Study of Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of VAY736 in Patients With Idiopathic Pulmonary Fibrosis

NCT03287414 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: CVAY736X22072017 002667 17
• Study Type: interventional
• Target: 30
• Locations: 6 countries
• Sites: 16

Brief Summary

The purpose of this study was to investigate the safety, tolerability and efficacy of VAY736 as potential therapy for the treatment of idiopathic pulmonary fibrosis (IPF).

Conditions

Idiopathic Pulmonary Fibrosis

Keywords

idiopathic pulmonary fibrosis; VAY736

Outcome Measures

Primary Outcomes (1)

• Change From Baseline to End of Treatment Epoch (48 Weeks of Treatment) in Forced Vital Capacity (FVC).

  FVC was defined as the maximum amount of air that an individual was able to forcibly exhale from his / her lungs after taking the deepest breath they could. Change from baseline to end of treatment epoch (48 weeks of treatment) in Forced Vital Capacity (FVC) was analyzed using a Mixed Effects Model for Repeated Measures (MMRM) and considering all assessments collected during the treatment epoch. The model included treatment and visit as fixed effects, standard of care treatment (Nintedanib, Pirfenidone, or no treatment) as factor and baseline value as a covariate, treatment-by-visit and baseline-by-visit as interaction terms. A positive change from baseline indicates improvement. Baseline was defined as the last available assessment pre-dose before or on randomization date.

  From baseline up to 48 weeks post first dose of study treatment

Secondary Outcomes (11)

• Percentage of Participants With All-cause Mortality Events

  Up to 48 weeks post first dose of study treatment

• Percentage of Participants With Survival Idiopathic Pulmonary Fibrosis (IPF) -Related Mortality Events

  Up to 48 weeks post first dose of study treatment

• Percentage of Participants With Progression-free Survival (PFS) Events

  Up to 48 weeks post first dose of study treatment

• Percentage of Participants With Disease Progression Events

  Up to 48 weeks post first dose of study treatment

• Percentage of Participants With Composite Events

  Up to 48 weeks post first dose of study treatment

Study Arms (2)

VAY736

EXPERIMENTAL

Participants received 300 mg VAY736 administered subcutaneously every 4 weeks for 48 weeks on top of current standard-of-care therapy

Drug: VAY736; Drug: Standard of Care (SoC)

Placebo

PLACEBO COMPARATOR

Participants received placebo administered subcutaneously every 4 weeks for 48 weeks on top of current standard-of-care therapy

Drug: Placebo; Drug: Standard of Care (SoC)

Interventions

VAY736 DRUG

300 mg VAY736 administered subcutaneously every 4 weeks for 48 weeks

Also known as: Ianalumab

VAY736

Placebo DRUG

Placebo administered subcutaneously every 4 weeks for 48 weeks

Placebo

Standard of Care (SoC) DRUG

Background standard-of-care treatment for IPF: nintedanib, pirfenidone, or no background therapy

Placebo; VAY736

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A diagnosis of definite or probable IPF within 5 years of the screening visit
• Forced Vital Capacity (FVC) 40-90% predicted (inclusive)
• Diffusing Capacity of the Lungs (DLCO), corrected for hemoglobin, 25-79% predicted (inclusive)
• Forced Expiratory Volume in first second (FEV1)/FVC \>70%
• Unlikely to die from cause other than IPF within the next 3 years, in the opinion of the investigator
• Unlikely to undergo lung transplantation during this trial

You may not qualify if:

• Emphysema \> fibrosis on screening high-resolution computed tomography (must be confirmed by central reader)
• History of major organ, hematopoietic stem cell or bone marrow transplant
• Clinically diagnosed acute exacerbation of idiopathic pulmonary fibrosis (AE-IPF) or other significant clinical worsening within 3 months of randomization
• New York Heart Association (NYHA) class III/IV Congestive Heart Failure (CHF), Ejection Fraction (EF) \<25%
• Current smoker
• Prior use of any B-cell depleting therapy (e.g., rituximab, ofatumumab, or other anti-CD20 mAb, anti-CD40, anti-CD19,anti-CD22 mAb, anti-CD52 mAb, or anti-BAFF mAb)

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (16)

Novartis Investigative Site

Birmingham, Alabama, 35294-0007, United States

Location

Novartis Investigative Site

Aurora, Colorado, 80045, United States

Location

Novartis Investigative Site

Miami, Florida, 33136, United States

Location

Novartis Investigative Site

Durham, North Carolina, 27710, United States

Location

Novartis Investigative Site

Pittsburgh, Pennsylvania, 15213, United States

Location

Novartis Investigative Site

Nashville, Tennessee, 37203, United States

Location

Novartis Investigative Site

Salt Lake City, Utah, 84108, United States

Location

Novartis Investigative Site

Calgary, Alberta, T2N 2T9, Canada

Location

Novartis Investigative Site

Coswig, 01640, Germany

Location

Novartis Investigative Site

Hanover, 30625, Germany

Location

Novartis Investigative Site

Dublin, D04, Ireland

Location

Novartis Investigative Site

Forlì, FC, 47100, Italy

Location

Novartis Investigative Site

Modena, MO, 41124, Italy

Location

Novartis Investigative Site

Siena, SI, 53100, Italy

Location

Novartis Investigative Site

Cambridge, Cambridgeshire, CB23 3RE, United Kingdom

Location

Novartis Investigative Site

High Heaton, Newcastle Upon Tyne, NE7 7DN, United Kingdom

Location

MeSH Terms

Conditions

Idiopathic Pulmonary Fibrosis

Interventions

ianalumab; Standard of Care

Condition Hierarchy (Ancestors)

Pulmonary Fibrosis; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Study director
• Organization: Novartis Pharmaceuticals

novartis.email@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 12, 2017
• First Posted: September 19, 2017
• Study Start: December 20, 2017
• Primary Completion: November 25, 2020
• Study Completion: February 14, 2022
• Last Updated: June 18, 2024
• Results First Posted: March 10, 2023

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will share

Locations

IE (1); CA (1); IT (3); DE (2); US (7); GB (2)