CSF-1R Inhibitor, Axatilimab, for Prevention of Acute and Chronic GVHD After HLA-Matched Related and Unrelated Donor Allogeneic Stem Cell Transplantation

Conditions

• Graft -Versus-host-disease

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Hematopoietic Cell Transplantation — PROCEDURE
  Subjects will receive a first HLA-matched related or (7/8 or 8/8) unrelated donor myeloablative allogeneic HCT.
• Cyclophosphamide — DRUG
  Cyclophosphamide will be given on Day +3 and Day +4 per institutional practices, at a dose of 50 mg/kg IBW. Cyclophosphamide will be given as an IV infusion over 1-2 hours (depending on volume), or according to institutional standards.
• Tacrolimus — DRUG
  Tacrolimus will be given per institutional practices, starting on Day +5. Subsequent dosing will be based on blood levels per institutional guidelines with a suggested range of 5-12 ng/mL.
• Mycophenolate Mofetil (MMF) — DRUG
  Mycophenolate mofetil (MMF) will be given per institutional practices, at a dose of 15 mg/kg three times daily (TID; based upon actual body weight) with the maximum total daily dose not to exceed 3 grams (1g TID, IV or PO). MMF prophylaxis will start Day +5 and continue until Day +35 post-transplant, at which time it should be discontinued (no taper necessary).
• Axatilimab — DRUG
  The dose is dependent on the results of the safety run-in. The starting dose of the safety run-in is 0.3 mg/kg (maximum of 35 mg). If Dose Level 1 has an unacceptable level of toxicity (≥2 patients having significant toxicity), then axatilimab may be de-escalated to 0.2 mg/kg (maximum of 23 mg).

Study Details

This is a randomized, multisite, Phase II study designed to compare the 1-year GVHD-free survival of the control arm (no axatilimab) to the experimental arm (axatilimab) after myeloablative allogeneic HCT.

Key Dates

First listed

Sep 8, 2026

Start date

Nov 30, 2026

Status verified

Sep 2026

Primary completion

Nov 30, 2031

Completion

Nov 30, 2031

Study Design

Enrollment

72 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Safety Run-in
  An early safety analysis will be performed after the first 6 evaluable patients have been accrued to Dose Level 1 of the study and observed for four cycles of axatilimab (Cycles 1-4). Accrual will be temporarily halted while these patients are evaluated. If 2 or more of the first 6 patients experience a significant toxicity, then the protocol may be amended and the dose of axatilimab may be de-escalated to Dose Level -1.
• Experimental: Axatilimab Treatment
  Starts 35 (±5) days from stem cell transplant; Dosing will occur every 14 days for a maximum of 12 cycles.
• Experimental: No Axatilimab Treatment
  Starts 35 (±5) days from stem cell transplant.

Primary Outcome Measure

GVHD-free survival [ Time Frame: 1 Year Post HCT ]

Central Contacts

• Medical College of Wisconsin Cancer Center Clinical Trials Office
  866-680-0505
  [email protected]

Locations (1)

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