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

NCT ID: NCT07012304

Last Updated: 2025-06-10

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-06-30
• Study Completion Date: 2029-02-28

Brief Summary

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.

Conditions

Chronic Graft Versus Host Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Gecacitinib

Group Type: EXPERIMENTAL

Gecacitinib Hydrochloride Tablets

Intervention Type: DRUG

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.

Interventions

Gecacitinib Hydrochloride Tablets

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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
• Received 2-5 prior systemic cGVHD therapies with persistent disease
• ECOG PS score of 0-2
• Able to swallow tablets
• Concomitant use of non-interacting immunosuppressants permitted

Exclusion Criteria

• 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
• Significant abnormalities affecting safety assessment, such as uncontrolled hypertension (SBP ≥160 mmHg or DBP ≥100 mmHg) despite ≤2 antihypertensives; ALT/AST >3×ULN; DBIL/TBIL >1.5×ULN; serum creatinine >1.5×ULN
• History of major cardiovascular events within 6 months.
• Arrhythmia requiring treatment at screening
• Gastrointestinal conditions impairing drug absorption
• Surgery within 4 weeks of screening with incomplete recovery
• Active/uncontrolled infections (viral, bacterial, parasitic, fungal) requiring treatment
• Active tuberculosis within 6 months
• Epilepsy or use of psychotropic/sedative drugs
• Pregnant/breastfeeding or unwilling to use contraception during and 4 weeks post-study
• Malignancy within 5 years (except the indication for transplant)
• Use of anticoagulants/platelet inhibitors (except low-molecular-weight heparin)
• Herbal medicine use within 1 week prior to enrollment
• Hypersensitivity to gecacitinib or its components
• Participation in another clinical trial within 4 weeks (or 5 half-lives of the previous study drug, whichever is longer)
• Deemed unsuitable by the investigator

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Yujun DONG

OTHER

Sponsor Role: lead

Responsible Party

Yujun DONG

Director of the Hematology Department

Responsibility Role: SPONSOR_INVESTIGATOR

Locations

Peking University First Hospital

Beijing, , China

Site Status: RECRUITING

Countries

China

Facility Contacts

Yujun Dong

Role: primary

dongy@hsc.pku.edu.cn

0086010-83572211

Other Identifiers

cGVHD-06

Identifier Type: -

Identifier Source: org_study_id