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

NCT ID: NCT03287414

Last Updated: 2024-06-18

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-20

Study Completion Date

2022-02-14

Brief Summary

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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).

Detailed Description

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This was an exploratory (non-confirmatory) randomized, patient-, investigator-, sponsor- blinded, placebo controlled study of VAY736 in IPF patients. This study investigated the safety and efficacy of 300 mg VAY736 administered subcutaneously (s.c.) every 4 weeks for 48 weeks.

Participants were randomized in a 1:1 ratio on top of local standard of care (SOC), to receive VAY736 or placebo. Randomized subjects entered the treatment epoch (for up to 48 weeks), followed by two follow-up epochs: the PK/safety follow-up epoch and the PD/safety follow-up epoch. The PK/safety follow-up epoch lasted for 20 weeks. When the PK/safety follow-up epoch was completed, participants in the placebo arm were discharged from the study; but participants in the active arm (those who had received VAY736) continued into the PD/safety follow-up epoch. Participants in the PD/safety follow-up epoch were followed until B-cell recovery (in the peripheral blood), defined as: B cells \>=50/μL or B cells \>= 80% of baseline (whichever occurred first). If a participant had not recovered his/her B-cells after a period of 2 years from the last dose of VAY736, then this participant was discharged from the study.

Conditions

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Idiopathic Pulmonary Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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VAY736

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

Group Type EXPERIMENTAL

VAY736

Intervention Type DRUG

300 mg VAY736 administered subcutaneously every 4 weeks for 48 weeks

Standard of Care (SoC)

Intervention Type DRUG

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

Placebo

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo administered subcutaneously every 4 weeks for 48 weeks

Standard of Care (SoC)

Intervention Type DRUG

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

Interventions

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VAY736

300 mg VAY736 administered subcutaneously every 4 weeks for 48 weeks

Intervention Type DRUG

Placebo

Placebo administered subcutaneously every 4 weeks for 48 weeks

Intervention Type DRUG

Standard of Care (SoC)

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

Intervention Type DRUG

Other Intervention Names

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Ianalumab

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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)
Minimum Eligible Age

40 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Birmingham, Alabama, United States

Site Status

Novartis Investigative Site

Aurora, Colorado, United States

Site Status

Novartis Investigative Site

Miami, Florida, United States

Site Status

Novartis Investigative Site

Durham, North Carolina, United States

Site Status

Novartis Investigative Site

Pittsburgh, Pennsylvania, United States

Site Status

Novartis Investigative Site

Nashville, Tennessee, United States

Site Status

Novartis Investigative Site

Salt Lake City, Utah, United States

Site Status

Novartis Investigative Site

Calgary, Alberta, Canada

Site Status

Novartis Investigative Site

Coswig, , Germany

Site Status

Novartis Investigative Site

Hanover, , Germany

Site Status

Novartis Investigative Site

Dublin, , Ireland

Site Status

Novartis Investigative Site

Forlì, FC, Italy

Site Status

Novartis Investigative Site

Modena, MO, Italy

Site Status

Novartis Investigative Site

Siena, SI, Italy

Site Status

Novartis Investigative Site

Cambridge, Cambridgeshire, United Kingdom

Site Status

Novartis Investigative Site

High Heaton, Newcastle Upon Tyne, United Kingdom

Site Status

Countries

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United States Canada Germany Ireland Italy United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2017 002667 17

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CVAY736X2207

Identifier Type: -

Identifier Source: org_study_id

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