Idiopathic Pulmonary Fibrosis
Conditions
Keywords
Pirfenidone, ASCEND, IPF, FVC
Brief summary
PIPF-016 (ASCEND) is a Randomized, Double-Blind, Placebo Controlled, Phase 3 Study of the Efficacy and Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis. The study objectives are to confirm the treatment effect of pirfenidone compared with placebo on change in percent predicted forced vital capacity (%FVC) in patients with idiopathic pulmonary fibrosis (IPF), and to confirm the safety of treatment with pirfenidone compared with placebo in patients with IPF.
Interventions
DRUGPirfenidone
Pirfenidone, total daily dose of 2403 mg/ day, given as 3 divided doses 3 times per day.
DRUGPlacebo
Placebo equivalent given as 3 divided doses 3 times per day.
Sponsors
Genentech, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
40 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
Select Inclusion Criteria: 1. Diagnosis of idiopathic pulmonary fibrosis (IPF), consistent with the ATS 2011 Guidelines, of 6-48 months' duration 2. Age 40 to 80 at randomization 3. Percent Forced Vital Capacity (%FVC) ≥50% and ≤90% at screening 4. Percent Carbon Monoxide Diffusing Capacity (%DLCO) ≥30% and ≤90% at screening Select
Exclusion criteria
1. Forced expiratory volume in one second (FEV1)/FVC ratio \<0.8 after administration of bronchodilator at Screening 2. Expected to receive a lung transplant within 1 year from randomization or, for patients at sites in the United States, on a lung transplant waiting list at randomization 3. Known explanation for interstitial lung disease 4. History of asthma or chronic obstructive pulmonary disease 5. Active infection 6. Ongoing IPF treatments including investigational therapy, immunosuppressants, and cytokine modulating agents 7. History of unstable or deteriorating cardiac or pulmonary disease (other than IPF) within the previous 6 months
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in Percent Predicted Forced Vital Capacity (%FVC) From Baseline to Week 52 | 52 weeks |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Active Arm Pirfenidone: Pirfenidone, total daily dose of 2403 mg/ day, given as 3 divided doses 3 times per day. | 278 |
| Placebo Arm Placebo: Placebo equivalent given as 3 divided doses 3 times per day. | 277 |
| Total | 555 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 6 | 7 |
| Overall Study | Death | 12 | 19 |
| Overall Study | Lost to Follow-up | 2 | 1 |
| Overall Study | Lung transplantation | 6 | 1 |
| Overall Study | Physician Decision | 1 | 0 |
| Overall Study | Sponsors decision | 0 | 1 |
| Overall Study | Withdrawal by Subject | 4 | 4 |
| Overall Study | Withdrew consent | 4 | 3 |
Baseline characteristics
| Characteristic | Active Arm | Placebo Arm | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 205 Participants | 189 Participants | 394 Participants |
| Age, Categorical Between 18 and 65 years | 73 Participants | 88 Participants | 161 Participants |
| Region of Enrollment Australia | 33 participants | 31 participants | 64 participants |
| Region of Enrollment Brazil | 15 participants | 16 participants | 31 participants |
| Region of Enrollment Croatia | 1 participants | 1 participants | 2 participants |
| Region of Enrollment Israel | 11 participants | 9 participants | 20 participants |
| Region of Enrollment Mexico | 12 participants | 5 participants | 17 participants |
| Region of Enrollment New Zealand | 1 participants | 3 participants | 4 participants |
| Region of Enrollment Peru | 18 participants | 26 participants | 44 participants |
| Region of Enrollment Singapore | 0 participants | 2 participants | 2 participants |
| Region of Enrollment United States | 187 participants | 184 participants | 371 participants |
| Sex: Female, Male Female | 56 Participants | 64 Participants | 120 Participants |
| Sex: Female, Male Male | 222 Participants | 213 Participants | 435 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 271 / 278 | 253 / 277 |
| serious Total, serious adverse events | 55 / 278 | 69 / 277 |
Outcome results
Primary
Change in Percent Predicted Forced Vital Capacity (%FVC) From Baseline to Week 52
Time frame: 52 weeks
Population: Intent to Treat all randomized Patient
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Active Arm | Change in Percent Predicted Forced Vital Capacity (%FVC) From Baseline to Week 52 | Decline or >=10% or Death | 16.5 percentage of patients |
| Active Arm | Change in Percent Predicted Forced Vital Capacity (%FVC) From Baseline to Week 52 | No Decline (Change >0%) | 22.7 percentage of patients |
| Placebo Arm | Change in Percent Predicted Forced Vital Capacity (%FVC) From Baseline to Week 52 | Decline or >=10% or Death | 31.8 percentage of patients |
| Placebo Arm | Change in Percent Predicted Forced Vital Capacity (%FVC) From Baseline to Week 52 | No Decline (Change >0%) | 9.7 percentage of patients |