A Phase 1/2 Trial of Donor Regulatory T-cells for Steroid-Refractory Chronic Graft-versus-Host-Disease

Phase 1/2 Unknown 22 enrolled Gulbenkian Institute for Molecular Medicine Hospital de Santa Maria, Portugal · collab IPOFG Lisboa, Portugal · collab IPOFG Porto, Portugal · collab

Non-randomizedSingle-groupOpen-labelTreatment

Summary

Phase 1/2 clinical study for the treatment of steroid-refractory chronic graft versus host disease after an allogeneic transplant of hematopoietic progenitors with donor CliniMACS-selected regulatory T cells

Timeline

Start: 2015-03
Primary completion: 2019-03
Completion: 2019-12

Outcome

Results reported · no primary-endpoint met/missed verdict (typical of a dose-finding / safety study)

paper Although these studies were not powered to assess efficacy PMID 41637631 ↗

Publications

Soares MVD, Gomez VE, Azevedo RI, Pereira PNG, Velazquez TC, Mendes L, Alho AC, Garcia-Guerrero E, Garcia-Calderon CB, Tharmaratnam K, Cabral IA, Ribeiro AC, Juncal C, Roncon S, Pais AT, Gil AR, Espada E, Rodrigues A, Garcao A, Yaspo ML, Warnatz HJ, Lehrach H, Ward L, Barbosa-Morais NL, Quintas AM, Palmela P, Caldas C, Ferreira R, Leite L, Martins C, Lourenco F, Moreno R, Campilho F, Cheyne CP, Garcia-Finana M, Campos A, Baron F, Arpinati M, Hoffmann P, Edinger M, Koreth J, Ritz J, Vaz CP, Perez-Simon JA, Lacerda JF. Phase 1/2 trials of donor regulatory T cells for the treatment of steroid-refractory chronic graft-versus-host disease. Blood Adv. 2026 May 12;10(9):2904-2915. doi: 10.1182/bloodadvances.2025017996.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	donor regulatory T cells	Cell therapy	500000 cells/kg	—
Subject	donor regulatory T cells	Cell therapy	1e+06 cells/kg	—
Subject	donor regulatory T cells	Cell therapy	2e+06 cells/kg	—
Subject	donor regulatory T cells	Cell therapy	3e+06 cells/kg	—