NCT04256317

A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)

A Multi-phase, Pharmacokinetics, Safety, and Efficacy Study of ASTX030 (Azacitidine and Cedazuridine) as Monotherapy in Subjects With Myeloid Neoplasm or in Combination With Venetoclax in Subjects With AML (AZTOUND Study)

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 316 (estimated)
• Sponsor: Taiho Oncology, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 Monotherapy arms, and Phase 1 and Phase 2 Combination Therapy arms. Phase 1 Monotherapy consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B). Phase 2 Monotherapy is a randomized, open-label, crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 Monotherapy is a randomized open-label crossover study comparing the final fixed dose of oral ASTX030 to SC azacitidine. Phase 1 Combination Therapy is an open-label, multicenter, randomized, exploratory study comparing ASTX030 and SC azacitidine in combination with venetoclax in participants with treatment-naïve AML. Phase 2 Combination Therapy is an open-label, single arm, study evaluating the efficacy, safety, pharmacokinetics (PK), and drug interactions of ASTX030 in combination with venetoclax in participants with treatment-naïve AML. The duration of this multi-phase study is approximately 8 years.

Detailed description

The Phase 1 and Phase 2 Monotherapy arms have completed enrollment. The Phase 3 Monotherapy, Phase 1 Combination Therapy, and Phase 2 Combination Therapy arms are open for enrollment.

Conditions

• Myelodysplastic Syndromes
• Acute Myeloid Leukemia
• Myelodysplastic Syndrome/Neoplasm
• Chronic Myelomonocytic Leukemia

Interventions

Type	Name	Description
DRUG	Azacitidine	Tablets/Capsules for oral administration and powder for reconstitution to aqueous suspension for SC administration.
DRUG	ASTX030 (cedazuridine + azacitidine)	FDC Capsules for oral administration.
DRUG	Azacitidine	Powder for reconstitution to aqueous suspension for SC administration.
DRUG	ASTX030 (cedazuridine + azacitidine)	Tablets/Capsules for oral administration.
DRUG	Cedazuridine	Tablets for oral administration.
DRUG	Venetoclax	Oral tablets.

Timeline

• Start date: 2020-05-21
• Primary completion: 2027-11-01
• Completion: 2028-11-01
• First posted: 2020-02-05
• Last updated: 2026-04-03

Locations

71 sites across 10 countries: United States, Canada, Czechia, France, Germany, Hungary, Italy, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study