Chronic Graft Versus Host DiseasePhase 1/2NCT07012304
Gecacitinib for cGVHD: Safety and Efficacy in Patients After ≥2 Lines of Prior Therapy
A Phase 1/2 study testing Gecacitinib Hydrochloride Tablets in people with Chronic Graft Versus Host Disease, run by Yujun DONG at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Graft Versus Host Disease
- What is being tested?
- Gecacitinib Hydrochloride Tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteChina
- Study dates
- 30 Jun 2025to 28 Feb 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking University First Hospital
Beijing
Yujun Dong, 0086010-83572211, dongy@hsc.pku.edu.cn
Yujun Dong
About this study
Official title: 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
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
- Being tested
- Gecacitinib Hydrochloride Tablets
- Keywords
- cGVHD, JAK inhibitor, Gecacitinib
- Medical subject headings
- Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gecacitinib Hydrochloride TabletsDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Gecacitinib | Experimental | Not described Receives: Drug: Gecacitinib Hydrochloride Tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Week 24
Secondary outcomes (11)
ORR at Week 12, 48 and 60
Measured: week 12, 48 and 60
Failure-free Survival (FFS)
Measured: 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.
Measured: Through study completion, an average of 24 months
Best Overall Response (BOR)
Measured: Up to week 24
Duration of Response (DOR)
Measured: Up to 24 months
Percentage of Participants With a ≥50% Reduction in Daily Corticosteroid Dose for ≥4 Weeks
Measured: Up to Day 180
Relapse Rate
Measured: Up to 24 months
Overall Survival (OS)
Measured: 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.
Measured: 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.
Measured: Through study completion, an average of 24 months
Adverse Events (AEs) and Adverse Drug Reactions (ADRs)
Measured: Up to 28 days after the last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2025
- Study start30 Jun 2025 (estimated)
- Primary completion28 Feb 2027 (estimated)
- Study completion28 Feb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Graft Versus Host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yujun DONG
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Yujun DONG, Peking University First Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07012304
- Sponsor's protocol ID
- cGVHD-06
- Status last verified
- Apr 2025
- First submitted
- 9 May 2025
- Last updated
- 10 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07012304
- Acronym / sponsor ID
- cGVHD-06
- Conditions
- Chronic Graft Versus Host Disease
- Keywords
- cGVHD, JAK inhibitor, Gecacitinib
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Yujun DONGUniversity, hospital or other
- Study start
- 30 Jun 2025 (estimated)
- Primary completion
- 28 Feb 2027 (estimated)
- Study completion
- 28 Feb 2029 (estimated)
- First posted
- 10 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gecacitinib | Experimental | Drug: Gecacitinib Hydrochloride Tablets |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (19 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gecacitinib Hydrochloride TabletsGecacitinib 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. | Drug | Gecacitinib |
Primary outcome
Overall Response Rate (ORR) at Week 24
Time frame: 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.
11 secondary outcomes
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
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
Time frame: Through study completion, an average of 24 months
Change from Baseline in EQ-5D-5L
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Gecacitinib Hydrochloride Tablets in people with Chronic Graft Versus Host Disease, run by Yujun DONG at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) at Week 24 (time frame: Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jun 2025.