NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia

Study Overview
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Brief Summary
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The overall objective is to improve the cure rate of children with newly diagnosed acute myeloid leukemia (AML) who undergo risk-adapted therapy. Stem cell transplantation (SCT) is reserved to high-risk patients defined by cytogenetics and response to chemotherapy. The efficacy and toxicity of Gemtuzumab ozogamicin (GO, Mylotarg) will be evaluated.

Detailed Description
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The overall objective is to improve the cure rate of pediatric patients with newly diagnosed acute myeloid leukemia (AML). The specific aims are as follows: 1.1 Therapeutic aims To improve the event-free survival (EFS) of AML patients who undergo risk-adapted therapy. To improve the overall survival (OS) by reserving stem cell transplantation (SCT) to high-risk patients based on cytogenetics and response to induction therapy. To compare the outcome of SCT using HLA-matched sibling donor (MSD) or HLA-matched unrelated donor (MUD). To assess the efficacy and toxicity of Gemtuzumab ozogamicin (GO, Mylotarg) as post consolidation therapy. 1.2 Biologic aims To study minimal residual disease (MRD) levels in blood and bone marrow (BM) at defined time points and to study the prognostic impact of MRD. To test in vitro cellular drug resistance at diagnosis and relapse, and correlate these data to background factors and clinical outcome. To secure storage of biological material from diagnosis for future biologic studies

Official Title
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NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia

Conditions
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Acute Myeloid Leukemia

Intervention / Treatment
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* Drug: Gemtuzumab ozogamicin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: AML as defined by the diagnostic criteria, Age < 19 years at time of study entry, Written informed consent Exclusion Criteria: Previous chemo- or radiotherapy, AML secondary to previous bone marrow failure syndrome, Down syndrome (DS), Acute promyelocytic leukemia (APL), Juvenile myelomonocytic leukemia (JMML), Myelodysplastic syndrome (MDS), Fanconi anemia, Positive pregnancy test

Ages Eligible for Study
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Minimum Age: 1 Day
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>Gemtuzumab 5 mg / m2 two courses with three week interval | Drug: Gemtuzumab ozogamicin<br>* Two courses of Gemtuzumab vs. no further therapy<br>* Other names: Mylotarg;|
| No Intervention: 2<br>No further therapy | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Event free survival |  | 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival |  | 5 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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AML, Children, Gemtuzumab ozogamicin, Stem cell transplantation