NCT02103478

Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS)

A Phase1-2 Pharmacokinetic Guided Dose-Escalation and Dose-Confirmation Study of ASTX727, a Combination of the Oral Cytidine Deaminase Inhibitor (CDAi) E7727 With Oral Decitabine in Subjects With Myelodysplastic Syndromes (MDS)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 130 (actual)
• Sponsor: Astex Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This first-in-human, 3-stage, open-label study evaluated the safety and pharmacokinetics of ASTX727, as well as determined the dose for later stages.

Detailed description

The trial was designed to define daily doses of the individual components (cedazuridine \[E7727\] or decitabine) so that decitabine exposure after oral administration would be comparable to exposure after IV decitabine at the approved daily dose of 20 mg/m\^2. The main objective of Phases 1 and 2 was to establish and confirm the doses of the 2 components to be used in the final fixed-dose combination (FDC) product (ASTX727) using mainly pharmacokinetics and pharmacodynamics as endpoints.

Conditions

• Myelodysplastic Syndrome
• MDS

Interventions

Type	Name	Description
DRUG	ASTX727 Dose Escalation	Oral investigational product and approved IV decitabine
DRUG	ASTX727 Dose Confirmation	Randomization cross over design for courses 1 and 2
DRUG	ASTX727 Fixed-Dose Combination	Fixed-dose investigational product

Timeline

• Start date: 2014-10-28
• Primary completion: 2018-06-05
• Completion: 2019-12-04
• First posted: 2014-04-04
• Last updated: 2024-08-27
• Results posted: 2020-10-19

Locations

17 sites across 2 countries: United States, Canada