NCT00476541 · CITED IN SOURCE DOCUMENTS

NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase: PHASE3
Status at capture: COMPLETED
Registry last update: 2016-05-13

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.

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What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

No posted result sections were captured. Registered plans do not establish that a treatment works.

Who could take part

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
healthy Volunteers: false
maximum Age: 18 Years
minimum Age: 1 Day
sex: ALL
std Ages: CHILD
ADULT

Treatment arms and interventions

arm Groups

description
Gemtuzumab 5 mg / m2 two courses with three week interval
intervention Names
Drug: Gemtuzumab ozogamicin
label
1
type
EXPERIMENTAL
description
No further therapy
label
2
type
NO_INTERVENTION

interventions

arm Group Labels
1
description
Two courses of Gemtuzumab vs. no further therapy
name
Gemtuzumab ozogamicin
other Names
Mylotarg
type
DRUG

Study design

allocation

RANDOMIZED

intervention Model

PARALLEL

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 250
type: ESTIMATED

Registered outcome plans (not posted results)

primary Outcomes

measure
Event free survival
time Frame
5 years

secondary Outcomes

measure
Overall survival
time Frame
5 years

Full study description

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

Source references

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.
pmid
16304571
type
BACKGROUND
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.
pmid
12846889
type
BACKGROUND
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.
pmid
22730539
type
DERIVED