A Study Evaluating the Efficacy and Safety of Vixarelimab in Participants With Idiopathic Pulmonary Fibrosis and in Participants With Systemic Sclerosis-Associated Interstitial Lung Disease
- Summary
- The main purpose of the study is to evaluate the efficacy of vixarelimab compared with placebo on lung function in participants with idiopathic pulmonary fibrosis (IPF) and in participants with systemic sclerosis-associated interstitial lung disease (SSc-ILD). Participants who complete 52-weeks of treatment in the Double-blind Treatment (DBT) period can choose to enroll in the optional Open-label Extension (OLE) period to receive treatment with vixarelimab for another 52 weeks. Cohort 1 has completed enrollment and has been closed for further enrollment. Cohort 2 is enrolling participants.
- Short title
- A Study Evaluating the Efficacy and Safety of Vixarelimab in Participants With Idiopathic Pulmonary Fibrosis and in Participants With Systemic Sclerosis-Associated Interstitial Lung Disease
- Official title
- A Two-Cohort, Phase II, Multicenter, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Study Evaluating the Efficacy and Safety of Vixarelimab Compared With Placebo in Patients With Idiopathic Pulmonary Fibrosis and in Patients With Systemic Sclerosis-Associated Interstitial Lung Disease
- Control arm
- Placebo
- Registry reports results
- Listing at source
- Listed
- Recruitment status
- Terminated
- Phase
- Phase 2
- Study type
- Interventional
- Reason stopped
- Sponsor decided to discontinue the study after review of clinical data by the iDMC. This decision was based on unblinded evaluation of clinical efficacy which showed a trend towards futility \& was not related to any safety concerns of vixarelimab.
- Completion date
- 2026-01-12
- First posted
- 2023-03-27
- Registry last updated
- 2026-02-09
- Primary completion date
- 2026-01-12
- Start date
- 2023-05-26
- Actual participants
- Participants
- Participant count basis
- Actual
- Protocol amendments
- Number of sites
- Data monitoring committee
- Registry sponsor category
- Industry
- Lead sponsor
- Genentech, Inc.
- Source registry
- ClinicalTrials.gov
- Registry ID
- NCT05785624
- ClinicalTrials.gov ID
- NCT05785624
- Registry record
- Open registry record
- Allocation
- Randomised
- Model
- Parallel groups
- Purpose
- Treatment
- Masking
- Quadruple masking
- Masked roles
- Participants, Care providers, Investigators, Outcome assessors
Cohorts 1 and 2: Absolute Change From Baseline in Forced Vital Capacity (FVC)
Timeframe: Baseline up to Week 52
FVC is a pulmonary function test parameter that indicates the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It's measured by spirometry, which is a common breathing test to check lung function.
Cohorts 1 and 2: Absolute Change From Baseline in 6-Minute Walk Test (6MWT) Distance
Timeframe: Baseline up to Week 52
The 6MWT test is performed indoors on a flat, straight corridor with a hard surface at least 30 meters (m) in length. 6MWT measures the distance a participant is able to walk quickly on a flat, hard surface in a period of 6 minutes. 6MWT measure will be calculated as the simple difference between baseline distance walked over 6 minutes and week 52 distance walked over 6 minutes as measured in meters.
Cohorts 1 and 2: Absolute Change From Baseline in Percentage of Predicted FVC
Timeframe: Baseline up to Week 52
FVC is a pulmonary function test parameter that indicates the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It's measured by spirometry, which is a common breathing test to check lung function.
Cohorts 1 and 2: Change From Baseline in Diffusion Capacity of the Lung for Carbon Monoxide Adjusted for Hemoglobin (DLco [Hb])
Timeframe: Baseline up to Week 52
DLCO measures the ability of the lungs to transfer gas from inhaled air to the red blood cells in the blood. DLCO is adjusted for hemoglobin as small changes in hemoglobin concentration can affect the carbon monoxide transfer.
Cohorts 1 and 2: Time to Disease Progression
Timeframe: From the start of study treatment until disease progression or death, whichever occurs first (up to Week 52 of DBT)
Time to disease progression is defined as time to first occurrence of ≥10% absolute decline in percentage of predicted FVC, ≥15% relative decline in 6MWT distance, lung transplantation, or death. FVC=pulmonary function test parameter that indicates the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It's measured by spirometry, which is a common breathing test to check lung function. 6MWT test is performed indoors on a flat, straight corridor with a hard surface at least 30 m in length. 6MWT measures the distance a participant is able to walk quickly on a flat, hard surface in a period of 6 minutes. 6MWT measure will be calculated as the simple difference between baseline distance walked over 6 minutes and week 52 distance walked over 6 minutes as measured in meters.
Cohorts 1 and 2: Time to First Acute Exacerbation of ILD, or Suspected Acute Exacerbation of ILD
Timeframe: From the start of study treatment until end of DBT (up to Week 52)
Cohorts 1 and 2: Change From Baseline in Quantitative Lung Fibrosis on High-Resolution Computed Tomography (HRCT) Scan of the Thorax
Timeframe: Baseline up to Week 52
High-resolution computer tomography (HRCT) is a type of computed tomography (CT) with specific techniques to enhance image resolution. It is used in the diagnosis of various health problems, most commonly for lung disease. These images show cross sections (slices) through the lungs.
Cohorts 1 and 2: Percentage of Participants with Deaths
Timeframe: Up to Week 52
Cohort 2: Change From Baseline in Skin Sclerosis Assessed Using Modified Rodnan Skin Score (mRSS)
Timeframe: Baseline up to Week 52
mRSS is a measure of skin thickness. Skin thickness will be assessed by the investigator by palpation across 17 different body sites and scored on a scale of 0 (normal) to 3 (severe skin thickening). The total score is the sum of the individual skin scores from all of these sites and ranges from 0 (normal) to 51 (severe thickening in all 17 areas) units. Higher scores indicate disease worsening.
Cohorts 1 and 2: Change From Baseline in Health-Related Quality of Life (HRQoL) Measured Using King's Brief Interstitial Lung Disease (K-BILD) Questionnaire
Timeframe: Baseline up to Week 52
K-BILD is a questionnaire that assesses HRQoL in ILDs. It consists of 15 items grouped into psychological, breathlessness and activity, and chest symptom domains, each scored individually on a 7-point scale, with domain-level and total scores transformed 0-100, with higher scores indicating better quality of life. It uses a 2-week recall period.
Cohorts 1 and 2: Change From Baseline in Cough Measured Using Living with Pulmonary Fibrosis (L-PF) Symptoms Cough Domain Score
Timeframe: Baseline up to Week 52
The L-PF symptoms module is a 23-item tool with domains capturing shortness of breath, cough, and energy symptoms using a 0-4 numeric response scale (NRS) response format and a 24-hour recall period. The cough scores ranges from 0 to 100 with higher scores indicating greater symptom burden/impairment.
Cohorts 1 and 2: Change From Baseline in Dyspnea Measured Using L-PF Symptoms Dyspnea Domain Score
Timeframe: Baseline up to Week 52
The L-PF symptoms module is a 23-item tool with domains capturing shortness of breath, cough, and energy symptoms using a 0-4 NRS response format and a 24-hour recall period. L-PF Symptoms Dyspnea Domain score (dyspnea score) ranges from 0 to 100, with higher score indicating greater impairment.
Cohort 2: Change From Baseline in Pruritus Measured Using the Five-Dimension Itch Scale (5-D Itch) Total Score
Timeframe: Baseline up to Week 52
The 5-D-Itch questionnaire that measures itch and its impact. It consists of 8 items organized into 5 domains (duration, degree, direction, disability, and distribution). Each domain is scored 1 to 5 with a total score ranging from 5 to 25 with higher scores indicating greater itch severity.
Cohorts 1, 2 and OLE Period: Number of Participants With Adverse Events (AEs)
Timeframe: Up to Week 52
Cohorts 1 and 2: Serum Concentration of Vixarelimab
Timeframe: Baseline up to Week 52
Cohorts 1 and 2: Number of Participants With Anti-Drug Antibodies (ADAs) to Vixarelimab
Timeframe: Baseline up to Week 52
- Sex
- All
- Age groups
- Adults, Older adults
- Minimum age
- 18 Years
- Maximum age
- 85 Years
- Healthy volunteers
- No
Inclusion Criteria for all Participants:
- FVC ≥45% predicted during screening as determined by the over-reader
- Forced expiratory volume in 1 second (FEV1)/FVC ratio >0.70 during screening as determined by the over-reader
- DLco ≥30% and ≤90% of predicted during screening (Hgb corrected) as determined by the over-reader
- Minimum 6-MWT distance of 150 m with maximum use of 6 liters per minute (L/min) at sea-level and up to 8 L/min at altitude (> 5000 feet [1524 m] above sea level) of supplemental oxygen while maintaining oxygen saturation of >83% during the 6MWT during screening
- Participant and investigator consideration of all medicinal treatment options and/or possibly lung transplantation prior to consideration of participation in the study
Inclusion Criteria for Cohort 1:
- Age 40-85 years
- Documented diagnosis of IPF or IPF (likely)
- HRCT pattern consistent with the diagnosis of IPF, confirmed by central review of chest HRCT and central review of any available lung biopsy
- For participants receiving pirfenidone or nintedanib treatment for IPF: treatment for ≥3 months with a stable dose for ≥4 weeks prior to screening and during screening, with plans to continue treatment during the study period
Inclusion Criteria for Cohort 2:
- Age 18-85 years
- Diagnosis of SSc as defined using the American College of Rheumatology/European Alliance of Associations for Rheumatology (EULAR) criteria
- HRCT demonstrating ≥10% extent of fibrosis, confirmed by central review of Chest HRCT
- Evidence of progression of pulmonary fibrosis
- For participants receiving tocilizumab treatment for SSc-ILD: treatment for ≥3 months with a stable dose for ≥4 weeks prior to screening and during screening, with no contraindications according to local prescribing information, and no intention to change or modify their treatment regimen for the duration of the study
- For participants receiving nintedanib treatment for SSc-ILD: treatment for ≥ 3 months with a stable dose for ≥ 4 weeks prior to screening and during screening, with no contraindications according to local prescribing information, and no intention to change or modify their treatment regimen for the duration of the study
- Availability of skin for biopsy preferably on proximal forearms having Modified Rodnan Skin Score (mRSS) ≥2 at the biopsy location
Inclusion Criteria for OLE Period:
- Completion of 52 weeks of treatment in the double-blinded treatment period
- Investigator determination of a favorable benefit-risk for the individual participant, i.e., the expectation of reasonable likelihood for therapeutic benefit and tolerability of the study drug after evaluation of the preceding 52 weeks of double-blinded treatment
Exclusion Criteria for all Participants:
- Percentage of predicted FVC value showing improvement in the 6-month period prior to screening and including screening value
- Known post-bronchodilator response in FEV1 and/or FVC (defined as an increase in percent predicted values by ≥ 10)
- Resting oxygen saturation of <89% using up to 4 L/min of supplemental oxygen at sea level and up to 6 L/min at altitude (5000 feet [1524 m] above sea level) during screening
- History of lung transplant
- Previous treatment with vixarelimab
- Acute respiratory or systemic bacterial, viral, or fungal infection either during screening or prior to screening not successfully resolved by 4 weeks prior to screening visit
- Presence of pulmonary hypertension requiring treatment
- History of malignancy within the 5 years prior to screening
- Positive hepatitis C virus (HCV) antibody test result accompanied by a positive HCV ribonucleic acid (RNA) test at screening
- Known immunodeficiency
- Known evidence of active or untreated latent tuberculosis
Exclusion Criteria for Cohort 1:
- Evidence of other known causes of ILD
- Emphysema present on ≥50% of the HRCT, or the extent of emphysema is greater than the extent of fibrosis, according to central review of the HRCT
Exclusion Criteria for Cohort 2:
- Evidence of other known causes of ILD
- Rheumatic autoimmune disease other than SSc
- Receiving pirfenidone treatment within 4 weeks prior to screening
- Receipt of nintedanib in combination with tocilizumab
Exclusion Criteria for OLE Period:
- Significant non-compliance in the double-blinded treatment period, per investigator's judgment
- Any new clinically significant pulmonary disease other than IPF or SSc-ILD since enrolling in the double-blinded treatment period
- University of Alabama at BirminghamBirmingham, United States
- Banner University Medicine Lung InstitutePhoenix, United States
- Southern Arizona VA Health Care System NAVREF PPDSTucson, United States
- University of California, San Francisco-FresnoFresno, United States
- University of Southern California Keck School of MedicineLos Angeles, United States
- UCLA RheumatologyLos Angeles, United States
- University of California, San Francisco Medical CenterSan Francisco, United States
- The Lundquist Institute for BioMedical Innovation at Harbor-UCLA Medical CenteTorrance, United States
- Renstar Medical ResearchOcala, United States
- Coastal Pulmonary and Critical Care PLCSt. Petersburg, United States
- Weston HospitalWeston, United States
- Piedmont Pulmonary and Sleep Medicine BuckheadAtlanta, United States
- Emory ClinicAtlanta, United States
- GenHarp Clinical SolutionsEvergreen Park, United States
- Northwestern Medicine - Northwestern Medicine GlenGlenview, United States
- IU Health Ball Memorial Physicians Pulmonary and Critical Care MedicineMuncie, United States
- University of Kansas Medical CenterKansas City, United States
- University of Michigan HospitalAnn Arbor, United States
- Hannibal Regional Healthcare System HRMG HannibalHannibal, United States
- Pulmonix LLCGreensboro, United States
- Southeastern Research CenterWinston-Salem, United States
- The University of Oklahoma Health Sciences CenterOklahoma City, United States
- Baylor Scott and White Health - Advanced Lung Disease Specialists - DallasDallas, United States
- IntraCare Health Center ? PrestigeDallas, United States
- El Paso Pulmonary Association Elligo PPDSEl Paso, United States
And 89 more, listed on the interactive page.
- 2022-502828-42-00
- 2022-502828-42
Related records
Drugs (1)
Lead sponsors (1)
Conditions (1)
- Idiopathic pulmonary fibrosis Primary condition