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Recruiting Phase 2, Phase 3 NCT04256317

NCT04256317 A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)

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Clinical Trial Summary
NCT ID NCT04256317
Status Recruiting
Phase Phase 2, Phase 3
Sponsor Taiho Oncology, Inc.
Condition Myelodysplastic Syndromes
Study Type INTERVENTIONAL
Enrollment 316 participants
Start Date 2020-05-21
Primary Completion 2027-11-01

Trial Parameters

Condition Myelodysplastic Syndromes
Sponsor Taiho Oncology, Inc.
Study Type INTERVENTIONAL
Phase Phase 2, Phase 3
Enrollment 316
Sex ALL
Min Age 18 Years
Max Age N/A
Start Date 2020-05-21
Completion 2027-11-01
Interventions
AzacitidineASTX030 (cedazuridine + azacitidine)Azacitidine

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Brief 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.

Eligibility Criteria

Inclusion Criteria: * Phase 2 Monotherapy: 1\. Has Confirmed MDS, CMML, or other MDS/MPN diagnosis who are candidates to receive and benefit from single agent azacitidine and as applicable according to local country approvals and/or local institution standard practice. * Phase 3 Monotherapy: 1. Has confirmed MDS or CMML and is a candidate to receive and benefit from single agent azacitidine as applicable according to local country approvals and/or local institution standard practice: a) French-American-British myelodysplastic syndrome subtypes: refractory anemia (RA) or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and CMML or MDS with intermediate-2 or high risk MDS according to the International Prognostic Scoring System (IPSS). 2. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1

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