Clinical trial NCT00476541: NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia
Official title: NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Acute Myeloid Leukemia
Keywords: AML
Children
Gemtuzumab ozogamicin
Stem cell transplantation
Lead sponsor: University of Aarhus
Sponsor class: OTHER
First posted: 2007-05-22
Enrollment: 250
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 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

Eligibility criteria: 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

Primary outcomes:
measure: Event free survival
timeFrame: 5 years
Secondary outcomes:
measure: Overall survival
timeFrame: 5 years
Other outcomes:
Locations:
city: Aarhus
country: Denmark
facility: Department of Pediatrics, Aarhus University Hospital Skejby
city: Copenhagen
country: Denmark
facility: Rigshospitalet
Study officials:
affiliation: Department of Pediatrics, Aarhus University Hospital Skejby, Aarhus, Denmark
name: Henrik Hasle, MD
role: STUDY_CHAIR
References:
citation: Lie SO, Abrahamsson J, Clausen N, Forestier E, Hasle H, Hovi L, Jonmundsson G, Mellander L, Siimes MA, Yssing M, Zeller B, Gustafsson G; Nordic Society of Pediatric Hematology and Oncology (NOPHO); AML Study Group. Long-term results in children with AML: NOPHO-AML Study Group--report of three consecutive trials. Leukemia. 2005 Dec;19(12):2090-100. doi: 10.1038/sj.leu.2403962.
citation: Lie SO, Abrahamsson J, Clausen N, Forestier E, Hasle H, Hovi L, Jonmundsson G, Mellander L, Gustafsson G. Treatment stratification based on initial in vivo response in acute myeloid leukaemia in children without Down's syndrome: results of NOPHO-AML trials. Br J Haematol. 2003 Jul;122(2):217-25. doi: 10.1046/j.1365-2141.2003.04418.x.
citation: Hasle H, Abrahamsson J, Forestier E, Ha SY, Heldrup J, Jahnukainen K, Jonsson OG, Lausen B, Palle J, Zeller B; Nordic Society of Paediatric Haematology and Oncology (NOPHO). Gemtuzumab ozogamicin as postconsolidation therapy does not prevent relapse in children with AML: results from NOPHO-AML 2004. Blood. 2012 Aug 2;120(5):978-84. doi: 10.1182/blood-2012-03-416701. Epub 2012 Jun 22.