NCT07124078

A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)

A Phase 2, Randomized, Open-Label Study of Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Incyte Corporation · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.

Conditions

• Chronic Graft-versus-host-disease

Interventions

Type	Name	Description
DRUG	INCA034176	Axatilimab at the protocol-defined dose.
DRUG	Best available Treatment (BAT)	Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.

Timeline

• Start date: 2026-05-31
• Primary completion: 2029-01-31
• Completion: 2029-07-31
• First posted: 2025-08-15
• Last updated: 2026-04-14

Locations

42 sites across 6 countries: United States, Belgium, Germany, Italy, Spain, United Kingdom

Regulatory

• FDA-regulated drug study