Low-Dose Cytarabine in Treating Infants With Down Syndrome and Transient Myeloproliferative Disorder

Phase 3

Withdrawn

• Conditions: Leukemia
• Interventions: Procedure: observation; Drug: cytarabine

• Registration Number: NCT00411281
• Lead Sponsor: Children's Oncology Group

Brief Summary

RATIONALE: Drugs used in chemotherapy, such as cytarabine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing. Giving low-doses of cytarabine may be an effective treatment for Down syndrome and transient myeloproliferative disorder. Sometimes the disease may not need treatment until it progresses. In this case, observation may be sufficient.

PURPOSE: This phase III trial is studying low-dose cytarabine to see how well it works in treating infants with Down syndrome and transient myeloproliferative disorder.

Detailed Description

OBJECTIVES:

Primary

* Determine whether very low-dose cytarabine can improve event-free survival (EFS) rates in infants with high-risk transient myeloproliferative disorder (TMD), using high-risk TMD patients from clinical trial COG-A2971 for historic comparison, and in infants with intermediate-risk TMD, using intermediate-risk TMD patients from clinical trial COG-A2971 for historic comparison.

* Maintain the current high overall EFS rate in low-risk TMD patients.

Secondary

* Assess the toxicity of this regimen in these patients.

OUTLINE: This is a nonrandomized, multicenter, crossover study. Patients are stratified according to disease risk (high or intermediate vs low).

* Group I (patients with high- or intermediate-risk transient myeloproliferative disorder \[TMD\]): Patients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation.

* Group II (patients with low-risk TMD): Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I.

After completion of study treatment, patients are followed periodically for 10 years.

PROJECTED ACCRUAL: A total of 180 patients will be accrued for this study.

Study Timeline

• Study Start: 2006-03
• Study First Submitted: 2006-12-11
• Study First Submitted QC: 2006-12-11
• Study First Posted: 2006-12-13
• Primary Completion: 2007-11
• Study Completion: 2007-11
• Status Verified: 2013-08
• Last Update Submitted: 2015-09-28
• Last Update Posted: 2015-09-30

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Group II	observation	Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I.
Group I	cytarabine	Patients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation.

Primary Outcome Measures

Name	Time	Method
Event-free survival

Secondary Outcome Measures

Name	Time	Method
Percentage of patients experiencing grade 3-4 toxicity
Incidence of subsequent leukemia in patients for whom transient myeloproliferative disorder is resolved
Overall survival
Disease-related mortality