A Study to Evaluate 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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Genentech Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08], Diseases [C] - Immune System Diseases [C20]

Trial duration

1 Sep 2023 → 12 Jan 2026

Decision date (initial)

2023-08-01

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Genentech Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Efficacy

To evaluate the efficacy of vixarelimab compared with placebo on lung function for each cohort

Secondary objectives 6

1. To evaluate the efficacy of vixarelimab compared with placebo for each cohort
2. To evaluate symptoms and quality of life of patients treated with vixarelimab compared with placebo for each cohort
3. To evaluate the safety of vixarelimab compared with placebo for each cohort
4. To evaluate the safety of longer-term treatment with vixarelimab for each cohort
5. To characterize the pharmacokinetics of vixarelimab for each cohort
6. To evaluate the immune response to vixarelimab for each cohort

Conditions and MedDRA coding

Idiopathic Pulmonary Fibrosis

Study design 1 period

Regulatory references

Plan to share IPD

No

IPD plan description

N/A

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Forced vital capacity (FVC) ≥45% predicted during screening as determined by the over-reader
2. FEV1/FVC ratio >0.70 during screening as determined by the over-reader
3. Diffusion capacity of the lung for carbon monoxide (DLCO) ≥30% and ≤90% of predicted during screening (Hgb corrected) as determined by the over-reader
4. Minimum 6-minute walk test (6MWT) distance of 150 meters with maximum use of 6 L/min at sea-level and up to 8 L/min at altitude (>5000 feet [1524 meters] above sea level) of supplemental oxygen while maintaining oxygen saturation of >83% during the 6MWT during screening
5. Cohort 1: Documented diagnosis of idiopathic pulmonary fibrosis (IPF) or IPF (likely) per the 2022 American Thoracic Society (ATS)/ European Respiratory Society (ERS)/ Japanese Respiratory Society (JRS)/ Latin American Thoracic Society (ALAT) Clinical Practice Guideline or high-resolution computed tomography (HRCT) pattern consistent with the diagnosis of IPF
6. Cohort 2: Diagnosis of systemic sclerosis (SSc), as defined using the American College of Rheumatology/European League against Rheumatism (EULAR) criteria

Exclusion criteria 6

1. Percentage of predicted FVC value showing improvement in the 6-month period prior to screening and including screening value, as assessed by the investigator
2. Known post-bronchodilator response in FEV1 and/or FVC (defined as an increase in percent predicted values by ≥ 10)
3. 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 meters] above sea level) during screening
4. History of lung transplant
5. Inability to refrain from use of the following: – Short-acting bronchodilators within 4 hours before pulmonary function test (PFT), DLCO, and 6MWT assessments – Once-daily, long-acting bronchodilators within 24 hours before PFT, DLCO, and 6MWT assessments – Twice-daily, long-acting bronchodilators within 12 hours before PFT, DLCO, and 6MWT assessments
6. Receipt of nintedanib in combination with pirfenidone. Receipt of systemic (oral, IV, IM, or IA) corticosteroids equivalent to prednisone >10 mg/day or equivalent within 2 weeks prior to screening

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 1. Absolute change from baseline to Week 52 in FVC (mL)

Secondary endpoints 17

1. 1. Absolute change from baseline to Week 52 in 6MWT distance (in meters)
2. 2. Absolute change from baseline to Week 52 in percentage of predicted FVC
3. 3. Change from baseline to Week 52 in DLCO [Hb]
4. 4. Time to disease progression, 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
5. 5. Time to first acute exacerbation of ILD, or suspected acute exacerbation of ILD, as determined by the CAC
6. 6. Change from baseline to Week 52 in quantitative lung fibrosis on HRCT scan of the thorax
7. 7. Survival, as measured by all-cause mortality
8. 8. Cohort 2: Change from baseline to Week 52 in skin sclerosis, as measured by the modified Rodnan skin score (mRSS)
9. 9. Change from baseline to Week 52 in health-related quality of life (HRQoL), as measured by the King’s Brief Interstitial Lung Disease (K-BILD) Questionnaire total score
10. 10. Change from baseline to Week 52 in cough, as measured by the Living with Pulmonary Fibrosis (L-PF) Symptoms cough domain score
11. 11. Change from baseline to Week 52 in dyspnea, as measured by the L-PF Symptoms dyspnea domain score
12. 12. Cohort 2: Change from baseline to Week 52 in pruritus, as measured by the 5-D Itch Scale total score
13. 13. Incidence and severity of adverse events, with severity determined according to the Division of AIDS (DAIDS) toxicity grading scale
14. 14. Change from baseline in targeted vital signs
15. 15. Change from baseline in targeted clinical laboratory test results
16. 16. Serum concentration of vixarelimab at specified timepoints
17. 17. Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Genentech Inc.

Sponsor organisation

Genentech Inc.

Address

1 Dna Way

City

South San Francisco

Postcode

94080-4990

Country

United States

Scientific contact point

Organisation

Genentech Inc.

Contact name

US Program Manager Product Development Regulatory

Public contact point

Organisation

Genentech Inc.

Contact name

US Program Manager Product Development Regulatory

Third parties 5

Locations

8 EU/EEA countries · 39 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 145 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

17 submissions — initial application plus substantial / non-substantial modifications