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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
Gecacitinib hydrochloride tablets are taken orally on an empty stomach. The starting dose is 50 mg once daily (QD). The maximum dose is 100 mg twice daily (BID), and the minimum dose is 50 mg every other day (QOD). Dose adjustments should be made in 50-mg increments or decrements.
Peking University First Hospital
Beijing, China
RECRUITINGOverall Response Rate (ORR) at Week 24
ORR was defined as the proportion of patients achieving complete response (CR) or partial response (PR) without requiring additional systemic therapy for cGVHD.
Time frame: Week 24
ORR at Week 12, 48 and 60
Time frame: week 12, 48 and 60
Failure-free Survival (FFS)
Time frame: Up to 24 months
Change From Baseline in Lee cGVHD Symptom Scale Scores
Subscale scores and the summary score range from 0 to 100, with a higher score indicating worse symptoms.
Time frame: Through study completion, an average of 24 months
Best Overall Response (BOR)
Time frame: Up to week 24
Duration of Response (DOR)
Time frame: Up to 24 months
Percentage of Participants With a ≥50% Reduction in Daily Corticosteroid Dose for ≥4 Weeks
Time frame: Up to Day 180
Relapse Rate
Time frame: Up to 24 months
Overall Survival (OS)
Time frame: Up to 24 months
Change from Baseline in FACT-BMT
The FACT-BMT (Functional Assessment of Cancer Therapy-Bone Marrow Transplant) total score ranges from 0 to 148. Higher scores reflect a better quality of life.
Time frame: Through study completion, an average of 24 months
Change from Baseline in EQ-5D-5L
The EQ-5D-5L (EuroQol 5-Dimension 5-Level Questionnaire) assesses health across five dimensions using five severity levels. It also includes a Visual Analogue Scale (VAS) ranging from 0 (worst imaginable health) to 100 (best imaginable health), where a higher score indicates a better health outcome.
Time frame: Through study completion, an average of 24 months
Adverse Events (AEs) and Adverse Drug Reactions (ADRs)
Time frame: Up to 28 days after the last dose
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