TREATMENT STUDY FOR CHILDREN AND ADOLESCENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA - ICC APL STUDY 01

Conditions: Patients with a clinical diagnosis of initial APL and subsequently confirmed to have PML-RARa, NPM1-RARa or NUMA-RARa fusion.
MedDRA version: 9.1Level: LLTClassification code 10001020Term: Acute promyelocytic leukemia

Registration Number: EUCTR2008-002311-40-IT

Lead Sponsor: A.I.E.O.P. - ASSOCIAZIONE ITALIANA EMATOLOGIA ONCOLOGIA PEDIATRICA

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 300

Inclusion Criteria

- Patients with a clinical diagnosis of initial APL and subsequently confirmed to have PML-RARα, NPM1-RARα or NUMA-RARα fusion. - Less than 21 years of age at initial diagnosis (less than 18 years for italian centers) - Considered suitable for anthracycline-based chemotherapy - Written informed consent available - Females of childbearing age must have a negative pregnancy test and subsequently must attempt to avoid pregnancy
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Patients with a clinical diagnosis of APL but subsequently found to have PLZF-RARα fusion or lacking PML-RAR, NPM-RAR or NuMA-RAR rearrangement should be withdrawn from the study and treated on an alternative protocol. - Refractory/relapsed APL (the guidelines in this protocol for that subgroup are intended for patients treated from initial diagnosis according to this protocol) - Concurrent active malignancy - Pregnant or lactating - Physician and patient/guardian think that intensive chemotherapy is not an appropriate treatment option - Patients who have received alternative chemotherapy for 7 days or longer without ATRA for any reason (either APL not initially suspected or ATRA not available).

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: - to conduct an international pediatric study for APL with optimal outcome and less toxicity - to reduce cumulative anthracycline dosage - to deliver risk stratified treatment based on modified Sanz criteria - to monitor minimal residual disease by RQ-PCR for PML-RAR&#945; and adjust treatment accordingly;Secondary Objective: - To monitor cardiotoxicity by echocardiography;Primary end point(s): - Achievement of molecular complete remission (CRm) and reasons for failure - duration of remission, rates of molecular and frank relapse and deaths in first CR - Overall survival - Toxicity - hematological and non-hematological - Supportive care requirements

Secondary Outcome Measures