A Phase 3 Double-Blind Study of Axatilimab and Corticosteroids for Chronic Graft-Versus-Host Disease
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft Versus-Host Disease
IRAS ID
1010624
Contact name
Carol Penning
Contact email
Sponsor organisation
Incyte Corporation
Research summary
This is a Phase III study comparing the effect of a study drug called Axatilimab and corticosteroids with placebo and corticosteroids. Patients involved in this study have been diagnosed with either moderate or severe chronic Graft Versus Host Disease (cGVHD). The study design includes patients being randomly assigned to one of two treatment groups: one with Axatilimab and corticosteroids, or one with placebo and corticosteroids. Neither the patient nor their investigative doctor will know which group the patient is allocated to.
This study will enrol approximately 240 patients, 120 patients per treatment group. It is estimated that patients may participate for as long as 37 months, including the following:
• Up to 28 days for Screening
• Continuous treatment for a maximum of 24 months
• Safety follow-up period 30 days (±7 days) after the last dose of Axatilimab/placebo
• Post-Treatment cGVHD for qualifying participants, up to 24 months from first day of treatment or until the start of a new therapy or death, whichever occurs first
• Participants will be followed up for survival until death, withdrawal of consent or end of study, whichever occurs first, up to a maximum of 36 months from first day of treatment
Chronic graft-versus-host disease is a severe complication of allo-SCT that affects various organs, leading to a reduced quality of life. Standard initial treatment of moderate and severe cGVHD includes systemic corticosteroids at a prednisone dose ranging between 0.5 and 1 mg/kg. Treatment of cGVHD with systemic corticosteroids is often ineffective, with frequent incomplete responses and recurrences. Approximately 50% to 60% of patients with cGVHD will require a second-line treatment within 2 years. The goal of this study is to increase improve the quality of life of patients by evaluating a new treatment regimen which can hopefully reduce the need for a second therapy and minimize the adverse events from corticosteroids.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
24/EM/0244
Date of REC Opinion
15 Jan 2025
REC opinion
Further Information Favourable Opinion