ICC APL STUDY 01TREATMENT 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: <Manually entered code. Term in E.1.1>

Registration Number: EUCTR2008-002311-40-NL

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-RARa, NPM1-RARa or NUMA-RARa fusion.
• Less than 21 years of age at initial diagnosis • 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-RARa fusion or lacking PML-RARa, NPM-RARa or NuMARARa
rearrangement should be withdrawn from the study and treated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 based on the<br>GIMEMA-AIEOP/AIDA 93 protocol (the study from the Italian GIMEMA<br>-AIEOP group which has produced the best results in children with<br>APL to date), with optimal outcome and less toxicity<br>• to reduce cumulative anthracycline dosage<br>• to deliver risk stratified treatment based on modified Sanz criteria<br>• to monitor minimal residual disease by RQ-PCR for PML-RARa and<br>adjust treatment accordingly;Secondary Objective: • To monitor cardiotoxicity by echocardiography;Primary end point(s): • Achievement of molecular complete remission (CRm) and reasons for<br>failure<br>• Duration of remission, rates of molecular and frank relapse and deaths<br>in first CR<br>• Overall survival<br>• Toxicity - hematological and non-hematological<br>• Supportive care requirements

Secondary Outcome Measures