NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia
Study Details
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
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
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: 1 Gemtuzumab 5 mg / m2 two courses with three week interval |
Drug: Gemtuzumab ozogamicin
Two courses of Gemtuzumab vs. no further therapy
Other Names:
|
| No Intervention: 2 No further therapy |
Outcome Measures
Primary Outcome Measures
- Event free survival [5 years]
Secondary Outcome Measures
- Overall survival [5 years]
Eligibility Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Department of Pediatrics, Aarhus University Hospital Skejby | Aarhus | Denmark | 8200 | |
| 2 | Rigshospitalet | Copenhagen | Denmark | 2100 |
Sponsors and Collaborators
- University of Aarhus
Investigators
- Study Chair: Henrik Hasle, MD, Department of Pediatrics, Aarhus University Hospital Skejby, Aarhus, Denmark
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
- 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.
- 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.
- NOPHO-AML 2004