Treatment Study for Children and Adolescents With Acute Promyelocitic Leukemia
This study is open to all patients with a diagnosis of acute promyelocytic leukemia (APL) who are PCR positive for the PML-RARα transcript or rarer retinoid sensitive subtypes (i.e. NPM-RAR-alpha, NuMA-RARalpha) and less than 21 years of age (for AIEOP, see appendix A).
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Treatment Study for Children and Adolescents With Acute Promyelocitic Leukemia|
- • to conduct an international pediatric study for APL based on the GIMEMA-AIEOP/AIDA 93 protocol (the study from the Italian GIMEMA -AIEOP group which has produced the best results in children with APL to date), with optimal outcome and less toxicity [ Time Frame: 5 years ] [ Designated as safety issue: No ]• to conduct an international pediatric study for APL based on the GIMEMA-AIEOP/AIDA 93 protocol (the study from the Italian GIMEMA -AIEOP group which has produced the best results in children with APL to date), with optimal outcome and less toxicity
- • To monitor cardiotoxicity by echocardiography [ Time Frame: 5 years ] [ Designated as safety issue: No ]• To monitor cardiotoxicity by echocardiography
|Study Start Date:||January 2009|
|Estimated Study Completion Date:||January 2018|
|Estimated Primary Completion Date:||January 2016 (Final data collection date for primary outcome measure)|
Experimental: standard risk
are defined as those patients with a WBC less than 10x10 9 /L at presentation
see the protocol
Active Comparator: high risk
are defined as those patients whose highest treatment WBC is equal to or greater than 10x10 9 /L at presentation
Drug: ATRA + IDA
see the protocol
This study is open to all patients with a diagnosis of acute promyelocytic leukemia (APL) who are PCR positive for the PML-RARα transcript or rarer retinoid sensitive subtypes (i.e. NPM-RARalpha, NuMA-RARalpha) and less than 21 years of age (for AIEOP, see appendix A). APL is a rare disease with each national group recruiting small numbers of patients to their trials annually. Therefore this will be an international study expecting to recruit 60-70 patients per annum and a total of 300 patients in 5 years. The study aims to limit the use of anthracyclines and stratify treatment by risk group: standard risk - WBC <10 x 109/l : high risk - WBC ≥10 x 109/l. All-trans retinoic acid (ATRA) is included in all phases of therapy and intermediate dose Ara-C (IDARAC) is given during consolidation treatment. Following one induction course of treatment standard risk patients have 2 consolidation blocks whilst high risk patients have 3 consolidation blocks.
The PML-RARα transcript will be monitored throughout and standard risk patients with detectable minimal residual disease by real time quantitative reverse transcriptase polymerase chain reaction (RQ-PCR+) at the end of the second consolidation block will receive a third consolidation block identical to high risk patients. Patients who are RQ-PCR+ for PML-RARα after completion of the third block of consolidation therapy will be candidates for refractory/relapse treatment, but will remain on study. Refractory/relapsed patients who remain RQ-PCR+ for PML-RARα will be candidates for allogeneic bone marrow transplantation (allo-BMT), whilst those who become RQ-PCR- for PML-RARα will have individualised treatment with ongoing MRD monitoring.
These study guidelines are intended to describe a collaborative international study in APL in children and adolescents and to provide information about procedures for the entry, treatment and follow-up of patients. It is not intended that these guidelines be used as an aide-memoir or guide for the treatment of other patients. Every care has been taken in its drafting, but corrections and amendments may be necessary. Before entering patients into the study, clinicians must ensure that the study has received clearance from their Local Research Ethics Committee and any other necessary body.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01226303
|Contact: Annamaria Testi, PI||06.857951 ext +email@example.com|
|Contact: Andrea Pession, Prof||051.-6364443 ext +firstname.lastname@example.org|
|Dipartimento di Biotecnologie Cellulari ed Ematologia||Recruiting|
|Roma, Italy, 00161|
|Contact: Testi, Dr 06.857951 ext +39 email@example.com|
|Principal Investigator: Annamaria Testi, Dr|
|Principal Investigator:||Annamaria Testi, Dr||Associazione Italiana Ematologia Oncologia Pediatrica|