Gecacitinib in the Treatment of Steroid-Refractory/Dependent Chronic Graf Versus Host Disease (cGVHD).

NCT07476872 | Not Yet Recruiting Phase 1

• 1 other identifier: IIT2026016
• Study Type: interventional
• Target: 33
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study aims to evaluate the safety and efficacy of Gecacitinib in patients with steroid-refractory/dependent active chronic graft versus host disease (cGVHD).

Conditions

cGVHD; HSCT

Keywords

cGVHD; Gecacitinib; HSCT

Outcome Measures

Primary Outcomes (2)

• phase Ib: Maximal Tolerable Dose (MTD)

  If dose limiting toxicity (DLT) occurs in 2 or more subjects in a given dose group, the dose level in the previous dose group is considered MTD. (Patients in phase Ib)

  Baseline up to 28 days

• phase IIa: Overall response rate (ORR) on Cycle 7 Day 1

  Percentage of participants achieving complete response (CR) and partial response (PR) during the study according to the cGVHD 2014 NIH Consensus Criteria.

  Cycle 7 Day 1

Secondary Outcomes (12)

• phase Ib: Recommended phase II dose (RP2D)

  Baseline up to 28 days

• phase IIa: Rate of Failure-free Survival (FFS)

  Baseline to when the last participant reached Cycle 7 Day 1

• phase IIa: Rate of Participants With Clinically Relevant Improvement of the Modified Lee cGvHD Symptom Scale Score

  Cycle 7 Day 1

• phase IIa: Best overall response (BOR)

  Cycle 7 Day 1

• phase IIa: Proportion of patients who achieved ORR (CR+PR) at Cycle 4 Day 1.

  Cycle 4 Day 1

Other Outcomes (3)

• phase IIa: Changes in concentration of cGVHD-associated biomarkers

  Baseline; up to Cycle 7 Day 1

• phase IIa: Changes in concentration of inflammatory cytokines.

  Baseline; up to Cycle 7 Day 1

• phase IIa: Changes in absolute counts of lymphocyte subsets

  Baseline; up to Cycle 7 Day 1

Study Arms (1)

Gecacitinib treatment

EXPERIMENTAL

Drug: Gecacitinib

Interventions

Gecacitinib DRUG

Phase Ib: Four dose cohorts of Gecacitinib are planned: 50 mg qd, 50 mg bid, 150 mg/day, and 100 mg bid. Dose escalation or de-escalation will follow the standard "3+3" design, starting at 50 mg BID. The dose may be escalated to 150 mg/day or 100 mg BID, or de-escalated to 50 mg qd. Subjects will receive continuous dosing for 28 days, or until they experience Dose-Limiting Toxicities (DLTs), cGVHD progression, or initiate new systemic therapy (whichever occurs first). Subjects who do not experience DLTs during Phase Ib may proceed to the Phase IIa dose-expansion stage. Phase IIa: Subjects will receive Gecacitinib at the Recommended Phase II Dose (RP2D). Treatment will continue until the completion of 6 treatment cycles (each cycle is comprised of 4 weeks), or until the occurrence of intolerable toxicity, cGVHD progression, or initiation of new systemic therapy (whichever occurs first).

Gecacitinib treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years, regardless of gender.
• Underlying hematologic malignancies or non-malignant disorders having received allogeneic hematopoietic stem cell transplantation.
• Diagnosis of active cGVHD according to the 2014 NIH consensus criteria, meeting the definition of steroid-refractory or steroid-dependent cGVHD. Prior lines of cGVHD therapy are not restricted. Definitions are as follows:
• Steroid-refractory cGVHD is defined as meeting any of the following criteria: disease progression despite the use of prednisone ≥1 mg/kg/day (or equivalent dose of corticosteroids) for at least 1 week; OR, persistent disease symptoms with no improvement despite the use of prednisone ≥0.5 mg/kg/day or ≥1 mg/kg every other day (or equivalent dose of corticosteroids) for at least 4 weeks.
• Steroid-dependent cGVHD is defined as the requirement for a maintenance dose of prednisone \>0.25 mg/kg/day or \>0.5 mg/kg every other day (or equivalent dose of corticosteroids) to prevent disease flare or progression, and failure to successfully taper the dose to a lower level in at least 2 separate attempts spaced ≥8 weeks apart.
• Platelet count ≥50 × 10⁹/L and absolute neutrophil count (ANC) ≥0.5 × 10⁹/L, without the use of colony-stimulating factors, androgens, erythropoietin, thrombopoietin, or platelet transfusion within 7 days prior to screening.
• Adequate major organ function, defined as meeting the following criteria: ALT and AST ≤ 2.5 × upper limit of normal (ULN); direct and total bilirubin ≤ 2.0 × ULN; serum creatinine ≤ 1.5 × ULN.
• Stable underlying disease without evidence of progression or relapse.
• Karnofsky Performance Status (KPS) ≥ 60%.
• Voluntarily participate in this study, provide signed informed consent, demonstrate good compliance, and be able to adhere to the study and follow-up procedures

You may not qualify if:

• Post-transplant lymphoproliferative disorder, or loss of full donor chimerism due to other reasons.
• Previous use of, or current treatment with, other JAK inhibitors at the time of screening.
• History or presence of major diseases or clinically significant organ dysfunction that cannot be adequately controlled by treatment and may interfere with study completion:
• Congestive heart failure of New York Heart Association (NYHA) class III-IV, or documented history of diastolic or systolic dysfunction (e.g., left ventricular ejection fraction \<40% by echocardiography), or uncontrolled/unstable angina or myocardial infarction.
• Uncontrolled diabetes (blood glucose \>250 mg/dL or \>13.9 mmol/L).
• Hypertension that cannot be adequately controlled to systolic blood pressure \<160 mmHg and diastolic blood pressure \<100 mmHg despite combination antihypertensive therapy.
• Peripheral neuropathy (Grade 2 or higher per NCI-CTCAE v5.0 criteria).
• Patients with any uncontrolled bacterial, viral, or fungal infection.
• Positive for HIV at screening, or active hepatitis B virus infection (HBsAg positive and HBV-DNA positive or above the upper limit of normal), or positive for HCV antibody with detectable HCV-RNA.
• History of tuberculosis or positive interferon-γ release assay at screening.
• Concurrent use of strong CYP3A4 inhibitors.
• History of progressive multifocal leukoencephalopathy.
• Known or suspected hypersensitivity to Gecacitinib hydrochloride, drugs of the same class, or any of their excipients.
• Pregnant or lactating women, or patients unwilling to use effective contraception during Gecacitinib treatment and for 1 week after the last dose.
• Inability to swallow oral tablets.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2026
• First Posted: March 17, 2026
• Study Start: March 16, 2026
• Primary Completion (Estimated): November 1, 2027
• Study Completion (Estimated): November 1, 2028
• Last Updated: March 17, 2026

Record last verified: 2026-03