Clinical Trials Register

Summary
EudraCT Number:	2017-002383-40
Sponsor's Protocol Code Number:	ICC_APL_Study_02
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2020-11-25
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

France - ANSM

A.2

EudraCT number

2017-002383-40

A.3

Full title of the trial

Treatment study for children and adolescents with Acute Promyelocytic Leukemia

Étude du traitement des enfants et des adolescents atteints de leucémie aiguë promyélocytaire : Étude multicentrique internationale associant le trioxyde d'arsenic (ATO) et l'acide rétinoïque tout trans (ATRA) +/- le Gemtuzumab Ozogamicine (GO) pour les patients atteints de leucémie aiguë promyélocytaire de novo

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Treatment study for children and adolescents with Acute Promyelocytic Leukemia

Étude sur le traitement de la leucémie promyélocytaire aiguë chez des enfants et des adolescents

A.3.2

Name or abbreviated title of the trial where available

ICC APL Study 02

A.4.1

Sponsor's protocol code number

ICC_APL_Study_02

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	AIEOP- Associazione Italiana Ematologia Oncologia Pediatrica
B.1.3.4	Country	Italy
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	AIEOP
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	AIEOP
B.5.2	Functional name of contact point	Centro Operativo
B.5.3	Address:
B.5.3.1	Street Address	Via Massarenti 11
B.5.3.2	Town/ city	Bologna
B.5.3.3	Post code	40138
B.5.3.4	Country	Italy
B.5.4	Telephone number	+390512144667
B.5.5	Fax number	+39051345759
B.5.6	E-mail	studiclinici@aieop.org

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.2	Country which granted the Marketing Authorisation	France
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Cytarabine
D.3.4	Pharmaceutical form	Concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intrathecal use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Cytarabine
D.3.9.1	CAS number	147-94-4
D.3.9.2	Current sponsor code	IMP1
D.3.9.3	Other descriptive name	CYTARABINE
D.3.9.4	EV Substance Code	SUB06880MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	up to
D.3.10.3	Concentration number	20
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.2	Country which granted the Marketing Authorisation	France
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Tretinoïne
D.3.4	Pharmaceutical form	Capsule
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Tretinoïne
D.3.9.1	CAS number	302-79-4
D.3.9.2	Current sponsor code	IMP2
D.3.9.3	Other descriptive name	TRETINOIN
D.3.9.4	EV Substance Code	SUB11246MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	up to
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.2	Country which granted the Marketing Authorisation	France
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Méthotrexate
D.3.4	Pharmaceutical form	Powder for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intrathecal use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Méthotrexate
D.3.9.1	CAS number	59-05-2
D.3.9.2	Current sponsor code	IMP3
D.3.9.3	Other descriptive name	METHOTREXATE
D.3.9.4	EV Substance Code	SUB08856MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	up to
D.3.10.3	Concentration number	25
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 4
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.2	Country which granted the Marketing Authorisation	France
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Trioxyde d'arsenic
D.3.4	Pharmaceutical form	Powder for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Trioxyde d'arsenic
D.3.9.1	CAS number	1327-53-3
D.3.9.2	Current sponsor code	IMP4
D.3.9.3	Other descriptive name	ARSENIC TRIOXIDE
D.3.9.4	EV Substance Code	SUB12467MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/l milligram(s)/litre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 5
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Mylotarg
D.2.1.1.2	Name of the Marketing Authorisation holder	Pfizer Ltd
D.2.1.2	Country which granted the Marketing Authorisation	France
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/00/005-EMEA/OD/022/00
D.3 Description of the IMP
D.3.4	Pharmaceutical form	Powder for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	GEMTUZUMAB OZOGAMICINE
D.3.9.1	CAS number	220578-59-6
D.3.9.3	Other descriptive name	Gentuzumab ozogamicin
D.3.9.4	EV Substance Code	SUB20794
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 6
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.2	Country which granted the Marketing Authorisation	France
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Méthilprednisolone
D.3.4	Pharmaceutical form	Concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intrathecal use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Méthylprednisolone
D.3.9.1	CAS number	83-43-2
D.3.9.3	Other descriptive name	METHYLPREDNISOLONE
D.3.9.4	EV Substance Code	SUB08872MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	up to
D.3.10.3	Concentration number	40
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

acute promyelocytic leukemia (APL) in children and adolescents

leucémie aiguë promyélocytaire (LAP) des enfants et adolescents

E.1.1.1

Medical condition in easily understood language

acute promyelocytic leukemia (APL) in children and adolescents

leucémie aiguë promyélocytaire (LAP) des enfants et adolescents

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

• 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.

Evaluer l'efficacité en termes de survie sans événement d'un traitement associant le trioxyde de diarsenic (ATO) et l'acide tout-trans rétinoïque (ATRA) pour les patients de risque standard ainsi que la sécurité et l’efficacité de l’ATRA et l’ATO associés au gemtuzumab ozogamicine (GO) pour les patients de haut risque.

E.2.2

Secondary objectives of the trial

• 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

• Évaluer le profil de toxicité à court et à long terme de l’ATO chez les patients pédiatriques en association avec l’ATRA (SR LAP) ou ATRA plus GO ( HR LAP)
• Comparer la cinétique de clairance de la maladie résiduelle minimale (MRD) avec celle des protocoles antérieurs : protocole AIDA, protocole COG et de l'étude ICC APL 01.
• estimer l'incidence cumulative des rechutes moléculaires et hématologiques
• calculer la probabilité de survie globale et le taux de mortalité précoce
• évaluer de manière prospective l’impact de FLT3-ITD sur cette population de patients
• Comparer la durée d'hospitalisation et la qualité de vie avec les protocoles antérieurs (AIDA et l'étude ICC APL 01)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Newly diagnosed APL confirmed by the presence of PML/RARα fusion gene
- Age <18 years
- Written informed consent by parents or legal guardians
- WBC at diagnosis ≥10 x 109/L
- If applicable, female participants must have pregnancy test by beta-HCG dosing and be negative.
- Patients of child-bearing or child-fathering potential must be willing to practice and must contact their physician. With their physician, they must agree on the most appropriate approach for birth control from the time of enrollment in this study and for 3 months after receiving the latest infusion.

- LPA nouvellement diagnostiquée confirmée par la présence du gène de fusion PML / RARα
- Age < 18 ans
- Consentement éclairé écrit des parents ou tuteurs légaux et assentiment de l’enfant
- Test sérique bêta-HCG négatif chez les participantes en âge de procréer.
- Les patients en âge ou potentiellement en âge de procréer doivent convenir avec leur médecin de la méthode de contraception la plus appropriée à compter du moment de leur participation à cette étude et jusqu’à trois mois après la dernière perfusion.

E.4

Principal exclusion criteria

Patients with a clinical diagnosis of APL but subsequently found to lack PML/RARα rearrangement should be withdrawn from the study and treated with 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, EKG abnormalities (*see below), other cardiac contraindications (L-FEV < 50% or LV-FS <28%)
- Neuropathy grade 2 or greater
- Concurrent active malignancy
- Uncontrolled life-threatening infections
- Pregnant or lactating females
- Patients who had received alternative therapy (APL not initially suspected; ATRA and/or ATO not available)

- Les patients ayant reçu un diagnostic clinique de LPA mais dont on a constaté ultérieurement l'absence de réarrangement de la PML / RARα doivent être retirés de l'étude et traités selon un protocole alternatif.
- Dysfonctionnement hépatique important (taux sériques de bilirubine> 3 mg / dL, taux sériques d'ALT / AST > 5 LNS)
- Taux sériques de créatinine> 2 fois la valeur normale pour l'âge
- Arythmies importantes, anomalies ECG (* voir ci-dessous), autres contre-indications cardiaques (VEMS <50% ou VFS <28%)
- Neuropathie ≥ grade 2
- Tumeur maligne concurrente
- Infections non contrôlées menaçant le pronostic vital
- Participante enceinte ou allaitantes
- Patients ayant reçu un traitement alternatif (LAP non initialement suspectée; ATRA et / ou ATO non disponible)
* Anomalies ECG:
Syndrome du QT long congénital
Antécédents ou présence de tachyarythmie ventriculaire ou auriculaire importante
Bradycardie au repos cliniquement significative (<50 bpm)
QTc> 450 msec documenté lors du dépistage ECG

E.5 End points

E.5.1

Primary end point(s)

• The primary endpoint of the study is event-free survival (EFS). This cumulative endpoint includes the following events: 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.

• Le critère d'évaluation principal de l'étude est la survie sans événement (SSE). Ce critère d'évaluation cumulatif comprend les événements suivants: aucune réalisation de rémission hématologique complète après le traitement d'induction; aucune réalisation de rémission moléculaire après trois cours de consolidation (résistance moléculaire); rechute (hématologique / moléculaire); décès, y compris un décès prématuré, à 2 ans du diagnostic. Nous visons à atteindre une probabilité de SSE à 3 ans de 90% (IC 95%: 84,1-95,9%) et 80% (IC 95%: 72,1-87,9%) chez les patients SR et HR, respectivement.

E.5.1.1

Timepoint(s) of evaluation of this end point

2 years

2 ans

E.5.2

Secondary end point(s)

• 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

• Taux de RC hématologique après induction
• Taux de mortalité précoce et aplasique lors de l’induction
• Survie globale (OS)
• Incidence cumulative de rechute hématologique et moléculaire (CIR)
• Incidence de la toxicité hématologique et non hématologique
• Cinétique de la clairance MRD
• Taux de rémission moléculaire après 3 cycles de consolidation
• Évaluation de la réduction du niveau de transcription PML / RARα pendant le traitement
• Toxicité - hématologique et non hématologique
• Exigences en matière de soins de soutien
• Nombre total de jours d’hospitalisation pendant la thérapie et impact économique sur la santé

E.5.2.1

Timepoint(s) of evaluation of this end point

2 years

2 ans

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

Yes

F.1.1.2.1

Number of subjects for this age range:

F.1.1.3

Newborns (0-27 days)

Yes

F.1.1.3.1

Number of subjects for this age range:

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

Yes

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Children

Enfants

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

NONE

Nessuno

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2021-01-08

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-02-02

P. End of Trial
P.	End of Trial Status	Ongoing

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