NCT04710576

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)

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

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 241 (actual)
• Sponsor: Syndax Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received at least 2 prior lines of systemic therapy.

Detailed description

AGAVE-201 is a Phase 2, open-label, randomized, multicenter study to evaluate the efficacy, safety, and tolerability of axatilimab in participants with recurrent or refractory active cGVHD after failure of at least 2 prior lines of systemic therapy due to progression of disease, intolerability, or toxicity. Participants will be randomized to receive 1 of 3 different axatilimab treatment regimens in 28-day treatment cycles for up to 2 years.

Conditions

• Chronic Graft-versus-host-disease

Interventions

Type	Name	Description
DRUG	Axatilimab	Axatilimab is a high-affinity antibody targeting the colony stimulating factor 1 receptor (CSF-1R). CSF-1R signaling has been demonstrated in nonclinical studies to be the key regulatory pathway involved in the expansion and infiltration of donor-derived macrophages that mediate the disease processes involved in cGVHD.

Timeline

• Start date: 2021-03-04
• Primary completion: 2023-04-07
• Completion: 2027-09-01
• First posted: 2021-01-14
• Last updated: 2026-03-16
• Results posted: 2024-10-10

Locations

121 sites across 16 countries: United States, Australia, Belgium, Canada, France, Germany, Greece, Israel, Italy, Poland, Portugal, Singapore, South Korea, Spain, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study