A Multi-phase, Dose-Escalation followed by an Open-label, Randomized, Crossover Study of Oral ASTX030 (Cedazuridine and Azacitidine Given in Combination) Versus Subcutaneous Azacitidine in Subjects with Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML)

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-515098-93-00

Acronym

ASTX030-01

Enrollment

588

Registered

2025-05-30

Start date

2025-07-22

Completion date

Unknown

Last updated

2025-12-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chronic Myelomonocytic Leukemia (CMML), Myelodysplastic Syndromes (MDS)

Brief summary

• Ratio of azacitidine total cycle AUC 0-24 exposures after oral ASTX030 over SC azacitidine at 75 mg/m2/day

Detailed description

1 TEAEs including SAEs, 2 Percent LINE-1 demethylation change from baseline between SC azacitidine and ASTX030 in Cycles 1 and 2, 3 Participants with MDS or CMML: - Clinical response rate - AML-free survival - Duration of response - OS - Time to response - Transfusion independence

Interventions

DRUGAzacitidine

DRUGCedazuridine

DRUGVidaza 25 mg/ml powder for suspension for injection

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
• Ratio of azacitidine total cycle AUC 0-24 exposures after oral ASTX030 over SC azacitidine at 75 mg/m2/day	—

Secondary

Measure	Time frame
1 TEAEs including SAEs, 2 Percent LINE-1 demethylation change from baseline between SC azacitidine and ASTX030 in Cycles 1 and 2, 3 Participants with MDS or CMML: - Clinical response rate - AML-free survival - Duration of response - OS - Time to response - Transfusion independence	—

Countries

Czechia, France, Germany, Hungary, Italy, Poland, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026