PTCy and Ruxolitinib vs PTCy, Tacrolimus and MMF in MUD and Haploidentical HSCT

Summary

This is multicenter investigator-initiated randomized open-label phase II clinical trial to compare prophylaxis of graft versus host disease treated with tacrolimus and mycophenolate mofetil versus ruxolitinib after post-transplant cyclophosphamide. In total 128 patients will be included in the study. After inclusion into the study and performing of transplantation patients will be randomized in 1:1 proportion in two arms (64 patients per arm): arm A will include patients who will be treated with cyclophosphamide and ruxolitinib for GVHD prophylaxis; arm B will include patients who will be treated with cyclophosphamide, tacrolimus and MMF for GVHD prophylaxis. After the end of the treatment patients will be followed-up during two years.

Timeline

Start

2020-11-03

Primary completion

2023-02-27

Completion

2025-03-28

Outcome

Met primary endpoint

paper The study met its primary endpoint of noninferior efficacy of PTCy-Ruxo GVHD prophylaxis. PMID 42066960 ↗

paper noninferiority P = .0041 PMID 42066960 ↗

Publications

• Moiseev I, Drokov M, Kitaeva Y, Rudakova T, Barabanshchikova M, Chernyshova D, Bondarenko S, Kuzmina L, Rodionov G, Svetkina E, Lapin S, Kholopova I, Morozova E, Vasilyeva V, Vlasova Y, Zhogolev D, Konstantinova T, Parovichnikova E, Kulagin A. A randomized, multicenter phase II trial to compare prophylaxis of graft versus host disease with tacrolimus and mycophenolate mofetil versus ruxolitinib after post-transplant cyclophosphamide. Transplant Cell Ther. 2026 Apr 30:S2666-6367(26)00333-7. doi: 10.1016/j.jtct.2026.04.042. Online ahead of print.

Drugs

Evaluation	Drug	Modality	Dose	Route
Comparator	Mycophenolate Mofetil	Small molecule	30 mg/kg	Oral
Comparator	Mycophenolate Mofetil	Small molecule	45 mg/kg	Oral
Subject	Ruxolitinib	Small molecule	5 mg	Oral
Comparator	Tacrolimus	Small molecule	0.03 mg/kg	—

Indications