A Study to Evaluate Safety , Efficacy and Pharmacokinetics of WJ01024 Tablets Combined With Ruxolitinib in Patients With Myelofibrosis

A Phase Ib/II Clinical Study to Evaluate the Safety , Efficacy and Pharmacokinetics of WJ01024 Tablets Combined With Ruxolitinib Tablets in Patients With Myelofibrosis

This is a Phase Ib/II clinical study to evaluate the safety , efficacy and pharmacokinetics of WJ01024 tablets combined with Ruxolitinib tablets in patients with myelofibrosis.The study will be conducted in two phases: Phase 1b and Phase 2.Phase Ib is a dose extension study of WJ01024 tablets combined with ruxolitinib tablets. It is planned to recruit patients with medium to high-risk myelofibrosis accompanied by splenomegaly who have had poor response or intolerance to the previously approved JAK inhibitors for myelofibrosis. Phase II is the efficacy extension stage of WJ01024 tablets combined with ruxolitinib Tablets. It is planned to expand two groups of people. Group A will expand to recruit patients with medium-high risk of myelofibrosis accompanied by splenomegaly who have not responded well to the previously approved JAK inhibitors for myelofibrosis. Group B expanded to recruit patients with medium-high-risk myelofibrosis accompanied by splenomegaly who were intolerant after treatment with previously approved JAK inhibitors for myelofibrosis.

Study Overview

• Status: Active, not recruiting
• Conditions: Intermediate- and High-risk Myelofibrosis (MF) Patients With Splenomegaly
• Intervention / Treatment: Drug: Ruxolitinib phosphate tablet（Jakavi,NOVARTIS,9104733)

Detailed Description

Ruxolitinib tablets have been approved in China since April 2017 as the first-line treatment for most intermediate- and high-risk myelofibrosis (MF) patients with splenomegaly. Compared with supportive treatment alone, ruxolitinib tablets have the efficacy of reducing spleen size and improving the symptoms of myelofibrosis, as well as reducing the risk of death and prolonging the survival of MF patients.Although the clinical efficacy of Ruxolitinib tablets has been confirmed, only about half of MF patients can achieve the ideal therapeutic effect (≥35% reduction in spleen volume and ≥50% improvement in disease symptoms at 24 weeks). Therefore, there is an urgent need for innovative drugs that can be combined with Ruxolitinib tablets to enhance therapeutic efficacy and meet clinical needs.

WJ01024 is a small molecule inhibitor of XPO-1, belonging to the same target small molecule compounds as Selinexor. Compared with Selinexor, the metabolism rate of this product is faster, which can reduce the toxicity in the body. In the Ba/F3-EPOR-JAK2-V617F cell model, the combination of this product and ruxolitinib tablets can enhance its anti-cell proliferation activity. In a clinical study initiated by researchers, this product has shown preliminary efficacy as a monotherapy in patients with myelofibrosis who have relapsed, are refractory to, or intolerant of JAK inhibitors, and is expected to have fewer toxic and side effects than drugs targeting the same site. Based on this, the following research is planned.

This is a Phase Ib/II clinical study to evaluate the safety , efficacy and pharmacokinetics of WJ01024 tablets combined with Ruxolitinib tablets in patients with myelofibrosis.The study will be conducted in two phases: Phase 1b and Phase 2.

Phase Ib is a dose extension study of WJ01024 tablets combined with ruxolitinib tablets. It is planned to recruit patients with medium to high-risk myelofibrosis accompanied by splenomegaly who have had poor response or intolerance to the previously approved JAK inhibitors for myelofibrosis.

Phase II is the efficacy extension stage of WJ01024 tablets combined with ruxolitinib Tablets. It is planned to expand two groups of people. Group A will expand to recruit patients with medium-high risk of myelofibrosis accompanied by splenomegaly who have not responded well to the previously approved JAK inhibitors for myelofibrosis. Group B expanded to recruit patients with medium-high-risk myelofibrosis accompanied by splenomegaly who were intolerant after treatment with previously approved JAK inhibitors for myelofibrosis.

• Study Type: Interventional
• Enrollment (Actual): 33
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Zhengzhou
    • Zhengzhou, Zhengzhou, China, 450000
      • Henan Cancer Hospital

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Subjects voluntarily participate in the study after receiving full informed consent and sign informed consent;
• Age ≥18 years old, gender unlimited;
• Patients diagnosed with primary myelofibrosis (PMF) according to WHO criteria (2016 edition), or with ET secondary myelofibrosis (PET-MF) or PV secondary myelofibrosis (PPV-MF) according to International Working Group on Myelofibrosis Research and Treatment (IWG-MRT) criteria; They could be included regardless of JAK2 mutation；
• Participants with international prognostic scoring system (DIPSS) risk category of intermediate-1, or intermediate-2, or high-risk；
• ECOG score 0~2;
• No stem cell transplantation plan in the near future;
• Spleen enlargement:palpable splenomegaly(≥5cm below left costal margin)or radiologically confirmed spleen volume ≥450 cm^3 using MRI/CT;
• Patients with intolerance or a suboptimal response to prior JAK inhibitor therapy；
• Sufficient hematology and organ function；

Exclusion Criteria:

• More than 10% blasts in peripheral blood or bone marrow；
• Previous treatment with XPO1 inhibitors；
• Unable to cooperate with or unable to perform MRI or CT scans as deemed necessary by sponsor and investigator；
• Treatment with a powerful CYP3A inhibitor or inducer within 14 days prior to initial administration；

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• 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: WJ01024 tablet; If needed, additional descriptive information (including which interventions are administered in each arm) to differentiate each arm from other arms in the clinical trial.	Drug: Ruxolitinib phosphate tablet（Jakavi,NOVARTIS,9104733); 5-20mg BID (dosage per investigator judgement,JAKi intolerable pts will receive recuded dose of RUX(≥ 5mg BID),and suboptimal JAKi response pts will receive RUX of 15-20 mg BID）

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
AE	Incidence rate and severity of adverse events and serious adverse events,as well as abnormal changes in clinical significance laboratory tests and other examinations	3 years
SVR35	At weeks 12 and 24, the ratio of splenic volume reduction ≥35% (SVR35) was assessed by the investigator	3 years
The absolute value changes of the total symptom score in MPN-SAF-TSS	The absolute value changes of the overall symptom score in MPN-SAF-TSS based on subject assessment in weeks 12 and 24 compared with the baseline	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
OS	Overall survival	3 years
The blood concentration of WJ01024	AUC of WJ01024,C1D1,C2D1,C4D1,C6D1 1 h after WJ01024 administration Both in phase Ib and II	1.5 years
Anemia response rate	Anemia response rate at Week 12 and Week 24 as assessed by the investigator according to IWG-MRT criteria	3 years
LDH	Evaluation of changes in serum LDH levels	3 years
Incidence rate and severity of adverse events nd serious adverse events	Incidence and severity of adverse events nd serious adverse events,as well as abnormal changes in clinical significance laboratory tests and other examinations	3 years
ORR：CR + PR + clinical improvement	Overall response rate (ORR, CR + PR + clinical improvement) as determined by the investigator according to IWG-MRT criteria	3 years
PFS	PFS as assessed by the investigator	3 years
Phase II：The rate of a reduction of ≥50% in the total symptom score in MPN-SAF-TSS	The rate of a reduction of ≥50% in the overall symptom score in MPN-SAF-TSS based on subject assessment in weeks 12 and 24 compared with the baseline	3 years

Collaborators and Investigators

• Sponsor: Suzhou Junjing BioSciences Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): July 28, 2025
• Primary Completion (Estimated): October 31, 2027
• Study Completion (Estimated): May 15, 2028

Study Registration Dates

• First Submitted: March 27, 2025
• First Submitted That Met QC Criteria: March 27, 2025
• First Posted (Actual): April 3, 2025

Study Record Updates

• Last Update Posted (Actual): March 30, 2026
• Last Update Submitted That Met QC Criteria: March 25, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathological Conditions, Anatomical; Hematologic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders; Hypertrophy; Primary Myelofibrosis; Splenomegaly; Gastrointestinal Agents; Cathartics; Lactitol
• Other Study ID Numbers: JS110-003-Ib/II

Drug and device information, study documents

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