Myelodysplastic SyndromesPhase 1NCT01571648

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

A Phase 1 study testing Oral azacitidine in people with Myelodysplastic Syndromes, run by Celgene at 5 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

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

Who is it for?: Ages 20 years and older
Conditions the study is for: Myelodysplastic Syndromes
What is being tested?: Oral azacitidine
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 5 participants
Where: 5 sitesJapan
Study dates: 1 Apr 2012to 1 Jan 2013
Phase: Phase 1
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 20 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

Japan5 sites

Celgene Trial Site
Fukuoka
Celgene Trial Site
Hiroshima
Celgene Trial Site
Nagoya
Celgene Trial Site
Osaka
Celgene Trial Site
Tokyo

Other trials in Japan

About this study

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

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

Conditions: Myelodysplastic Syndromes
Being tested: Oral azacitidine
Medical subject headings: Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT01571648
Study group	Role	What they receive
Oral azacitidine	Experimental	Not described Receives: Drug: Oral azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events
Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events
Measured: 1 month

Secondary outcomes (9)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Apr 20121 Jan 2013

First posted5 Apr 2012 (estimated)
Study start1 Apr 2012
Primary completion1 Jan 2013
Study completion1 Jan 2013

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
More trials run by CelgeneThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 5 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01571648
Sponsor's protocol ID: AZA-MDS-005
Status last verified: Nov 2019
First submitted: 3 Apr 2012
Last updated: 12 Nov 2019
Results posted: No
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Myelodysplastic Syndromes trials

Study at a glance

From the registry record, last updated by the sponsor on 12 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1, Multicenter, Open-label Study to Evaluate the Pharmacokinetics and Tolerability of Oral Azacitidine in Japanese Subjects With Myelodysplastic Syndromes
ClinicalTrials.gov ID: NCT01571648
Acronym / sponsor ID: AZA-MDS-005
Conditions: Myelodysplastic Syndromes
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 5 participants (actual)
Lead sponsor: CelgeneIndustry (pharma, biotech, devices)
Study start: 1 Apr 2012 (actual)
Primary completion: 1 Jan 2013 (actual)
Study completion: 1 Jan 2013 (actual)
First posted: 5 Apr 2012 (estimated)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Oral azacitidine	Experimental	Drug: Oral azacitidine

Who may qualify

Ages: 20 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

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

Key exclusion criteria (7 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Oral azacitidinePatients will receive 300 mg dose of oral azacitidine administered once daily for the first 21 days of each 28-day treatment cycle.	Drug	Oral azacitidine

Primary outcome

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

9 secondary outcomes

PK- Maximum concentration in plasma (Cmax)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose
Safety (type, frequency, severity, number of participants with adverse events)
Time frame: Up to 2 years
Efficacy (Hematologic response and hematologic improvement)
Time frame: Up to 2 years

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 Nov 2019.

Related trials for Myelodysplastic Syndromes

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RecruitingObservational study
Natural History of Trisomy 8-Associated Autoinflammatory Disease (TRIAD) and Related Disorders
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RecruitingPhase 1/2
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Masamitsu Harata study director

Celgene K.K.

5 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Oral azacitidine in people with Myelodysplastic Syndromes, run by Celgene at 5 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 12 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events (time frame: 1 month). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01571648.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Nov 2019.