NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia

Phase 3

Completed

• Conditions: Acute Myeloid Leukemia
• Interventions: Drug: Gemtuzumab ozogamicin

• Registration Number: NCT00476541
• Lead Sponsor: University of Aarhus

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

Study Timeline

• Study Start: 2004-01
• Study First Submitted: 2007-05-21
• Study First Submitted QC: 2007-05-21
• Study First Posted: 2007-05-22
• Primary Completion: 2012-01
• Study Completion: 2012-01
• Status Verified: 2015-05
• Last Update Submitted: 2016-05-12
• Last Update Posted: 2016-05-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 250

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1	Gemtuzumab ozogamicin	Gemtuzumab 5 mg / m2 two courses with three week interval

Primary Outcome Measures

Name	Time	Method
Event free survival	5 years

Secondary Outcome Measures

Name	Time	Method
Overall survival	5 years

Trial Locations

Locations (2)

Department of Pediatrics, Aarhus University Hospital Skejby; 🇩🇰 Aarhus, Denmark

Rigshospitalet; 🇩🇰 Copenhagen, Denmark