AGAVE-201- Phase 2 Study to evaluate Axatilimab in cGVHD
Research type
Research Study
Full title
AGAVE-201, A Phase 2, Open-label, Randomized, Multicenter Study to Evaluate the Efficacy, Safety and Tolerability of Axatilimab at 3 Different Doses in Patients with Recurrent or Refractory Active Chronic Graft Versus Host Disease who have Received at least 2 Lines of Systemic Therapy
IRAS ID
292892
Contact name
Emma Nicholson
Contact email
Sponsor organisation
Syndax Pharmaceuticals, Inc.
Eudract number
2020-005107-40
Duration of Study in the UK
1 years, 10 months, 25 days
Research summary
Chronic Graft Versus Host Disease (cGVHD) remains the major cause of morbidity and non-relapse mortality after allogeneic hematopoietic stem cell transplantation (HSCT). cGVHD typically manifests with multiorgan pathology, which often occurs during the first year post-HSCT but can also develop beyond the first year post-HSCT.
Treatment of cGVHD is currently based on steroid administration and although many other approaches are commonly used, none have proven clearly effective. Targeting pathogenic monocyte derived macrophages by preventing their differentiation and survival through the inhibition of colony stimulating factor 1 receptor (CSF-1R) has proven highly effective in animal systems.
The AGAVE-201 study is to evaluate the efficacy, safety and tolerability of axatilimab at 3 different dose levels, in patients with recurrent or refractory active cGVHD who have received at least 2 prior lines of systemic therapy due to progression of disease, intolerability or toxicity.
Axatilimab is a humanised IgG4 monoclonal antibody (mAb) directed against CSF-1R with the potential to treat cGVHD through blockade of macrophage activity.
The study will consist of 3 periods; Screening, Treatment, and Follow-up.
Patients will be randomised to one of 3 dose cohorts (agatolimod 0.3 mg/kg every 2 weeks [Q2W], 1 mg/kg Q2W, and 3 mg/kg Q4W). Patients must begin treatment (Cycle 1 Day 1) within 3 days of randomization/enrolment and will receive axatilimab from Cycle 1 Day 1, in 4-week (28-day) treatment cycles, until disease progression (as defined by the NIH 2014 consensus criteria), withdrawal of consent or unacceptable toxicity. Following treatment discontinuation, patients will receive an End-of-treatment (EOT) visit 30 days after the last dose of study drug and 2 further safety and disease evaluation visits at 60 and 90 days post last dose of study drug.
Once the study is over, participants will return to their normal standard of care.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
21/EM/0123
Date of REC Opinion
14 Jul 2021
REC opinion
Further Information Favourable Opinion