Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia
NCT ID: NCT04793919
Last Updated: 2024-04-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
89 participants
INTERVENTIONAL
2019-10-09
2027-10-10
Brief Summary
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Detailed Description
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Based on data coming from adults indicating that at least standard-risk APL patients may be cured without chemotherapy (i.e., with a treatment combining arsenic trioxide (ATO) and ATRA only) 10-12, this ICC APL 02 study was designed with the aim of validating the efficacy of a treatment combining:
* ATO and ATRA in newly diagnosed APL standard-risk (SR) children and adolescents and
* ATO, ATRA and gemtuzumab ozogamicin (GO) in newly diagnosed APL high-risk (HR) children and adolescents.
Following one induction course of treatment combining ATO and ATRA +/- GO depending on risk stratification, patients will receive 4 ATO/ATRA based consolidation blocks. This is the first pediatric trial delivering a non-chemotherapy-based treatment for children with APL, being the whole treatment based on the use of ATRA, ATO (and GO in HR patients). The aim of the study is to demonstrate at least an equivalent efficacy and safety of this treatment not containing cytostatic agents compared to the standard protocols combining ATRA and chemotherapy (i.e. ICC APL Study 01).
The trial is open to all patients with a diagnosis of acute promyelocytic leukemia (APL) who are PCR-positive for the PML-RARα transcript and less than 18 years of age.
This will be an international study, comprising the most important pediatric European groups, expecting to recruit 46 and 43 patients in SR and HR arms, respectively, in 3 years. The duration of study recruitment will be 36 months with a minimum follow-up per patient of 2 years.
The evaluation of morphological CR will be carried out after induction therapy, prior to the first block of consolidation therapy. MRD results after induction will not have an impact on subsequent therapy. By contrast, MRD results after the third consolidation course will influence the subsequent treatment, MRD-positive patients being eligible to rescue treatment, including hematopoietic stem cell transplantation (HSCT). BM aspirates will be repeated after the end of therapy, and 3 months, 6 months, 9 months and 12 months after treatment discontinuation.
This is a collaborative international study in APL in children and adolescents aimed at providing information about procedures for the entry, treatment and follow-up of pediatric patients with APL. It is not intended that this document 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.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Standard Risk (SR)
Patient with APL and WBC less than 10x10e9/L at presentation before start treatment
Arsenic Trioxide
See the protocol
All-trans retinoic acid
See the protocol
High Risk (HR)
Patient with APL, with the highest pre-treatment WBC count equal to or greater than 10x10e9/L at presentation
Mylotarg
See the protocol
Arsenic Trioxide
See the protocol
All-trans retinoic acid
See the protocol
Interventions
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Mylotarg
See the protocol
Arsenic Trioxide
See the protocol
All-trans retinoic acid
See the protocol
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Age \<18 years
* Written informed consent by parents or legal guardians
Exclusion Criteria
* Significant liver dysfunction (bilirubin serum levels \>3 mg/dL, ALT/AST serum levels greater than 5 times the normal values)
* Creatinine serum levels \>2 times the normal value for age
* Significant arrhythmias, EKG abnormalities (\*see below), other cardiac contraindications (L-FEV \<50% or LV-FS \<28%)
* Neuropathy
* Concurrent active malignancy
* Uncontrolled life-threatening infections
* Pregnant or lactating female
* Patients who had received alternative therapy (APL not initially suspected; ATRA and/or ATO not available
18 Years
ALL
No
Sponsors
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Associazione Italiana Ematologia Oncologia Pediatrica
OTHER
Responsible Party
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Principal Investigators
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Fanco Locatelli, Prof
Role: PRINCIPAL_INVESTIGATOR
Dept. of Pediatric Hematology Oncology - Bambino Gesù Children's Hospital Rome
Locations
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Hôpital Universitaire des Enfants Reine Fabiola (Huderf)
Brussels, , Belgium
University Hospital Motol
Prague, , Czechia
Pediatrics and Adolescent Medicine Aarhus University Hospital
Aarhus N, , Denmark
CHU de Bordeaux - Hôpital des Enfants
Bordeaux, , France
Universitätsklinikum Essen (AöR) Zentrum für Kinder-und Jugendmedizin Klinik für Kinderheilkunde III
Essen, , Germany
Our Lady's Children's Hospital Crumlin
Dublin, , Ireland
Rappaport Children'S Hospital, Rambam Health Care Campus
Haifa, , Israel
Ospedale "Casa Sollievo della Sofferenza" - UO Oncoematologia Pediatrica
San Giovanni Rotondo, Foggia, Italy
AOU Policlinico Dipartimento di Pediatria
Bari, , Italy
Ospedale Papa Giovanni XXIII - USS Oncoematologia Pediatrica
Bergamo, , Italy
AOU Policlinico Sant'Orsola-Malpighi - Oncologia ed Ematologia Pediatrica
Bologna, , Italy
Ospedale Pediatrico Microcitemico "A.Cau", Az.Ospedaliera Brotzu - SC Oncoematologia Ped. e Patologia della coagulazione
Cagliari, , Italy
AOU Policlinico Vittorio Emanuele - UOC Ematologia ed Oncologia Pediatrica con TNO
Catania, , Italy
A.O. Universitaria Meyer - DAI Oncoematologia Pediatrica
Florence, , Italy
IRCCS Istituto Gannina Gaslini - Dipartimento di Oncoematologia
Genova, , Italy
Fondazione Monza e Brianza per il Bambino e la sua Mamma (MBBM) - Ospedale San Gerardo
Monza, , Italy
AORN Santobono-Pausilipon
Napoli, , Italy
Univerità degli Studi della Campania- Luigi Vanvitelli - Sevizio di Oncologia Pediatrica
Napoli, , Italy
Azienda Ospedaliera di Padova - Oncoematologia Pediatrica
Padua, , Italy
ARNAS Civico di Cristina e Benfratelli - UOC Oncoematologia Pediatrica
Palermo, , Italy
Fondazione IRCCS Policlinico San Matteo - Oncoematologia Pediatrica
Pavia, , Italy
Ospedale santa Chiara - AOU Pisana, UO Oncoematologia Pediatrica
Pisa, , Italy
Policlinico Umberto I Università "LA Sapienza" - Dip. Biotecnologie cellulari ed ematologia UOS Ematologia Pediatrica
Roma, , Italy
Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica - Ospedale Pediatrico "Bambino Gesù"
Roma, , Italy
AOU Città della Salute e della Scienza di Torino - Presidio Infantile Regina Margherita
Torino, , Italy
VU medisch centrum
Amsterdam, , Netherlands
Centro Hospitalar Universitário de Coimbra - Hospital Pediátrico de Coimbra
Coimbra, , Portugal
Instituto Português de Oncologia de Lisboa Francisco Gentil, EPE
Lisbon, , Portugal
Instituto Português de Oncologia do Porto Francisco Gentil, E. P. E.
Porto, , Portugal
Valencia University Medical School University Hospital La Fe
Valencia, , Spain
Childrens hematology and oncology Uppsala University
Uppsala, , Sweden
Countries
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Central Contacts
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Facility Contacts
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Henrik Hasle, Prof.
Role: primary
Saverio Ladogana, Dr
Role: primary
Massimo Provenzi, Dr
Role: primary
Andrea Pession, Prof
Role: primary
Rosamaria Mura, Dr
Role: primary
Luca Lo Nigro, Dr
Role: primary
Tommaso Casini, Dr
Role: primary
Concetta Micalizzi, Dr
Role: primary
Carmelo Rizzari, Dr
Role: primary
Giuseppe Menna, Dr
Role: primary
Francesca Rossi, Prof
Role: primary
Alessandra Biffi, Prof
Role: primary
Paolo D'Angelo, Dr
Role: primary
Marco Zecca, Dr
Role: primary
Gabriella Casazza, Dr
Role: primary
Anna Maria Testi, Dr
Role: primary
Valentina Cirillo, Dr
Role: primary
Franca Fagioli, Prof
Role: primary
Gertjan Kasper, Prof. dr.
Role: primary
Manuel Brito, Dr.
Role: primary
Ximo Duarte, Dr.
Role: primary
Vítor Costa, Dr.
Role: primary
Other Identifiers
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2017-002383-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ICC APL STUDY 02
Identifier Type: -
Identifier Source: org_study_id
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