PK/Efficacy Bridging Study of ASTX727 in Chinese Subjects With Myelodysplastic Syndromes

NCT06091267 | Active Not Recruiting Phase 1

• 1 other identifier: 393-403-00072
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

Brief Summary

This is an Open-Label, Crossover, Pharmacokinetic and Efficacy Bridging Study of Oral ASTX727 versus IV Decitabine in Chinese Subjects with Myelodysplastic Syndromes

Conditions

Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (2)

• Complete Response Rate

  Assess efficacy \[Complete Response Rate (CR)\] of treatment with ASTX727 in Chinese subjects with myelodysplastic syndromes (MDS);

  An analysis is planned when the last enrolled patient have completed Follow-up 12 months.

• 5day_AUC0-τ

  Assess pharmacokinetic (PK) parameters (Total 5-day AUC exposures of decitabine) after treatment with ASTX727 (oral) versus decitabine for IV infusion for 5 days;

  An analysis is planned when the last enrolled patient have completed the treatment with ASTX727 (oral) versus decitabine for IV infusion for 5 day.

Secondary Outcomes (16)

• Objective Response Rate

  through study completion, an average of 1 year.

• Clinical Response Rate

  through study completion, an average of 1 year.

• Rate of transfusion independence

  through study completion, an average of 1 year.

• disease progression

  through study completion, an average of 1 year.

• Overall survival

  through study completion, an average of 1 year.

Study Arms (3)

ASTX727 and IV Decitabine

EXPERIMENTAL

Cycle1：ASTX727 tablets, oral, 1 tablet/day for 5 days；Cycle2：IV Decitabine, 20 mg/m\^2, is administered for 1 hour at a time for 5 days；≥ Cycle 3：ASTX727 tablets, oral, 1 tablet/day for 5 days

Drug: IV Decitabine; Drug: Decitabine and cedazuridine; Drug: only Decitabine and cedazuridine

IV Decitabine and ASTX727

ACTIVE COMPARATOR

Cycle1：IV Decitabine, 20 mg/m\^2, is administered for 1 hour at a time for 5 days； Cycle2：ASTX727 tablets, oral, 1 tablet/day for 5 days；≥ Cycle 3：ASTX727 tablets, oral, 1 tablet/day for 5 days

Drug: IV Decitabine; Drug: Decitabine and cedazuridine; Drug: only Decitabine and cedazuridine

ASTX727

EXPERIMENTAL

ASTX727 tablets, oral, 1 tablet/day for 5 days；

Drug: only Decitabine and cedazuridine

Interventions

IV Decitabine DRUG

The subjects will receive decitabine 20 mg/m\^2 IV daily × 5 days in 28-day cycles.

ASTX727 and IV Decitabine; IV Decitabine and ASTX727

Decitabine and cedazuridine DRUG

subjects will receive treatment with ASTX727, 1 tablet/day for 5 consecutive days, in 28-day cycles.

ASTX727 and IV Decitabine; IV Decitabine and ASTX727

only Decitabine and cedazuridine DRUG

subjects will receive treatment with ASTX727, 1 tablet/day for 5 consecutive days, in 28-day cycles, until disease progression, unacceptable toxicity, or the subject/investigator decides that the subject should discontinue treatment or withdraw from the trial.

ASTX727; ASTX727 and IV Decitabine; IV Decitabine and ASTX727

Eligibility Criteria

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

You may qualify if:

• Agree to participate in this trial and voluntarily sign the informed consent form.
• Men or women ≥ 18 years at the time of signing the informed consent form.
• Subjects with MDS previously treated or untreated with de novo or secondary MDS.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1 at screening.

You may not qualify if:

• Prior treatment with more than 1 cycle of azacitidine or decitabine.
• Cytotoxic chemotherapy or prior azacitidine or decitabine within 4 weeks of first dose of study treatment.
• Conditions as judged by the investigator to be inappropriate for participation in the clinical trial.
• Previous diagnosis of malignant tumor.
• History of immune deficiency.
• Acute myeloid leukemia (AML) with bone marrow or peripheral blast count ≥ 20% or other malignant hematological diseases.

Sponsors & Collaborators

• Otsuka Beijing Research Institute: lead

Study Sites (1)

The First Affiliated Hospital,Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310003, China

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Decitabine; decitabine and cedazuridine drug combination; cedazuridine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 22, 2023
• First Posted: October 19, 2023
• Study Start: October 16, 2023
• Primary Completion (Estimated): May 31, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: January 30, 2026

Record last verified: 2026-01

Locations

CN (1)