Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruitment ended
Participants planned
99
Countries
5
Sites
61
acute promyelocytic leukemia (APL) in children and adolescents
To assess, in an international pediatric study, the efficacy, in terms of event-free survival, of a combination of ATO and ATRA in newly diagnosed SR APL children and adolescents and to explore the safety and efficacy of a combination therapy comprising ATRA/ATO + GO in HR APL.
Key facts
- Sponsor
- Associazione Italiana Ematologia Oncologia Pediatrica
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04]
- Trial duration
- 5 Dec 2024 → ongoing
- Decision date (initial)
- 2024-12-06
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Pfizer S.r.l.
External identifiers
- EU CT number
- 2024-518669-83-00
- EudraCT number
- 2017-002383-40
- ClinicalTrials.gov
- NCT04793919
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Therapy, Safety
To assess, in an international pediatric study, the efficacy, in terms of event-free survival, of a combination of ATO and ATRA in newly diagnosed SR APL children and adolescents and to explore the safety and efficacy of a combination therapy comprising ATRA/ATO + GO in HR APL.
Secondary objectives 6
- To evaluate the short- and long-term toxicity profile of ATO in pediatric patients, when combined with ATRA (SR APL) or ATRA plus GO (HR APL)
- To compare the clearance kinetics of minimal residual disease (MRD) with that of the previous AIDA-like protocols, COG protocol and ICC APL Study 01
- To estimate the cumulative incidence of both molecular and hematological relapse
- To calculate the probability of overall survival and the early death rate
- To prospectively evaluate the impact of FLT3-ITD on this patient population
- To compare the duration of hospitalization and quality of life with those of the previous AIDA-like protocols and ICC APL study 01
Conditions and MedDRA coding
acute promyelocytic leukemia (APL) in children and adolescents
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- Newly diagnosed APL confirmed by the presence of PML/RARα fusion gene
- Age <18 years
- Written informed consent by parents or legal guardians
- If applicable, female participants must have a negative pregnancy test by beta-HCG dosing.
- Patients of child-bearing or child-fathering potential must be willing to adapt their own conduct so as not to procreate during the study participation and must contact their physician to identify the most appropriate approach strategy for this purpose starting from the time of enrolment and for 3 months after receiving the last drug dose
Exclusion criteria 9
- Patients with a clinical diagnosis of APL but subsequently found to lack PML/RARα rearrangement should be withdrawn from the study and treated on an alternative protocol
- 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, ECG abnormalities (ECG abnormalities: Congenital long QT syndrome; History or presence of significant ventricular or atrial tachyarrhythmia; Clinically significant resting bradycardia (<50 beats per minute); QTc >450 msec documented during screening EKG), 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)
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Event-free survival (EFS). This cumulative endpoint includes: no achievement of hematological complete remission after induction therapy; no achievement of molecular remission after three consolidation courses (molecular resistance); relapse (hematological/molecular); death, including early death, at 2 years from diagnosis. We aim at reaching a 3-year EFS probability of 90% (95% CI: 84.1-95.9%) and 80% (95% CI: 72.1-87.9%) in SR and HR patients, respectively
Secondary endpoints 11
- Rate of hematological CR after induction
- Rate of early and aplastic death during induction
- Overall survival (OS)
- Cumulative incidence of either hematological and molecular relapse (CIR)
- Incidence of hematological and non-hematological toxicity
- Kinetics of MRD clearance
- Rate of molecular remission after 3 consolidation cycles
- Assessment of PML/RARα transcript level reduction during treatment
- Toxicity - hematological and non-hematological
- Supportive care requirements
- Total hospitalization days during therapy and health economic impact
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 15
PRD2684476 · Product
- Active substance
- Tretinoin
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL USE
- Max daily dose
- 25 mg/m2 milligram(s)/sq. meter
- Max total dose
- 6720 mg milligram(s)
- Max treatment duration
- 23 Week(s)
- Authorisation status
- Authorised
- ATC code
- L01XF01 — -
- Marketing authorisation
- 44/0662/95-S
- MA holder
- CHEPLAPHARM ARZNEIMITTEL GMBH
- MA country
- Slovakia
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
DEPO-MEDROL 40 mg/1 mL, suspension injectable en flacon
PRD423072 · Product
- Active substance
- Methylprednisolone Acetate
- Pharmaceutical form
- SUSPENSION FOR INJECTION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 10 mg milligram(s)
- Max total dose
- 20 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- H02AB04 — METHYLPREDNISOLONE
- Marketing authorisation
- 34009 302 935 2 2
- MA holder
- PFIZER HOLDING FRANCE
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
PRD2857081 · Product
- Active substance
- Tretinoin
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL USE
- Max daily dose
- 25 mg/m2 milligram(s)/square meter
- Max total dose
- 6720 mg milligram(s)
- Max treatment duration
- 23 Week(s)
- Authorisation status
- Authorised
- ATC code
- L01XF01 — -
- Marketing authorisation
- 34009 365 869 7 0
- MA holder
- CHEPLAPHARM ARZNEIMITTEL GMBH
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SOLU MEDROL 500 mg/7,8 ml polvere e solvente per soluzione iniettabile
PRD452991 · Product
- Active substance
- Methylprednisolone Sodium Succinate
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 10 mg milligram(s)
- Max total dose
- 20 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- H02AB04 — METHYLPREDNISOLONE
- Marketing authorisation
- 023202056
- MA holder
- PFIZER ITALIA S.R.L.
- MA country
- Italy
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Methotrexat Accord 100 mg/ml koncentrát pro infuzní roztok
PRD1776737 · Product
- Active substance
- Methotrexate
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 12 mg milligram(s)
- Max total dose
- 24 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01BA01 — METHOTREXATE
- Marketing authorisation
- 44/359/14-C
- MA holder
- ACCORD HEALTHCARE POLSKA SP. Z O.O.
- MA country
- Czech Republic
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Arsenic trioxide Accord 1 mg/ml concentrate for solution for infusion
PRD7719453 · Product
- Active substance
- Arsenic Trioxide
- Substance synonyms
- ARSENICUM ALBUM, ARSENIOUS TRIOXIDE, ACIDUM ARSENICOSUM
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 0.15 mg/Kg milligram(s)/kilogram
- Max total dose
- 21 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 25 Week(s)
- Authorisation status
- Authorised
- ATC code
- L01XX27 — ARSENIC TRIOXIDE
- Marketing authorisation
- EU/1/19/1398/001
- MA holder
- ACCORD HEALTHCARE S.L.U.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
METHOTREXATE ACCORD 25 mg/ml, solution injectable
PRD4429185 · Product
- Active substance
- Methotrexate
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 12 mg milligram(s)
- Max total dose
- 24 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01BA01 — METHOTREXATE
- Marketing authorisation
- 34009 274 860 7 4
- MA holder
- ACCORD HEALTHCARE FRANCE SAS
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Metotressato Teva 25 mg/ml soluzione iniettabile
PRD734756 · Product
- Active substance
- Methotrexate
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 12 mg milligram(s)
- Max total dose
- 24 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01BA01 — METHOTREXATE
- Marketing authorisation
- 026544027
- MA holder
- TEVA ITALIA S.R.L.
- MA country
- Italy
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
ARACYTIN 100 mg/5 ml Polvere e Solvente per Soluzione Iniettabile
PRD411716 · Product
- Active substance
- Cytarabine
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 30 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01BC01 — CYTARABINE
- Marketing authorisation
- 022391015
- MA holder
- PFIZER ITALIA S.R.L.
- MA country
- Italy
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Cytarabine Accord 100 mg/ml injekční/infuzní roztok
PRD1957526 · Product
- Active substance
- Cytarabine
- Pharmaceutical form
- SOLUTION FOR INJECTION/INFUSION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 30 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01BC01 — CYTARABINE
- Marketing authorisation
- 44/409/13-C
- MA holder
- ACCORD HEALTHCARE POLSKA SP. Z O.O.
- MA country
- Czech Republic
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
DEPO-MEDROL 40 mg/ml injekční suspenze
PRD468132 · Product
- Active substance
- Methylprednisolone Acetate
- Pharmaceutical form
- SUSPENSION FOR INJECTION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 10 mg milligram(s)
- Max total dose
- 20 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- H02AB04 — METHYLPREDNISOLONE
- Marketing authorisation
- 56/191/71-C
- MA holder
- PFIZER SPOL. S R.O.
- MA country
- Czech Republic
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
TRISENOX 1 mg/ml concentrate for solution for infusion
PRD7331849 · Product
- Active substance
- Arsenic Trioxide
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 0.15 mg/Kg milligram(s)/kilogram
- Max total dose
- 21 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 25 Week(s)
- Authorisation status
- Authorised
- ATC code
- L01XX27 — ARSENIC TRIOXIDE
- Marketing authorisation
- EU/1/02/204/001
- MA holder
- TEVA B.V
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
MYLOTARG 5 mg powder for concentrate for solution for infusion
PRD6503065 · Product
- Active substance
- Gemtuzumab Ozogamicin
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 3 mg/m2 milligram(s)/square meter
- Max total dose
- 10 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01FX02 — -
- Marketing authorisation
- EU/1/18/1277/001
- MA holder
- PFIZER EUROPE MA EEIG
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
CYTARABINE ACCORD 20 mg/mL, solution injectable/pour perfusion
PRD6492277 · Product
- Active substance
- Cytarabine
- Pharmaceutical form
- SOLUTION FOR INJECTION/INFUSION
- Route of administration
- INTRATHECAL USE
- Max daily dose
- 30 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 2 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01BC01 — CYTARABINE
- Marketing authorisation
- 34009 550 579 6 3
- MA holder
- ACCORD HEALTHCARE FRANCE SAS
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
PRD2857077 · Product
- Active substance
- Tretinoin
- Substance synonyms
- RETINOIC ACID, ALL-TRANS-RETINOIC ACID, TRANS-RETINOIC ACID
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL USE
- Max daily dose
- 25 mg/m2 milligram(s)/sq. meter
- Max total dose
- 6720 mg/g milligram(s)/gram
- Max treatment duration
- 23 Week(s)
- Authorisation status
- Authorised
- ATC code
- L01XF01 — -
- Marketing authorisation
- 029838024
- MA holder
- CHEPLAPHARM ARZNEIMITTEL GMBH
- MA country
- Italy
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Associazione Italiana Ematologia Oncologia Pediatrica
- Sponsor organisation
- Associazione Italiana Ematologia Oncologia Pediatrica
- Address
- Via Giuseppe Massarenti 11
- City
- Bologna
- Postcode
- 40138
- Country
- Italy
Scientific contact point
- Organisation
- Associazione Italiana Ematologia Oncologia Pediatrica
- Contact name
- Arcangelo Prete
Public contact point
- Organisation
- Associazione Italiana Ematologia Oncologia Pediatrica
- Contact name
- Arcangelo Prete
Third parties 3
| Organisation | City, country | Duties |
|---|---|---|
| Julius Clinical International B.V. ORG-100028683
|
Zeist, Netherlands | On site monitoring |
| Oriola Sweden AB ORG-100011733
|
Molnlycke, Sweden | Other |
| Ospedale Pediatrico Bambino Gesu ORG-100009738
|
Rome, Italy | On site monitoring, Code 10, Code 11, Code 13, Code 14, Code 2, Laboratory analysis, Code 5, Data management, Code 8 |
Locations
5 EU/EEA countries · 61 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Czechia | Ongoing, recruitment ended | 3 | 2 |
| France | Ongoing, recruitment ended | 31 | 28 |
| Italy | Ongoing, recruitment ended | 60 | 24 |
| Netherlands | Ongoing, recruitment ended | 3 | 1 |
| Sweden | Authorised, recruiting | 2 | 6 |
| Rest of world | — | 0 | — |
Investigational sites
Fakultni Nemocnice Brno
Klinika dětské onkologie/Department of Pediatric Oncology, Cernopolni 9, Cerna Pole, Brno
Fakultni Nemocnice V Motole
Klinika dětské hematologie a onkologie/Department of Pediatric Hematology and Oncology, V Uvalu 84/1, Motol, Prague
Centre Hospitalier Universitaire De Nice
Service d’onco-hématologie pédiatrique, 151 Route De Saint Antoine, 06200, Nice
Centre Hospitalier Universitaire De Rennes
Unité d’hémato-oncologie pédiatrique, 16 Boulevard De Bulgarie, Bp 90349, Rennes
Centre Hospitalier Et Universitaire De Limoges
Service d’hématologie et d’oncologie pédiatrique, 2 Avenue Martin Luther King, 87042, Limoges Cedex 1
Centre Hospitalier Universitaire De Saint Etienne
Service d’hématologie et de cancérologie pédiatrique, Avenue Albert Raimond, 42270, Saint Priest En Jarez
Assistance Publique Hopitaux De Paris
Service d’hématologie et oncologie pédiatrique, 26 Avenue Du Docteur Arnold Netter, 75012, Paris
Hospices Civils De Lyon
Département d'hématologie pédiatrique, 1 Place Professeur Joseph Renaut, 69008, Lyon
CHU Besancon
Service d’onco-hématologie pédiatrique, 3 Boulevard Alexander Fleming, Cs 81816, Besancon Cedex
Les Hopitaux Universitaires De Strasbourg
Service d’hématologie et oncologie pédiatrique, 1 Avenue Moliere, Bp 49, Strasbourg Cedex 2
Centre Hospitalier Universitaire De Poitiers
Service médico-chirurgical de pédiatrie, 2 Rue De La Miletrie, 86000, Poitiers
Centre Hospitalier Regional Universitaire De Tours
Service d’oncologie pédiatrique, 49 Boulevard Beranger, 37000, Tours
Centre Hospitalier Universitaire De Caen Normandie
Service d’onco-hématologie, Avenue De La Cote De Nacre, Cs 30001, Caen Cedex 9
Centre Hospitalier Universitaire De Lille
Unité d’hématologie pédiatrique, Avenue Eugene Avinee, 59037, Lille Cedex
Centre Hospitalier Universitaire De Dijon
Service d’onco-hématologie pédiatrique, 14 Rue Paul Gaffarel, 21000, Dijon
CHRU De Nancy
Service d’hémato-oncologie pédiatrique, 11 Rue Du Morvan, Bp 80001, Vandoeuvre Les Nancy Cedex
University Hospital Of Clermont-Ferrand
Service d’onco-hématologie pédiatrique, 1 Place Lucie Et Raymond Aubrac, 63100, Clermont-Ferrand
Centre Hospitalier Universitaire De Toulouse
Service d’hémato-immuno-oncologie pédiatrique, 330 Avenue De Grande Bretagne, 31059, Toulouse Cedex 9
Centre Hospitalier Universitaire De Nantes
Service d’oncologie et hématologie pédiatrique, 9 Quai Moncousu, 44093, Nantes Cedex 1
Centre Hospitalier Universitaire Rouen
Service d’immuno-hémato-oncologie pédiatrique, 1 Rue De Germont, Bp 96031, Rouen Cedex
Centre Hospitalier Regional Et Universitaire De Brest
Service Hémato-Oncologie Pédiatrique, 2 Avenue Marechal Foch, 29200, Brest
Centre Hospitalier Universitaire De Montpellier
Service d’onco-hématologie pédiatrique, 371 Avenue Du Doyen Gaston Giraud, 34090, Montpellier
Centre Hospitalier Universitaire De Bordeaux
Unité d'hématologie et oncologie pédiatrique, Place Amelie Raba Leon, 33000, Bordeaux
Centre Hospitalier Universitaire Grenoble Alpes
Service immuno-hématologie et oncologie pédiatrique, Quai Yermoloff, 38700, La Tronche
Assistance Publique Hopitaux De Paris
Service d’hématologie et immunologie pédiatrique, 48 Boulevard Serurier, 75019, Paris
Centre Hospitalier Regional De Marseille
Service d’hématologie et oncologie pédiatrique, 264 Rue Saint Pierre, 13005, Marseille
Centre Hospitalier Universitaire D'Angers
Service d’onco-hématologie et immunologie pédiatrique, 4 Rue Larrey, 49100, Angers
Centre Hospitalier Universitaire De La Reunion
Service d'oncologie et d'hématologie pédiatrique, Allee Des Topazes, Cs 11021, Saint-Denis
Centre Hospitalier Universitaire Reims
Service d’hémato-oncologie pédiatrique, 45 Rue Cognacq Jay, 51100, Reims
Centre Hospitalier Universitaire Amiens Picardie
Service d’onco-hématologie et immunologie pédiatrique, 1 Rond Point Du Pr Christian Cabrol, 80054, Amiens Cedex 1
Azienda Ospedaliera-Universitaria Di Cosenza
U.O.C. Pediatria, Via Felice Migliori 1, 87100, Cosenza
Azienda Unita Sanitaria Locale Della Romagna
SSD Oncoematologia Pediatrica, Viale Luigi Settembrini 2, 47923, Rimini
Grande Ospedale Metropolitano Bianchi Melacrino Morelli
UOSD Oncoematologia Pediatrica, Viale Europa, 89133, Reggio Calabria
Azienda Ospedaliera di Padova
U.O.C. Oncoematologia Pediatrica, Via Nicolo' Giustiniani 2, 35128, Padova
Azienda Ospedaliera Universitaria Integrata Verona
U.O.C. Oncoematologia Pediatrica, Piazzale Aristide Stefani 1, 37126, Verona
ARNAS G. Brotzu
SC Oncoematologia Pediatrica, Piazzale Alessandro Ricchi 1, 09121, Cagliari
Azienda Ospedaliero Universitaria Renato Dulbecco
SOC Ematoncologia Pediatrica, Viale Tommaso Campanella 115, 88100, Catanzaro
Casa Sollievo Della Sofferenza
Oncoematologia Pediatrica, Viale Convento Cappuccini 1, 71013, San Giovanni Rotondo
ARNAS Civico Di Cristina Benfratelli
U.O.C Oncoematologia Pediatrica, Piazza Nicola Leotta 4, 90127, Palermo
IRCCS Istituto Giannina Gaslini
UOC EMATOLOGIA, Via Gerolamo Gaslini 5, 16147, Genoa
Fondazione IRCCS Policlinico San Matteo
Oncoematologia Pediatrica, Viale Camillo Golgi 19, 27100, Pavia
Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii
UOS Oncologia Pediatrica, Piazza Oms 1, 24127, Bergamo
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania
Ematologia ed Oncologia Pediatrica, Via Santa Sofia 78, 95123, Catania
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Oncoematologia Pediatrica, Piazzale Spedali Civili 1, 25123, Brescia
Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
DAI Materno Infantile e di Nutrizione Clinica, Via Santa Maria Di Costantinopoli 104, 80138, Naples
Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
SOC Emato-Oncologia e Centro Trapianti, Via Dell' Istria 65/1, 34137, Trieste
Azienda Ospedaliera Universitaria Meyer IRCCS
Dipartimento di Oncologia ed Ematologia Pediatrica, Viale Gaetano Pieraccini 24, 50139, Florence
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
SC Oncoematologica Pediatrica, Piazza Polonia 94, 10126, Turin
Fondazione IRCCS San Gerardo Dei Tintori
Emato-Oncologia Pediatrica, Via Giovanni Battista Pergolesi 33, 20900, Monza
Azienda Ospedaliero Universitaria Delle Marche
SOSD Oncoematologia Pediatrica, Via Conca 71, 60126, Ancona
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Oncologia ed Ematologia Pediatrica, Via Pietro Albertoni 15, 40138, Bologna
Azienda Ospedaliero Universitaria Pisana
UO Oncoematologia pediatrica, Via Roma 67, 56126, Pisa
Azienda Ospedaliera Santobono Pausilipon
Oncologia, Ematologia e Terapie Cellulari, Via Posillipo 226, 80123, Naples
Ospedale Pediatrico Bambino Gesu
Area Studi Clinici Oncoematologici e Terapie Cellulari, Piazza Di Sant'onofrio 4, 00165, Rome
Prinses Maxima Centrum voor Kinderoncologie B.V.
Trial and data center, Heidelberglaan 25, 3584 CS, Utrecht
Karolinska University Hospital
Children’s ward, level 12, Astrid Lindgrens Barnsjukhus, Eugeniavagen 3, 171 64, Solna
Uppsala University Hospital
Department of Pediatric hemtology and oncology 95A, Akademiska Barnsjukhuset, Akademiska Sjukhuset, 751 85, Uppsala
Region Skane Skanes Universitetssjukhus
Childhood Cancer Centrum Ward 64, Skånes universitetssjukhus, Entregatan 7, 222 42, Lund
Region Oestergoetland
Ward B153 BOND, HKH Kronprinsessan Victorias barn- och ungdomssjukhus, Universitetssjukhuset I, 58185, Linkoping
Queen Silvia Childrens Hospital - Sahlgrenska University Hospital - Vaestra Goetalandsregionen
Childhood Cancer Centrum Ward 1, Drottning Silvias barnsjukhus, Behandlingsvagen 7, Harlanda, Gothenburg
Region Vaesterbotten
Department of Pediatrics Ward 3, Norrlands Universitetssjukhus, Koksvagen 11, Alidhem, Umea
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Czechia | 2024-12-06 | 2024-12-06 | 2025-10-09 | ||
| France | 2024-12-06 | 2024-12-06 | 2025-10-09 | ||
| Italy | 2025-01-21 | 2025-01-21 | 2025-10-09 | ||
| Netherlands | 2024-12-09 | 2024-12-09 | 2025-10-09 | ||
| Sweden | 2024-12-05 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 74 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-518669-83-00 | 4.0 |
| Protocol (for publication) | D2_Protocol modification 1_FR_2024-518669-83-00 | 1 |
| Protocol (for publication) | D2_Protocol modification 1_NL_2024-518669-83-00 | 1 |
| Protocol (for publication) | D2_Protocol modification 1_SE_2024-518669-83-00 | 1.1 |
| Protocol (for publication) | D2_Protocol modification 2_FR_2024-518669-83-00 | 1 |
| Protocol (for publication) | D2_Protocol modification 3_FR_2024-518669-83-00 | 2.0 |
| Protocol (for publication) | D2_Protocol modification 4_FR_2024-518669-83-00 | 2.0 |
| Recruitment arrangements (for publication) | APL02_Blank_Document_Transition | 1 |
| Recruitment arrangements (for publication) | APL02_Blank_Document_Transition | 1 |
| Recruitment arrangements (for publication) | APL02_Blank_Document_Transition | 1 |
| Recruitment arrangements (for publication) | ICC APL STUDY 02_Blank_Document_Transition | 1 |
| Recruitment arrangements (for publication) | ICC APL STUDY 02_Blank_Document_Transition | 1 |
| Subject information and informed consent form (for publication) | L1_ICF Sweden | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 12-17 yr_HR_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 12-17 yr_SR_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 6-11 yr_HR | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 6-11 yr_SR | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF adults_HR_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF adults_SR_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech 12-14 yr HR | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech 12-14 yr SR | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech 15-17 yr HR | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech 15-17 yr SR | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech 8-11 yr HR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech 8-11 yr SR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech adults HR | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech adults SR | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech parents HR | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech parents SR | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech privacy over18 yr_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Czech privacy parents_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France 13-17 yr_HR | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France 13-17 yr_SR | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France 4-7 yr_HR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France 4-7 yr_SR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France 8-12 yr_HR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France 8-12 yr_SR | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France adults_HR | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France adults_SR | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France Parents_HR | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF France Parents_SR | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Netherlands 12-16_HR_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Netherlands 12-16_SR_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Netherlands over 16 yr_HR_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Netherlands over 16 yr_SR_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Netherlands parents_HR_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Netherlands parents_SR_Redacted | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents_HR_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents_SR_redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF privacy adults_HR_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF privacy adults_SR_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF privacy parents_HR_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF privacy parents_SR_redacted | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS Sweden_children | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS Sweden_parents and over 18 | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS Sweden_young people | 1.1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Aracytin | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC CZ arsenic-trioxide | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC CZ cytarabine | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC CZ methotrexate | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC CZ methylprednisolon | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC CZ mylotarg | na |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC CZ vesanoid | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Methotrexate | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Mylotarg | na |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Solu Medrol | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Trisenox | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Vesanoid | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_FR_CYTARABINE | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_FR_DEPO-MEDROL | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_FR_METHOTREXATE | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_FR_MYLOTARG_GO | na |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_FR_TRISENOX_ATO | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_FR_VESANOID_ATRA | 1 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-15 | Italy | Acceptable 2024-12-04
|
2024-12-05 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-10-01 | Acceptable 2024-12-04
|
2025-10-01 | |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-04-29 | Italy | Acceptable 2024-12-04
|
2026-04-29 |