A Phase 1 Study to Evaluate the Pharmacokinetics and Tolerability of Oral Azacitidine in Japanese Patients With Myelodysplastic Syndromes

A Phase 1, Multicenter, Open-label Study to Evaluate the Pharmacokinetics and Tolerability of Oral Azacitidine in Japanese Subjects With Myelodysplastic Syndromes

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01571648

Enrollment

Registered

2012-04-05

Start date

2012-04-01

Completion date

2013-01-01

Last updated

2019-11-12

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

Conditions

Myelodysplastic Syndromes

Brief summary

The purpose of this study is to evaluate the tolerability of oral azacitidine in the treatment of patients with Myelodysplastic Syndromes (MDS).

Interventions

DRUGOral azacitidine

Patients will receive 300 mg dose of oral azacitidine administered once daily for the first 21 days of each 28-day treatment cycle.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

20 Years to No maximum

Healthy volunteers

Inclusion criteria

Patients must satisfy the following criteria to be enrolled in the study: * Have a documented diagnosis of myelodysplastic syndromes (MDS) according to World Health Organization (WHO) 2008 classification * Age ≥ 20 years; * Written informed consent; * Eastern Cooperative Oncology Group (ECOG) performance status of 0-2; * Resolution of any toxic effects of prior anti-cancer therapy; and * Negative urine or serum pregnancy test on females of childbearing potential.

Exclusion criteria

The presence of any of the following will exclude a patient from enrollment: * Treatment with chemotherapy, radiotherapy, or surgery within 4 weeks of study registration; * Pregnant or breast-feeding females; * Previous or concomitant malignancy other than MDS; * Significant active cardiac disease within the previous 6 months; * Uncontrolled systemic infection or * Known infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C

Design outcomes

Primary

Measure	Time frame	Description
Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events	1 month	Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events

Secondary

Measure	Time frame	Description
PK- Time to maximum plasma concentration (Tmax)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK- Time to maximum plasma concentration (Tmax)
PK-Elimination rate constant (Kel)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Elimination rate constant (Kel)
PK-Terminal half-life (T1/2,z)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Terminal half-life (T1/2,z)
PK-Area under the plasma concentration-time curve (AUC)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Area under the plasma concentration-time curve (AUC)
PK- Maximum concentration in plasma (Cmax)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK- Maximum concentration in plasma (Cmax)
PK-Apparent volume of distribution (Vz/f)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Apparent volume of distribution (Vz/f)
Safety (type, frequency, severity, number of participants with adverse events)	Up to 2 years	Safety (type, frequency, severity, number of participants with adverse events)
Efficacy (Hematologic response and hematologic improvement)	Up to 2 years	Efficacy (Hematologic response and hematologic improvement)
PK-Apparent total body clearance (CL/F)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Apparent total body clearance (CL/F)

Countries

Japan

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026