Gecacitinib treatment for moderate-to-severe chronic GVHD after two or more prior therapies

A Study on the Safety and Efficacy of Gecacitinib in Patients With Chronic Graft-versus-Host Disease (cGVHD) Who Have Received Prior Treatment With Two or More Systemic Therapies

PHASE1; PHASE2 · Peking University First Hospital · NCT07012304

This trial will try oral gecacitinib in adults with moderate-to-severe chronic GVHD who have had at least two prior systemic treatments to see if it is safe and helps control disease.

Quick facts

PhasePHASE1; PHASE2
Study typeInterventional
Enrollment24 (estimated)
Ages18 Years and up
SexAll
SponsorPeking University First Hospital (other)
Drugs / interventionsgecacitinib
Locations1 site (Beijing)
Trial IDNCT07012304 on ClinicalTrials.gov

What this trial studies

This Phase 1/2, single-site, interventional study will enroll 24 adults with moderate-to-severe chronic graft-versus-host disease who previously received two to five systemic therapies. Participants will take gecacitinib hydrochloride tablets for a minimum of 24 weeks, with the option to continue treatment up to 60 weeks if the investigator judges the disease stable and the drug is tolerated. The study focuses on feasibility, safety, and tolerability of this dual JAK/ACVR1 inhibitor in a treatment-refractory population and will monitor clinical status and adverse events throughout dosing. Concomitant non-interacting immunosuppressants are allowed under protocol-specified conditions.

Who should consider this trial

Good fit: Adults (≥18 years) with clinically diagnosed moderate-to-severe cGVHD after allogeneic HSCT who have persistent disease despite two to five prior systemic therapies and meet basic blood count and performance-status criteria.

Not a fit: Patients with recurrent malignancy, loss of full donor chimerism, concurrent use of other JAK inhibitors or certain excluded severe organ involvement (for example severe pulmonary cGVHD) are unlikely to be eligible or to benefit from this protocol.

Why it matters

Potential benefit: If successful, gecacitinib could become a new oral option to reduce disease activity and improve symptoms for patients with treatment-refractory chronic GVHD.

How similar studies have performed: Other JAK inhibitors such as ruxolitinib have shown benefit in steroid-refractory cGVHD, but gecacitinib's combined JAK and ACVR1 inhibition is a novel approach with limited prior clinical data.

Eligibility criteria

Show full inclusion / exclusion 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

Where this trial is running

Beijing

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Chronic Graft Versus Host Disease, cGVHD, JAK inhibitor, Gecacitinib

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.