Hepatobiliary disorders
CHOLELITHIASIS
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Immune system disorders
ALLERGY TO ARTHROPOD BITE
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Metabolism and nutrition disorders
FLUID RETENTION
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Musculoskeletal and connective tissue disorders
OSTEONECROSIS
|
1.7%
1/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.00%
0/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Renal and urinary disorders
RENAL FAILURE ACUTE
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Skin and subcutaneous tissue disorders
SKIN HAEMORRHAGE
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
Other adverse events
| Measure |
Placebo
n=59 participants at risk
Matching placebo, once daily
Placebo : matching placebo, once daily
|
ACT-064922
n=119 participants at risk
ACT-064922 tablet, 10 mg, once daily
ACT-064992 (macitentan) : tablet, 10 mg, once daily
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
15.3%
9/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
20.2%
24/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
35.6%
21/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
18.5%
22/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
20.3%
12/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
16.8%
20/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Respiratory, thoracic and mediastinal disorders
IDIOPATHIC PULMONARY FIBROSIS
|
16.9%
10/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
14.3%
17/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Infections and infestations
BRONCHITIS
|
15.3%
9/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
13.4%
16/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
OEDEMA PERIPHERAL
|
6.8%
4/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
11.8%
14/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Blood and lymphatic system disorders
ANAEMIA
|
0.00%
0/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
10.9%
13/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Nervous system disorders
DIZZINESS
|
8.5%
5/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
9.2%
11/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Investigations
PULMONARY FUNCTION TEST DECREASED
|
8.5%
5/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
7.6%
9/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Investigations
ALANINE AMINOTRANSFERASE INCREASED
|
6.8%
4/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
7.6%
9/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
NAUSEA
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
7.6%
9/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
DIARRHOEA
|
8.5%
5/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
6.7%
8/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Investigations
ASPARTATE AMINOTRANSFERASE INCREASED
|
6.8%
4/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
6.7%
8/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Psychiatric disorders
INSOMNIA
|
5.1%
3/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
6.7%
8/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Nervous system disorders
HEADACHE
|
13.6%
8/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
5.9%
7/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Infections and infestations
LOWER RESPIRATORY TRACT INFECTION
|
8.5%
5/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
5.9%
7/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
CHEST PAIN
|
5.1%
3/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
5.9%
7/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
5.0%
6/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
FATIGUE
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
5.0%
6/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Investigations
WEIGHT DECREASED
|
3.4%
2/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
5.0%
6/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
CONSTIPATION
|
5.1%
3/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
4.2%
5/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Vascular disorders
HYPERTENSION
|
8.5%
5/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
3.4%
4/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Skin and subcutaneous tissue disorders
RASH
|
5.1%
3/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
3.4%
4/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
10.2%
6/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
2.5%
3/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
PYREXIA
|
8.5%
5/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
2.5%
3/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
ANGINA PECTORIS
|
5.1%
3/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
2.5%
3/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Musculoskeletal and connective tissue disorders
NECK PAIN
|
6.8%
4/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
1.7%
2/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Metabolism and nutrition disorders
DIABETES MELLITUS
|
5.1%
3/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
1.7%
2/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Cardiac disorders
MITRAL VALVE INCOMPETENCE
|
5.1%
3/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
1.7%
2/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
General disorders
ASTHENIA
|
6.8%
4/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Gastrointestinal disorders
GASTROOESOPHAGEAL REFLUX DISEASE
|
6.8%
4/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
|
Ear and labyrinth disorders
VERTIGO
|
5.1%
3/59 • Up to 28 days after treatment discontinuation, approximately 2 years
|
0.84%
1/119 • Up to 28 days after treatment discontinuation, approximately 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60