Trial Outcomes & Findings for A Study to Assess the Safety and Tolerability of N-Acetylcysteine When Administered With Pirfenidone to Participants With Idiopathic Pulmonary Fibrosis (IPF) (NCT NCT02707640)
NCT ID: NCT02707640
Last Updated: 2016-05-20
Results Overview
Percentage of participants with dose reductions in N-Acetylcysteine and placebo cohorts during the 24-week treatment period.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
123 participants
Primary outcome timeframe
From baseline up to 24 weeks
Results posted on
2016-05-20
Participant Flow
Participant milestones
| Measure |
N-Acetylcysteine (NAC)
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Overall Study
STARTED
|
61
|
62
|
|
Overall Study
COMPLETED
|
52
|
55
|
|
Overall Study
NOT COMPLETED
|
9
|
7
|
Reasons for withdrawal
| Measure |
N-Acetylcysteine (NAC)
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Overall Study
Adverse Event
|
4
|
2
|
|
Overall Study
Death
|
1
|
2
|
|
Overall Study
Physician Decision
|
2
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
|
Overall Study
Participant's Personal Decision
|
1
|
2
|
|
Overall Study
Sponsor Discretion
|
1
|
0
|
Baseline Characteristics
A Study to Assess the Safety and Tolerability of N-Acetylcysteine When Administered With Pirfenidone to Participants With Idiopathic Pulmonary Fibrosis (IPF)
Baseline characteristics by cohort
| Measure |
N-Acetylcysteine (NAC)
n=60 Participants
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 Participants
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Total
n=122 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
66.7 years
STANDARD_DEVIATION 7.99 • n=99 Participants
|
67.5 years
STANDARD_DEVIATION 6.22 • n=107 Participants
|
67.1 years
STANDARD_DEVIATION 7.13 • n=206 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
18 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
53 Participants
n=99 Participants
|
51 Participants
n=107 Participants
|
104 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: From baseline up to 24 weeksPopulation: mITT population included participants who received at least 1 dose of double-blind study medication (NAC or placebo).
Percentage of participants with dose reductions in N-Acetylcysteine and placebo cohorts during the 24-week treatment period.
Outcome measures
| Measure |
N-Acetylcysteine (NAC)
n=60 Participants
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 Participants
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Percentage of Participants With Dose Reductions
|
5 percentage of participants
|
4.8 percentage of participants
|
PRIMARY outcome
Timeframe: From baseline up to 24 weeksPopulation: mITT population included participants who received at least 1 dose of double-blind study medication (NAC or placebo).
Percentage of participants with early treatment discontinuations in N-Acetylcysteine and placebo cohorts during the 24-week treatment period.
Outcome measures
| Measure |
N-Acetylcysteine (NAC)
n=60 Participants
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 Participants
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Percentage of Participants With Early Treatment Discontinuations
|
14.8 percentage of participants
|
11.3 percentage of participants
|
PRIMARY outcome
Timeframe: Until 28 days from last dose of study treatment (Week 28)Population: mITT Population included participants who received at least 1 dose of double-blind study medication (NAC or placebo).
An adverse event (AE) is defined as any untoward medical occurrence in a participant who is administered a study treatment regardless of whether or not the event has a causal relationship with the treatment. An AE, therefore, could be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the study treatment, whether or not related to the treatment.
Outcome measures
| Measure |
N-Acetylcysteine (NAC)
n=60 Participants
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 Participants
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)
|
76.7 percentage of participants
|
80.6 percentage of participants
|
PRIMARY outcome
Timeframe: Until 28 days from last dose of study treatment (Week 28)Population: mITT population included who received at least 1 dose of double-blind study medication (NAC or placebo).
A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose results in death, is life threatening, requires hospitalization or prolongation of hospitalization, or results in disability/incapacity, or congenital anomaly/birth defect.
Outcome measures
| Measure |
N-Acetylcysteine (NAC)
n=60 Participants
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 Participants
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Percentage of Participants With Treatment-Emergent Serious Adverse Events (SAEs)
|
5.0 percentage of participants
|
6.5 percentage of participants
|
PRIMARY outcome
Timeframe: Until 28 days from last dose of study treatment (Week 28)Population: mITT population included who received at least 1 dose of double-blind study medication (NAC or placebo).
Outcome measures
| Measure |
N-Acetylcysteine (NAC)
n=60 Participants
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 Participants
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Percentage of Participants With Treatment-Emergent Adverse Events Resulting in Permanent Discontinuation of Study Treatment
|
6.7 percentage of participants
|
1.6 percentage of participants
|
PRIMARY outcome
Timeframe: Until 28 days from last dose of study treatment (Week 28)Population: mITT population included who received at least 1 dose of double-blind study medication (NAC or placebo).
Outcome measures
| Measure |
N-Acetylcysteine (NAC)
n=60 Participants
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 Participants
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Percentage of Participants With Treatment-Emergent Deaths of All Causes
|
1.7 percentage of participants
|
4.8 percentage of participants
|
PRIMARY outcome
Timeframe: Until 28 days from last dose of study treatment (Week 28)Population: mITT population included who received at least 1 dose of double-blind study medication (NAC or placebo).
Outcome measures
| Measure |
N-Acetylcysteine (NAC)
n=60 Participants
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 Participants
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Percentage of Participants With Treatment-Emergent Adverse Events That Led to Dose Reduction or Temporary Discontinuation of Study Treatment
|
10 percentage of participants
|
6.5 percentage of participants
|
Adverse Events
N-Acetylcysteine (NAC)
Serious events: 3 serious events
Other events: 46 other events
Deaths: 0 deaths
Placebo
Serious events: 4 serious events
Other events: 50 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
N-Acetylcysteine (NAC)
n=60 participants at risk
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 participants at risk
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Injury, poisoning and procedural complications
CONTUSION
|
0.00%
0/60 • Until 28 days from last dose of study treatment (Week 28)
|
1.6%
1/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Injury, poisoning and procedural complications
FOREARM FRACTURE
|
0.00%
0/60 • Until 28 days from last dose of study treatment (Week 28)
|
1.6%
1/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Musculoskeletal and connective tissue disorders
INTERVERTEBRAL DISC PROTRUSION
|
1.7%
1/60 • Until 28 days from last dose of study treatment (Week 28)
|
0.00%
0/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
LUNG NEOPLASM MALIGNANT
|
1.7%
1/60 • Until 28 days from last dose of study treatment (Week 28)
|
0.00%
0/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Nervous system disorders
HEADACHE
|
1.7%
1/60 • Until 28 days from last dose of study treatment (Week 28)
|
0.00%
0/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
1.7%
1/60 • Until 28 days from last dose of study treatment (Week 28)
|
0.00%
0/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Respiratory, thoracic and mediastinal disorders
IDIOPATHIC PULMONARY FIBROSIS
|
0.00%
0/60 • Until 28 days from last dose of study treatment (Week 28)
|
3.2%
2/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Vascular disorders
AORTIC ANEURYSM
|
0.00%
0/60 • Until 28 days from last dose of study treatment (Week 28)
|
1.6%
1/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Vascular disorders
HYPERTENSION
|
1.7%
1/60 • Until 28 days from last dose of study treatment (Week 28)
|
0.00%
0/62 • Until 28 days from last dose of study treatment (Week 28)
|
Other adverse events
| Measure |
N-Acetylcysteine (NAC)
n=60 participants at risk
Participants randomized to this arm were administered 600 milligram (mg) NAC orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
Placebo
n=62 participants at risk
Participants randomized to this arm were administered matching placebo orally three times daily and a dose of pirfenidone of at least 1602 mg/day for 24 weeks. Participants were to be on a dose of pirfenidone of at least 1602 mg/day for a minimum of 8 weeks prior to randomization and were followed until 4 weeks after last study treatment dose.
|
|---|---|---|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
6.7%
4/60 • Until 28 days from last dose of study treatment (Week 28)
|
0.00%
0/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Gastrointestinal disorders
DIARRHOEA
|
10.0%
6/60 • Until 28 days from last dose of study treatment (Week 28)
|
14.5%
9/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Gastrointestinal disorders
NAUSEA
|
6.7%
4/60 • Until 28 days from last dose of study treatment (Week 28)
|
8.1%
5/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Infections and infestations
BRONCHITIS
|
6.7%
4/60 • Until 28 days from last dose of study treatment (Week 28)
|
4.8%
3/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Infections and infestations
INFLUENZA
|
5.0%
3/60 • Until 28 days from last dose of study treatment (Week 28)
|
1.6%
1/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Infections and infestations
NASOPHARYNGITIS
|
11.7%
7/60 • Until 28 days from last dose of study treatment (Week 28)
|
11.3%
7/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
5.0%
3/60 • Until 28 days from last dose of study treatment (Week 28)
|
8.1%
5/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Musculoskeletal and connective tissue disorders
MYALGIA
|
5.0%
3/60 • Until 28 days from last dose of study treatment (Week 28)
|
1.6%
1/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Nervous system disorders
HEADACHE
|
1.7%
1/60 • Until 28 days from last dose of study treatment (Week 28)
|
8.1%
5/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
13.3%
8/60 • Until 28 days from last dose of study treatment (Week 28)
|
11.3%
7/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
5.0%
3/60 • Until 28 days from last dose of study treatment (Week 28)
|
6.5%
4/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Respiratory, thoracic and mediastinal disorders
PRODUCTIVE COUGH
|
5.0%
3/60 • Until 28 days from last dose of study treatment (Week 28)
|
3.2%
2/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Skin and subcutaneous tissue disorders
DRY SKIN
|
5.0%
3/60 • Until 28 days from last dose of study treatment (Week 28)
|
0.00%
0/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Skin and subcutaneous tissue disorders
PHOTOSENSITIVITY REACTION
|
13.3%
8/60 • Until 28 days from last dose of study treatment (Week 28)
|
1.6%
1/62 • Until 28 days from last dose of study treatment (Week 28)
|
|
Skin and subcutaneous tissue disorders
RASH
|
3.3%
2/60 • Until 28 days from last dose of study treatment (Week 28)
|
9.7%
6/62 • Until 28 days from last dose of study treatment (Week 28)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER