Clinical trial NCT01571648: A Phase 1 Study to Evaluate the Pharmacokinetics and Tolerability of Oral Azacitidine in Japanese Patients With Myelodysplastic Syndromes
Official title: A Phase 1, Multicenter, Open-label Study to Evaluate the Pharmacokinetics and Tolerability of Oral Azacitidine in Japanese Subjects With Myelodysplastic Syndromes
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Myelodysplastic Syndromes
Lead sponsor: Celgene
Sponsor class: INDUSTRY
First posted: 2012-04-05
Enrollment: 5
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events
timeFrame: 1 month
Secondary outcomes:
measure: PK- Maximum concentration in plasma (Cmax)
timeFrame: 0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose
measure: PK- Time to maximum plasma concentration (Tmax)
timeFrame: 0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose
measure: PK-Elimination rate constant (Kel)
timeFrame: 0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose
measure: PK-Terminal half-life (T1/2,z)
timeFrame: 0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose
measure: PK-Area under the plasma concentration-time curve (AUC)
timeFrame: 0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose
measure: PK-Apparent total body clearance (CL/F)
timeFrame: 0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose
measure: PK-Apparent volume of distribution (Vz/f)
timeFrame: 0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose
measure: Safety (type, frequency, severity, number of participants with adverse events)
timeFrame: Up to 2 years
measure: Efficacy (Hematologic response and hematologic improvement)
timeFrame: Up to 2 years
Other outcomes:
Locations:
city: Fukuoka
country: Japan
facility: Celgene Trial Site
city: Hiroshima
country: Japan
facility: Celgene Trial Site
city: Nagoya
country: Japan
facility: Celgene Trial Site
city: Osaka
country: Japan
facility: Celgene Trial Site
city: Tokyo
country: Japan
facility: Celgene Trial Site
Study officials:
affiliation: Celgene K.K.
name: Masamitsu Harata
role: STUDY_DIRECTOR
References: