NCT01652781

5 Day Versus 7 Day Azacitidine in Lower Risk Myelodysplastic Syndrome

A Randomized, Phase II, Comparative Study With a Parallel Control for Evaluating the Efficacy and Safety of 5-day Azacitidine for Patients With Lower-risk Myelodysplastic Syndrome

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 92 (estimated)
• Sponsor: Seoul St. Mary's Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

Approved dosing schedule of azacitidine for myelodysplastic syndrome (MDS) is 75 mg/m\^2/day subcutaneous for 7 consecutive days every 28 days, which is based on the data from standard chemotherapy regimen and a Phase I safety clinical trial. Since the optimal dosage of this drug has not been found yet, it remains as a subject of clinical study that needs to be examined. If initial toxicity is minimized by developing dosage/regimen that replaces the standard therapy, it will be possible to provide continuous treatment with increased convenience by patients and treating physicians as well as improvement for safety in elderly patients or those with serious cytopenia. In addition, it is expected to lead to a better response by strictly keeping a treatment schedule. Recent US study showed that 5-day regimen showed similar treatment results, but retrospective data from Spain showed lower response rate in 5-day regimen. Considering the recent circumstances around dosage and schedule of azacitidine in lower risk MDS, a Phase II clinical trial is planned in lower risk MDS patients in order to explore the efficacy in 5-day treatment by comparing prospectively with 7-day standard regimen.

Detailed description

* Using block randomization, subjects of Low or intermediate (INT)-1 patients will be equally allocated to the following two types of regimens. 1. Group A: azacitidine 75mg/m\^2 subcutaneously for 7 days every 28 days + best supportive care 2. Group B: azacitidine 75mg/m\^2 subcutaneously for 5 days every 28 days + best supportive care * The study drug, azacitidine, is provided free of charge by Celgene until disease progression or relapse after response, or intolerable toxicity occurs in clinical study subject, or informed consent is withdrawn. * No crossover between arms is allowed. * Dose escalation in this study is not allowed; on the contrary, dose reduction or dose delay is possible based on adverse events and hematologic recovery.

Conditions

• Myelodysplastic Syndrome

Interventions

Type	Name	Description
DRUG	Azacitidine	5-day arm: azacitidine 75mg/m2 subcutaneously for 7 days every 28 days + best supportive care 7-day arm: azacitidine 75mg/m2 subcutaneously for 5 days every 28 days + best supportive care

Timeline

• Start date: 2012-03-01
• Primary completion: 2016-06-01
• Completion: 2016-12-01
• First posted: 2012-07-30
• Last updated: 2015-11-20

Locations

3 sites across 1 country: South Korea