Gecacitinib Pre-, During- and Post-HSCT for Patients With Primary or Secondary Myelofibrosis (CONTINUUM-MF)

A Phase 2 Clinical Study of Gecacitinib in Peri-transplant Period of Hematopoietic Stem Cell Transplantation in Patients With Myelofibrosis (MF)

The investigators evaluate the efficacy and safety of Gecacitinib in patients with myelofibrosis (MF) before, during, and after allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Study Overview

• Status: Not yet recruiting
• Conditions: Myelofibrosis (MF); HSCT
• Intervention / Treatment: Drug: Gecacitinib (also known as Jaktinib)

• Study Type: Interventional
• Enrollment (Estimated): 39
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Aged 18-75 years, regardless of gender;
2. Diagnosis of primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV-MF), or post-essential thrombocythemia myelofibrosis (post-ET-MF) according to the 2022 WHO diagnostic criteria;
3. Meeting the criteria for intermediate-risk or high-risk groups per the DIPSS-plus classification;
4. Scheduled to undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT), including transplants from HLA-matched or mismatched related or unrelated donors;
5. ECOG performance status ≤2 and Karnofsky performance status ≥60%;
6. Capable of understanding and signing the informed consent form, and able to comply with study and follow-up procedures.

Exclusion Criteria:

1. Patients using other JAK inhibitors (except for Gecacitinib) at the time of screening may be enrolled if they switch to Gecacitinib treatment prior to screening.
2. Patients who have previously undergone allogeneic hematopoietic stem cell transplantation or organ transplantation.
3. Disease progression to accelerated or blast phase (peripheral blood or bone marrow blast percentage ≥10% at any time prior to transplantation).

4. Presence of significant medical conditions or marked organ dysfunction that cannot be adequately controlled and may affect the completion of this study:

   1. Congestive heart failure classified as New York Heart Association (NYHA) Class III-IV, or documented history of diastolic or systolic dysfunction (e.g., LVEF <40% measured by echocardiography), or uncontrolled or unstable angina or myocardial infarction.
   2. Uncontrolled diabetes (>250 mg/dL or >13.9 mmol/L).
   3. Hypertension that cannot be reduced to the following range despite combination antihypertensive therapy (systolic blood pressure <160 mmHg, diastolic blood pressure <100 mmHg).
   4. Peripheral neuropathy (≥ Grade 2 per NCI-CTC AE v5.0 criteria).
   5. Serum creatinine >1.5 × ULN.
   6. ALT or AST >2.5 × ULN, or DBIL or TBIL >2.0 × ULN.
5. Patients with any bacterial, viral, or fungal infection not adequately controlled.
6. HIV-positive at screening, or active hepatitis B virus infection (HBsAg-positive with HBV-DNA positivity or above the normal reference range), or HCV antibody-positive with HCV-RNA positivity.
7. History of tuberculosis or positive interferon-gamma release assay at screening.
8. Suspected hypersensitivity to Gecacitinib Hydrochloride, drugs of the same class, or any of their excipients.
9. Pregnant or breastfeeding women, or patients unwilling to use effective contraception during Gecacitinib treatment and for one week after the last dose.
10. Patients with any other comorbidities that may interfere with the study or a history of prior malignancies.
11. Patients unable to take oral tablets.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Gecacitinib treatment

Drug: Gecacitinib (also known as Jaktinib); Gecacitinib treatment is initiated or continued at least two weeks before transplantation (Day -14) at a dose of 50 mg bid. This dose is maintained during preconditioning and the transplantation period until hematopoietic reconstitution, after which the dose is increased to 100 mg bid once platelet count recovers to ≥50×10⁹/L and absolute neutrophil count (ANC) recovers to ≥0.5×10⁹/L. The 100 mg bid dose is maintained until six months post-transplantation, after which it is adjusted to 50 mg bid until one year post-transplantation.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
1-year GVHD-free and relapse-free survival (GRFS) rate from the date of transplant	GRFS is defined as the absence of grade 3 to 4 acute GVHD, chronic GVHD requiring systemic immunosuppressive treatment, disease relapse, and death.	1 year post-HSCT

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative incidence of aGVHD	Cumulative incidence of grades II-IV and II-IV acute GVHD at +100 days and 6 months after HSCT	+100 days and 6 months post-HSCT
Cumulative incidence of cGVHD	Cumulative incidence of moderate to severe chronic GVHD at 6 months and 1 year post-HSCT	6 months and 1 year post-HSCT
The molecular relapse rate of MF	The molecular relapse rate of MF at 1 year post-HSCT.	1 year post-HSCT
Non-relapse mortality (NRM) rates	Non-relapse mortality (NRM) is defined as non-relapse death due to any cause without the recurrence or progression of myelofibrosis.	6 months and 1 year post-HSCT
Rate of Engraftment	Engraftment is defined as the patient achieving peripheral blood neutrophil counts >0.5×10⁹/L for three consecutive days and platelet counts >20×10⁹/L for seven consecutive days, without the need for platelet transfusion.	100 days post-HSCT
Proportion of patients with baseline splenomegaly achieving a ≥35% reduction in spleen volume.	Proportion of patients with baseline splenomegaly (palpable spleen edge at or beyond at least 5 cm below the costal margin) achieving a ≥35% reduction from baseline in spleen volume.	100 days, 6 months, and 1 year post-HSCT
Overall Survival	Overall survival is measured as the time from the hematopoietic stem cell transplantation (HSCT) until death. Participants without an event will be censored at the date of last contact.	1 year post-HSCT
Progression Free Survival (PFS)	PFS is defined as the time from the date of the first stem cell infusion until death from any cause, disease progression, or hematological relapse, whichever occurs first.	1 year post-HSCT
Toxicity rate	Cumulative incidence of treatment related adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE v5.0). Early deaths from all other causes are considered a competing risk.	From the first dose to 28 days after the last dose.

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in absolute counts of lymphocyte subsets	Assessment of changes in the absolute numbers (cells/µL) of major lymphocyte subsets in peripheral blood, including T cells (CD3+), helper T cells (CD3+CD4+), cytotoxic T cells (CD3+CD8+), B cells (CD19+), and natural killer (NK) cells (CD16+CD56+).	3 months, 6 months, and 1 year.
Changes in concentrations of immunoglobulins	Measurement of changes in the levels of serum immunoglobulins, including IgG, IgA, and IgM, reported in g/L.	3 months, 6 months, and 1 year

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Estimated): March 16, 2026
• Primary Completion (Estimated): April 1, 2029
• Study Completion (Estimated): April 1, 2030

Study Registration Dates

• First Submitted: February 26, 2026
• First Submitted That Met QC Criteria: March 12, 2026
• First Posted (Actual): March 13, 2026

Study Record Updates

• Last Update Posted (Actual): March 13, 2026
• Last Update Submitted That Met QC Criteria: March 12, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: HSCT; myelofibrosis; Gecacitinib
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders; Hemic and Lymphatic Diseases; Primary Myelofibrosis
• Other Study ID Numbers: IIT2026005

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No