NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia

University of Aarhus

Status and phase: Completed; Phase 3

Conditions: Acute Myeloid Leukemia

Treatments: Drug: Gemtuzumab ozogamicin

Study type: Interventional

Funder types: Other

Identifiers

NCT00476541

NOPHO-AML 2004

Details and patient eligibility

About

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.

Full 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

Enrollment: 250 estimated patients

Sex: All

Ages: 1 day to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

250 participants in 2 patient groups

1

Experimental group

Description:

Gemtuzumab 5 mg / m2 two courses with three week interval

Treatment:

Drug: Gemtuzumab ozogamicin

2

No Intervention group

Description:

No further therapy