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Sponsors and Collaborators: |
Children's Oncology Group National Cancer Institute (NCI) |
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Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00411281 |
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.
Condition | Intervention | Phase |
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Leukemia |
Drug: cytarabine Procedure: observation |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized |
Official Title: | Treatment of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS) |
Estimated Enrollment: | 180 |
Study Start Date: | March 2006 |
Estimated Primary Completion Date: | February 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Group I: Experimental
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.
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Drug: cytarabine
Given subcutaneously
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Group II: No Intervention
Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I.
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Procedure: observation
No intervention
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OBJECTIVES:
Primary
Secondary
OUTLINE: This is a nonrandomized, multicenter, crossover study. Patients are stratified according to disease risk (high or intermediate vs low).
PROJECTED ACCRUAL: A total of 180 patients will be accrued for this study.
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Diagnosis of Down syndrome or Down syndrome mosaicism (confirmed by karyotype analysis within the past 3 weeks) AND 1 of the following:
High-, intermediate-, or low-risk TMD, as defined by the following:
High-risk TMD, meeting 1 of the following criteria:
Life-threatening cardio-respiratory compromise due to complications of TMD (e.g., organomegaly or effusions)
Any degree of hepatomegaly (palpable on physical exam) combined with life-threatening hepatic dysfunction
Intermediate-risk TMD, meeting all of the following criteria:
Low-risk TMD, meeting all of the following criteria:
PATIENT CHARACTERISTICS:
PRIOR CONCURRENT THERAPY:
Study ID Numbers: | CDR0000518352, COG-AAML0532 |
Study First Received: | December 11, 2006 |
Last Updated: | January 30, 2009 |
ClinicalTrials.gov Identifier: | NCT00411281 History of Changes |
Health Authority: | Unspecified |
acute myeloid leukemia/transient myeloproliferative disorder |
Antimetabolites Immunologic Factors Hematologic Diseases Myeloproliferative Disorders Chromosome Disorders Leukemia, Myeloid Leukemia, Myeloid, Acute Immunosuppressive Agents Antiviral Agents Mental Retardation |
Leukemia Acute Myelocytic Leukemia Genetic Diseases, Inborn Abnormalities, Multiple Neurologic Manifestations Down Syndrome Congenital Abnormalities Bone Marrow Diseases Neurobehavioral Manifestations Cytarabine |
Antimetabolites Anti-Infective Agents Antimetabolites, Antineoplastic Immunologic Factors Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Physiological Effects of Drugs Leukemia Therapeutic Uses Abnormalities, Multiple Congenital Abnormalities Neurobehavioral Manifestations Cytarabine Neoplasms by Histologic Type |
Hematologic Diseases Nervous System Diseases Chromosome Disorders Myeloproliferative Disorders Antiviral Agents Immunosuppressive Agents Pharmacologic Actions Mental Retardation Neoplasms Genetic Diseases, Inborn Neurologic Manifestations Down Syndrome Bone Marrow Diseases |