Phase II Open-Label, Randomized-Controlled Trial of CSF-1R Inhibitor, Axatilimab, for Prevention of Acute and Chronic GVHD After HLA-Matched Related and Unrelated Donor Allogeneic Stem Cell Transplantation

Summary

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.

Description

Eligibility

Age range

18+ years

Sex

All

Healthy volunteers

No

Interventions

• Procedure
  Hematopoietic Cell Transplantation

  Subjects will receive a first HLA-matched related or (7/8 or 8/8) unrelated donor myeloablative allogeneic HCT.

• Drug
  Cyclophosphamide

  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.

• Drug
  Tacrolimus

  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.

• Drug
  Mycophenolate Mofetil (MMF)

  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).

• Drug
  Axatilimab

  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).

Location