Clinical Trials Register

Summary
EudraCT Number:	2022-001363-27
Sponsor's Protocol Code Number:	DAY101-002
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2023-01-16
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

Spain - AEMPS

A.2

EudraCT number

2022-001363-27

A.3

Full title of the trial

LOGGIC/FIREFLY-2: A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients with Pediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy

LOGGIC/FIREFLY-2: Ensayo en fase III, aleatorizado, internacional y multicéntrico de DAY101 en monoterapia frente a la quimioterapia estándar en pacientes con glioma pediátrico de bajo grado que presentan una alteración de RAF activadora que requiere tratamiento sistémico de primera línea

A.3.1

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

LOGGIC/FIREFLY-2: DAY101 vs. Standard of Care Chemotherapy in Pediatric Patients with Low-Grade Glioma Requiring First-Line Systemic Therapy

LOGGIC/FIREFLY-2: DAY101 frente a la quimioterapia estándar en pacientes pediátricos con glioma de bajo grado que requiere tratamiento sistémico de primera línea.

A.3.2

Name or abbreviated title of the trial where available

LOGGIC/FIREFLY-2

A.4.1

Sponsor's protocol code number

DAY101-002

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT05566795

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/500/2020

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Day One Biopharmaceuticals, Inc. (Day One)
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Day One Biopharmaceuticals, Inc. (Day One)
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Day One Biopharmaceuticals, Inc. (Day One)
B.5.2	Functional name of contact point	FIREFLY-2 Clinical Trial Team
B.5.3	Address:
B.5.3.1	Street Address	2000 Sierra Point Parkway
B.5.3.2	Town/ city	Suite 501, Brisbane, CA
B.5.3.3	Post code	94005
B.5.3.4	Country	United States
B.5.6	E-mail	FIREFLY-2@dayonebio.com

D. IMP Identification
D.IMP: 1
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	No
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/3/21/2434
D.3 Description of the IMP
D.3.1	Product name	DAY101
D.3.2	Product code	DAY101
D.3.4	Pharmaceutical form	Tablet
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	tovorafenib
D.3.9.1	CAS number	1096708-71-2
D.3.9.2	Current sponsor code	DAY101
D.3.9.3	Other descriptive name	MLN2480, TAK-580, BSK1369, BIIB024
D.3.9.4	EV Substance Code	SUB197662
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	100
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	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	No
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/3/21/2434
D.3 Description of the IMP
D.3.1	Product name	DAY101
D.3.2	Product code	DAY101
D.3.4	Pharmaceutical form	Powder for oral suspension
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Gastroenteral use Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	tovorafenib
D.3.9.1	CAS number	1096708-71-2
D.3.9.2	Current sponsor code	DAY101
D.3.9.3	Other descriptive name	MLN2480, TAK-580, BSK1369, BIIB024
D.3.9.4	EV Substance Code	SUB197662
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	300
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.2.1.1.1	Trade name	Cellcristin®
D.2.1.1.2	Name of the Marketing Authorisation holder	STADAPHARM GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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	Vincristine
D.3.4	Pharmaceutical form	Solution for injection
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	Vincristine sulfate
D.3.9.3	Other descriptive name	Vincristine sulfate
D.3.9.4	EV Substance Code	SUB05101MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
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: 4
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.2.1.1.1	Trade name	Ribocarbo®-L
D.2.1.1.2	Name of the Marketing Authorisation holder	Hikma Farmacêutica (Portugal), S.A.
D.2.1.2	Country which granted the Marketing Authorisation	Portugal
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	Carboplatin
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	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Carboplatin
D.3.9.1	CAS number	41575-94-4
D.3.9.4	EV Substance Code	SUB06614MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
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: 5
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.2.1.1.1	Trade name	Vinblastine STADA
D.2.1.1.2	Name of the Marketing Authorisation holder	STADAPHARM GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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	Vinblastine
D.3.4	Pharmaceutical form	Powder for solution for injection
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	Vinblastine sulfate
D.3.9.1	CAS number	143-67-9
D.3.9.4	EV Substance Code	SUB05098MIG
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	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.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

Pediatric low-grade glioma harboring an activating RAF alteration requiring first-line systemic therapy

Glioma pediátrico de bajo grado que presenta una alteración de RAF activadora que requiere tratamiento sistémico de primera línea

E.1.1.1

Medical condition in easily understood language

Brain tumor in children and young adults, requiring treatment for the first time

Tumor cerebral en niños y adultos jóvenes que requieren tratamiento por primera vez

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10065443
E.1.2	Term	Malignant glioma
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

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 compare the ORR assessed per RANO criteria by independent review committee (IRC) of DAY101 monotherapy versus SoC chemotherapy in patients with pediatric low-grade glioma harboring an activating RAF alteration requiring front-line systemic therapy.

Comparar la tasa de respuesta objetiva (TRO) evaluada según los criterios RANO por el comité de revisión independiente (CRI) de DAY101 en monoterapia frente a la quimioterapia estándar en pacientes con glioma pediátrico de bajo grado que presentan una alteración de RAF activadora que requiere tratamiento sistémico de primera línea.

E.2.2

Secondary objectives of the trial

KEY SECONDARY OBJECTIVES:
- To compare the PFS assessed by IRC of DAY101 monotherapy versus SoC chemotherapy per RANO criteria.
- To compare the duration of response (DOR) assessed by IRC of DAY101 monotherapy versus SoC chemotherapy per RANO criteria
- To compare the OS of DAY101 monotherapy versus SoC chemotherapy.

OBJETIVOS SECUNDARIOS CLAVE:
- Comparar la supervivencia sin progresión (SSP) con DAY101 en monoterapia frente a la quimioterapia estándar, de acuerdo con el CRI conforme a los criterios RANO.
- Comparar la duración de la respuesta (DR) con DAY101 en monoterapia frente a la quimioterapia estándar, de acuerdo con el CRI conforme a los criterios RANO
- Comparar la supervivencia global (SG) con DAY101 en monoterapia frente a la quimioterapia estándar.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Less than 25 years of age with LGG with known activating RAF alteration
- Histopathologic diagnosis of glioma or glioneuronal tumor
- At least one measurable lesion as defined by RANO criteria
- Meet indication for first-line systemic therapy

- Edad inferior a 25 años y glioma de bajo grado con una alteración de RAF activadora
- Diagnóstico histopatológico de glioma o tumor glioneuronal
- Al menos una lesión medible según lo definido por los criterios RANO
- Cumplir con la indicación de tratamiento sistémico de primera línea

E.4

Principal exclusion criteria

- Patient has any of the following tumor-histological findings:
a) Schwannoma
b) Subependymal giant cell astrocytoma (Tuberous Sclerosis)
c) Diffuse intrinsic pontine glioma, even if histologically diagnosed as WHO Grade I-II
- Patient’s tumor has additional activating molecular alterations
- Known or suspected diagnosis of neurofibromatosis Type 1 or 2 (NF-1/NF-2)
- Prior or ongoing nonsurgical anticancer therapy for this indication (eg, chemotherapy, oral/IV targeted therapy) including radiation.

- Presencia de alguno de los siguientes hallazgos histológicos a nivel del tumor:
a) Schwannoma
b) Astrocitoma subependimario de células gigantes (Esclerosis Tuberosa)
c) Glioma pontino intrínseco difuso, incluso si el diagnóstico histológico es de grado I-II según la OMS
- Presencia de otras alteraciones moleculares activadoras en el tumor
- Diagnóstico conocido o presunto de neurofibromatosis de tipo 1 o 2 (NF-1/NF-2)
- Tratamiento antineoplásico no quirúrgico anterior o en curso para esta indicación (p. ej., quimioterapia, terapias dirigidas por vía oral o i.v.), incluida la radioterapia.

E.5 End points

E.5.1

Primary end point(s)

ORR, per RANO criteria, defined as the proportion of patients with overall confirmed response of complete response (CR) or partial response (PR).

TRO, según los criterios de evaluación de la respuesta en neuroncología (RANO), definida como la proporción de pacientes que presentan una respuesta global confirmada, ya sea una respuesta completa (RC) o una respuesta parcial (RP)

E.5.1.1

Timepoint(s) of evaluation of this end point

After the last patient has been followed up for 12 months (anticipated to occur at approximately 36 months after first patient enrolled).

Una vez finalizado el seguimiento del último paciente durante 12 meses (que se prevé que tenga lugar aproximadamente 36 meses después de la inclusión del primer paciente)

E.5.2

Secondary end point(s)

- PFS per RANO criteria, defined as time from randomization to PD or death from any cause.
- DOR per RANO criteria, defined as time from confirmed response (CR, PR) to PD or death from any cause for patients with confirmed response.
- OS, defined as time from randomization up to death from any cause.
- Adverse events, vital signs, laboratory parameters.
- Change from baseline in Adaptive Behavior Composite ABS, Motor Skills Domain, Daily Living Domain, Socialization Domain, and Communication Domain Scores from the VABS by semi-structured telephone interview at 1, 2, and 5 years.
- Change in age-adjusted visual acuity (AVA), best corrected visual acuity (BCVA), and visual progression-free survival (V-PFS) defined as the time from start of treatment to visual event (blindness or VA loss) in one eye for OPG patients aged ≥ 3 years.
- ORR, defined as the proportion of patients with overall confirmed response per RANO.
- ORR, defined as the proportion of patients with overall confirmed response per RAPNO.
- CBR, defined as the proportion of patients with radiological tumor stabilization or regression per RANO or RAPNO criteria, as applicable.
- Measured by the time to first response following initiation of therapy in patients with best overall confirmed response per RANO or RAPNO criteria, as applicable.
- PFS per RANO or RAPNO (as applicable), defined as time from randomization to PD or death from any cause.
DOR, defined as time from confirmed response to PD or death from any cause for patients with confirmed response per RANO or RAPNO criteria, as applicable.

- SSP, según los criterios RANO, definida como el tiempo transcurrido desde la aleatorización hasta la PE o la muerte por cualquier causa.
- DR, según los criterios RANO, definida como el tiempo transcurrido desde la respuesta confirmada (RC, RP) hasta la PE o la muerte por cualquier causa en los pacientes que presentan una respuesta confirmada.
- SG, definida como el tiempo transcurrido desde la aleatorización hasta la muerte por cualquier causa.
- Acontecimientos adversos, constantes vitales, valores analíticos.
- Cambio con respecto al inicio en las puntuaciones de la escala de conducta adaptativa (ABS) compuesta y los dominios de habilidades motoras, vida diaria, socialización y comunicación de la escala de conducta adaptativa de Vineland (VABS) mediante una entrevista telefónica semiestructurada tras 1, 2 y 5 años.
- Cambio en la agudeza visual ajustada por la edad (AVA), la mejor agudeza visual corregida (MAVC) y la supervivencia sin progresión visual (SSP-V), definido como el tiempo transcurrido desde el inicio del tratamiento hasta el acontecimiento visual (ceguera o pérdida de AV) en un ojo en pacientes con glioma de las vías ópticas (OPG) y ≥3 años de edad.
- TRO, definida como la proporción de pacientes que presentan una respuesta global confirmada según los criterios RANO.
- TRO, definida como la proporción de pacientes que presentan una respuesta global confirmada según los criterios de evaluación de la respuesta en neuroncología pediátrica (RAPNO).
- TBC, definida como la proporción de pacientes que presentan una estabilización o una remisión radiológica del tumor según los criterios RANO o RAPNO, como corresponda.
- Medida a través del tiempo transcurrido hasta la primera respuesta tras el inicio del tratamiento en pacientes que presentan la mejor respuesta global confirmada según los criterios RANO o RAPNO, como corresponda.
- SSP, según los criterios RANO o RAPNO (como corresponda), definida como el tiempo transcurrido desde la aleatorización hasta la PE o la muerte por cualquier causa.
DR, definida como el tiempo transcurrido desde la respuesta confirmada hasta la PE o la muerte por cualquier causa en pacientes que presentan una respuesta confirmada según los criterios RANO o RAPNO, como corresponda.

E.5.2.1

Timepoint(s) of evaluation of this end point

Up to 60 months

Hasta 60 meses

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

Yes

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

Yes

E.6.13.1

Other scope of the trial description

Tolerability

Tolerabilidad

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

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)

Yes

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

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Canada

Israel

Korea, Republic of

New Zealand

Singapore

Taiwan

United States

Austria

Finland

France

Sweden

Netherlands

Spain

Switzerland

Czechia

Germany

Greece

Italy

Belgium

Denmark

Hungary

Ireland

Norway

Slovenia

United Kingdom

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

Última visita del último sujeto

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:

350

F.1.1.1

In Utero

F.1.1.2

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

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:

200

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

100

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)

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

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Young children not of age to provide consent. Parents/guardians may give consent on behalf of their children.

Niños pequeños que no tienen la edad para dar su consentimiento. Los padres/tutores pueden dar su consentimiento en nombre de sus hijos.

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

263

F.4.2.2

In the whole clinical trial

400

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

Ninguno

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	LOGGIC: LOw Grade Glioma In Children - SIOPe LGG Working Group
G.4.3.4	Network Country	Germany

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2023-02-09

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-01-31

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

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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