Gecacitinib for cGVHD: Safety and Efficacy in Patients After ≥2 Lines of Prior Therapy

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorYujun DONG

About this trial

Chronic Graft-versus-Host Disease (cGVHD) is a common late complication following allogeneic hematopoietic stem cell transplantation and a leading non-relapse cause of death. It is often treatment-refractory, significantly affecting patients' quality of life and prognosis. This study will evaluate the feasibility, safety, and tolerability of gecacitinib, a novel JAK and ACVR1 inhibitor, in 24 patients with moderate-to-severe cGVHD who have undergone two or more prior therapies. Participants will receive gecacitinib hydrochloride tablets for at least 24 weeks. Patients demonstrating disease stability, as assessed by the investigator, may continue treatment with the study drug until week 60, unless intolerability, disease progression, or initiation of new systemic therapy, whichever occurs first.

Eligibility criteria

Qualifiers

Voluntarily Signed informed consent and aged ≥18 years

Undergone nonmyeloablative, myeloablative, or reduced-intensity allo-HSCT using bone marrow, peripheral blood stem cells, or umbilical cord blood from any donor source

Confirmed myeloid and platelet engraftment: ANC >1.0×10⁹/L and platelet count >25×10⁹/L; no hematopoietic growth factors or blood product transfusions within 7 days before screening

Clinically diagnosed moderate-to-severe cGVHD according to the 2014 NIH

Disqualifiers

Recurrence of malignancy or loss of full donor chimerism

Concurrent use of other JAK inhibitors, mesenchymal stem cells, or belumosudil (Eligible if discontinued for >8 weeks post-aGVHD treatment or stopped JAK inhibitors for cGVHD due to side effects.)

Severe pulmonary cGVHD (FEV1 ≤39% or NIH lung symptom score of 3)

Post-transplant lymphoproliferative disease

Trial design

Treatments tested in this trial

  • Gecacitinib Hydrochloride Tablets

Treatment groups

24 Participants
are divided into 1 treatment group

Sponsors and collaborators

Yujun DONG

Lead sponsor

Peking University First Hospital

Sponsor institution