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

ConditionChronic Graft Versus Host Disease
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

This trial does not accept healthy volunteers

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

Design model

Single group

Treatments tested in this trial

  • Gecacitinib Hydrochloride Tablets

    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.

Treatment groups

24 Participants
are divided into 1 treatment group
Group A: GecacitinibExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Overall 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

Secondary outcomes

1

ORR at Week 12, 48 and 60

Time frame
week 12, 48 and 60
2

Failure-free Survival (FFS)

Time frame
Up to 24 months
3

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
4

Best Overall Response (BOR)

Time frame
Up to week 24

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Yujun DONG

Lead sponsor

Peking University First Hospital

Sponsor institution

Source ClinicalTrials.gov
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