A Study of XS-04 in Patients with Relapsed or Refractory Hematologic Malignancies

Phase 1

Recruiting

• Conditions: Acute Myeloid Leukemia; Myelodysplastic Syndrome; B-cell Lymphoma
• Interventions: Drug: XS-04 tablet

• Registration Number: NCT06820268
• Lead Sponsor: NovaOnco Therapeutics Co., Ltd.

Brief Summary

Evaluation of the safety, tolerability, pharmacokinetics, and preliminary efficacy of XS-04 in patients with relapsed or refractory hematologic malignancies

Detailed Description

Main Objective: To evaluate the safety and tolerability of XS-04 tablets in patients with relapsed or refractory hematologic malignancies. To determine the maximal tolerated dose (MTD) and/or the recommended phase 2 dose (RP2D) of XS-04 tablets. Secondary Objective: To determine the pharmacokinetic (PK) characteristics of XS-04 tablets after single and multiple oral administrations. To preliminarily evaluate the efficacy of XS-04 tablets in patients with relapsed or refractory hematologic malignancies. Exploratory Objective: To preliminarily assess the metabolic transformation characteristics after oral administration of XS-04 tablets. To evaluate the population PK and exposure-effect (E-R) relationship. To explore the association between relevant biomarkers and drug efficacy; to explore the correlation between PD biomarkers and PK. To assess the impact of food on the PK characteristics of orally administered XS-04 tablets in patients.

Study Timeline

• Study First Submitted: 2025-01-09
• Study Start: 2025-01-14
• Status Verified: 2025-02
• Study First Submitted QC: 2025-02-05
• Study First Posted: 2025-02-11
• Last Update Submitted: 2025-02-27
• Last Update Posted: 2025-03-04
• Primary Completion: 2027-11-01
• Study Completion: 2028-03-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 168

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
A multicenter, open-label, single-arm Phase I dose-escalation and dose-expansion clinical study	XS-04 tablet	For drugs, use the generic name and include dosage form, dosage, frequency, and duration. Note: Group description not yet entered.\] XS-04 tablet specifications: 0.5 mg, 5 mg, 20 mg. Dose escalation phase: 1 mg, 3 mg, 10 mg, 20 mg, 40 mg, 80 mg, 120 mg, 160 mg/day (dose adjustment allowed based on actual escalation situation); Cycle 0 phase, oral administration once (dose is half of the total daily dose for the respective dose group), observe for two days (based on the PK results of the first 1-2 dose groups, decide whether the remaining dose groups need Cycle 0 phase); thereafter Cycle 1-N phase, each 28 days is one treatment cycle, continuous administration, two times daily. Dose regimen adjustments are allowed based on PK, safety, and efficacy data in this trial. Dose expansion phase: Based on the results of the dose escalation phase, cohort one and cohort two are planned to use the RP2D dose, cohort three safety lead-in phase is

Primary Outcome Measures

Name	Time	Method
The occurrence of treatment-related adverse events meeting the criteria for dose limiting toxicities (DLTs)	At the end of C1D21(each cycle is 28 days)	Safety indicators
Maximum Tolerated Dose (MTD) and/or Recommended Phase II Dose (RP2D)	Up to 24 months	Safety indicators and efficacy indicators

Secondary Outcome Measures

Name	Time	Method
The occurrence of adverse events (AEs) reported in all subjects who received study drug	From enrollment up to 30 days after last dose	Safety indicators
Pharmacokinetic variables including maximum plasma concentration (Cmin)	Cycle1(28days）	Safety indicators
PK Parameters After Single and Multiple Oral Doses of XS-04 Tablets	Cycle 0 to Cycle 3	Safety indicators
The occurrence of treatment-emergent adverse events (TEAs)	From first dose up to 30 days after last dose	Safety indicators
Pharmacokinetic variables including maximum plasma concentration (Cmax)	Cycle1(28days）	Safety indicators

Trial Locations

Locations (4)

Beijing Cancer Hospital; 🇨🇳 Beijing, China

Sun Yat-sen University Cancer Center; 🇨🇳 GuangDong, China

Affiliated Hospital of Hebei University; 🇨🇳 Hebei, China

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology; 🇨🇳 Hubei, China