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)

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.

Description

Eligibility

Age range

18+ years

Sex

All

Healthy volunteers

No

Interventions

• 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.

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