Clinical Trials Register

Summary
EudraCT Number:	2016-003498-16
Sponsor's Protocol Code Number:	LOXO-TRK-15003
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2020-12-16
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2016-003498-16

A.3

Full title of the trial

A Phase 1/2 Study of the Oral TRK Inhibitor LOXO-101 in Pediatric Patients with Advanced Solid or Primary Central Nervous System Tumors

STUDIO DI FASE 1/2 SULL¿INIBITORE ORALE DI TRK, LOXO-101, IN PAZIENTI PEDIATRICI AFFETTI DA TUMORI SOLIDI AVANZATI O DA TUMORI PRIMARI DEL SISTEMA NERVOSO CENTRALE

A.3.1

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

A Phase 1/2 Clinical Trial of Pediatric Cancer Patients With Solid Tumors Treated with LOXO-101

Studio di Fase 1/2 in Pazienti Pediatrici Affetti da Tumori Solidi e Trattati con LOXO-101

A.3.2

Name or abbreviated title of the trial where available

SCOUT

A.4.1

Sponsor's protocol code number

LOXO-TRK-15003

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02637687

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	LOXO ONCOLOGY INCORPORATED
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	Loxo Oncology Inc.
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	Medpace
B.5.2	Functional name of contact point	Clinical Trials Information
B.5.3	Address:
B.5.3.1	Street Address	Le Rhone Alpes, 235 cours Lafayette
B.5.3.2	Town/ city	Lyon
B.5.3.3	Post code	69006
B.5.3.4	Country	France
B.5.4	Telephone number	00330437530980
B.5.5	Fax number	00330972369787
B.5.6	E-mail	regsubmissions@medpace.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/3/15/1606
D.3 Description of the IMP
D.3.1	Product name	larotrectinib
D.3.2	Product code	LOXO-101
D.3.4	Pharmaceutical form	Capsule, hard
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.3.9.2	Current sponsor code	larotrectinib
D.3.9.4	EV Substance Code	SUB179762
D.3.10	Strength
D.3.10.1	Concentration unit	mg/g milligram(s)/gram
D.3.10.2	Concentration type	equal
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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/3/15/1606
D.3 Description of the IMP
D.3.1	Product name	larotrectinib
D.3.2	Product code	LOXO-101
D.3.4	Pharmaceutical form	Oral solution
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.3.9.2	Current sponsor code	larotrectinib
D.3.9.4	EV Substance Code	SUB179762
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	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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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	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/3/15/1606
D.3 Description of the IMP
D.3.1	Product name	larotrectinib
D.3.2	Product code	LOXO-101
D.3.4	Pharmaceutical form	Capsule, hard
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.3.9.2	Current sponsor code	larotrectinib
D.3.9.4	EV Substance Code	SUB179762
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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

Central Nervous System Neoplasms and advanced solid tumors

Neoplasie del Sistema Nervoso Centrale e tumori solidi avanzati

E.1.1.1

Medical condition in easily understood language

Pediatric Cancer

Tumori Pediatrici

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	20.1
E.1.2	Level	HLT
E.1.2	Classification code	10007960
E.1.2	Term	Central nervous system neoplasms malignant NEC
E.1.2	System Organ Class	100000004852

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	LLT
E.1.2	Classification code	10049516
E.1.2	Term	Malignant tumor
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	LLT
E.1.2	Classification code	10007959
E.1.2	Term	Central nervous system neoplasm NOS
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	PT
E.1.2	Classification code	10007958
E.1.2	Term	Central nervous system neoplasm
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

Phase I:
To determine the safety of oral larotrectinib (LOXO-101), including Dose-Limiting Toxicity (DLT), in pediatric patients with advanced solid or primary Central Nervous System tumors.
Phase II:
To determine the overall response rate (ORR) as determined by an independent radiology review committee and measured by the proportion of subjects with best overall confirmed response of complete response (CR) or partial response (PR) by the Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1), or
Response Assessment in Neuro-Oncology (RANO) criteria, as appropriate, following treatment with larotrectinib (LOXO-101) in pediatric subjects with an advanced cancer harboring a fusion involving NTRK1, NTRK2, or NTRK3 (collective referred to as NTRK fusions).

Fase I:
Stabilire la sicurezza del trattamento con larotrectinib (LOXO-101) orale, inclusa la tossicit¿ dose-limitante (DLT), nei pazienti pediatrici affetti da tumori solidi avanzati o da tumori primitivi del sistema nervoso centrale (SNC).
Fase II:
Determinare l'ORR (tasso di risposta globale) misurato sulla base della percentuale di soggetti che hanno ottenuto la migliore risposta globale confermata di CR (risposta completa) o PR (risposta parziale) rispetto ai Criteri di Valutazione della Risposta nei Tumori Solidi, versione 1.1 (RECIST 1.1), oppure ai Criteri di Valutazione della Risposta in Neuro-Oncologia (RANO), a seconda del caso, dopo trattamento con larotrectinib (LOXO-101) nei pazienti pediatrici affetti da carcinoma avanzato con geni di fusione NTRK1, NTRK2 o NTRK3 (nell¿insieme indicati come geni di fusione del recettore tirosin-chinasico per il fattore di crescita neurotrofico [NTRK]).

E.2.2

Secondary objectives of the trial

To determine the ORR based on the treating investigator's response assessment using RECIST 1.1 or RANO criteria
To evaluate the duration of response in subjects with best overall response of CR or PR as determined by 1) an independant radilogy review committee and 2) the treating investigator
To estimate the proportion of subjects that are in tumor regression as a best response
To evaluate the duration of PPFS following initiation of larotrectinib (LOXO 101)
To evaluate the duration of OS following initiation of larotrectinib (LOXO 101)

¿ Determinare l¿ORR sulla base della valutazione della risposta a discrezione dello Sperimentatore che ha in cura il paziente applicando i criteri RECIST 1.1 o RANO, a seconda del caso per il tipo di tumore
¿ Valutare la DRO (durata della risposta) in soggetti con la migliore risposta globale di CR o PR misurata 1) da un comitato di revisione indipendente del reperto radiologico e 2) dallo Sperimentatore che ha in cura il paziente
¿ Calcolare la percentuale di soggetti che evidenziano come migliore risposta una qualunque regressione della neoplasia
¿ Stabilire la durata della PFS (sopravvivenza libera da progressione) dopo l¿inizio della terapia con larotrectinib (LOXO-101)
¿ Stabilire la durata dell'OS (sopravvivenza globale) dopo l¿inizio della terapia con larotrectinib (LOXO 101)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Phase 1: Birth through 21 years of age at C1D1 with a locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies for whIch no standard or available systemic curative therapy exists or
Infants from birth and older with a diagnosis of malignancy and with a documented NTRK fusion that hAs progressed or was nonresponsive to available therapies for which no standard or systemic curative therapy exists or
patients with locally advanced or metastatic infantile fibrosarcoma who would require in the opinion of the Investigator disfiguring surgery or limb amputation to achieve a complete surgical resection or
Phase 2: Birth through 21 years of age at C1D1 with a locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies for which no standard or available systemic curative therapy with a documented NTRK gene fusion with the exception of patients with IFS (Infantile FibroSarcoma), CMN (Congenital Mesoblastic Nephroma tumors) or SBC (Secretory Breast Cancer), they may enroll into this cohort with documentation of an ETV6 rearrangement by FISH or RT-PCR or a documented NTRK fusion by NGS (Next Generation Sequencing).Patients with NTRK fusion positive benign tumors are also eligible.
Patients must have fully recovered from the acute toxic effects of all prior anti-cancer chemotherapy
Willingness of male and female patients with reproductive potential to observe conventional and effective birth control for the duration of treatment and for 3 months following study completion. Ability to swallow capsules, liquid or gastric access via a naso- or gastric tube

1 Fase 1: dalla nascita a 21 anni di età il giorno C1D1 ed essere affetto da tumore solido localmente
avanzato o metastatico oppure da tumore del SNC recidivato, progredito o non responsivo alle terapie
disponibili e per il quale non vi è terapia curativa sistemica standard o disponibile, oppure:
infanti dalla nascita e oltre con diagnosi di tumore e documentata presenza di un gene di fusione NTRK
che è progredito o non ha risposto alle terapie disponibili e per il quale non vi è terapia curativa
sistemica standard o disponibile; oppure:
pazienti con IFS localmente avanzato che, secondo lo Sperimentatore, richiederebbe una chirurgia
mutilante o l’amputazione di un arto per ottenere una resezione chirurgica completa.
Fase 2: infanti dalla nascita e oltre¿nel giorno C1D1 ed essere affetto da IFS localmente avanzato o
metastatico; pazienti affetti da IFS localmente che, secondo lo Sperimentatore, richiederebbe una
chirurgia mutilante o l’amputazione di un arto per ottenere una resezione chirurgica completa oppure:
dalla nascita a 21 anni di età il giorno C1D1 ed essere affetto da tumore solido localmente avanzato o
metastatico oppure tumore primitivo del SNC che è recidivato, progredito o non ha risposto alle terapie
disponibili e per il quale non vi è terapia curativa sistemica standard o disponibile, con documentata
presenza di un gene di fusione NTRK (o nel caso di IFS, CMN o SBC con riarrangiamento di ETV6
documentato alla FISH o RT-PCR o fusione del gene NTRK documentata all’NGS).
Pazienti
con i tumori benigni positivi alla fusione NTRK sono anche ammissibili.
I pazienti devono essere completamente guariti dagli effetti tossici acuti di tutte le precedente chemioterapie antitumorali
Disponibilità di pazienti uomini e donne con potenziale riproduttivo a osservare il controllo delle nascite convenzionale ed efficace per la durata del trattamento e per 3 mesi dopo il completamento dello studio
Capacità di ingoiare capsule, accesso liquido o gastrico attraverso un tubo nasale o gastrico

E.4

Principal exclusion criteria

Patients meeting any of the following criteria are to be excluded from study participation:
1. Major surgery within 14 days (2 weeks) prior to C1D1.
2. Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to C1D1; ongoing cardiomyopathy; or current prolonged QTc interval > 480 milliseconds.
3. Active uncontrolled systemic bacterial, viral, or fungal infection.
4. Malabsorption syndrome or other condition affecting oral absorption.
5. Current treatment with a strong CYP3A4 inhibitor or inducer other than those allowed per Section 6.3.2 of the study protocol.
6. Pregnancy or lactation.
7. Phase 2 only: Prior progression while receiving approved or investigational tyrosine kinase inhibitors targeting TRK, including entrectinib, crizotinib and lestaurtanib. Patients who received a TRK inhibitor for less than 28 days of treatment and discontinued because of intolerance remain eligible.

studio:
1. chirurgia maggiore entro 14 giorni (2 settimane) prima del giorno C1D1;
2. patologia cardiovascolare clinicamente significativa in atto o pregresso infarto miocardico nei 6 mesi precedenti C1D1, cardiomiopatia cronica oppure attuale intervallo QT prolungato, corretto per la frequenza cardiaca (QTc) >480 millisecondi;
3. infezione batterica, virale, micotica sistemica non controllata in atto;
4. sindrome da malassorbimento o altra patologia che compromette l’assorbimento orale;
5. attuale trattamento con un forte inibitore o induttore del citocroma P450 3A4 (CYP3A4 ) diverso da quelli consentiti secondo il capitolo 6.3.2;
6. gravidanza o allattamento;
7. solo fase 2: pregressa progressione in corso di terapia con inibitori della tirosinchinasi approvati o sperimentali diretti contro il gene TRK, inclusi entrectinib, crizotinib e lestaurtanib. I pazienti trattati con un inibitore delle chinasi correlate alla tropomiosina (TRK) per meno di 28 giorni e che hanno interrotto la terapia per intolleranza sono comunque eleggibili a partecipare.

E.5 End points

E.5.1

Primary end point(s)

(repeat as necessary)
Phase I: Safety of oral larotrectinib (LOXO-101), including dose-limiting toxicity (DLT) until the Maximum Tolerated Dose or suitable dose based on PK exposure or unacceptable DLT are reached. Pase II: Overall response rate (ORR) as determined by an independent radiology review committee and measured by the proportion of subjects with best overall confirmed response of complete response (CR) or partial response (PR) by the Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1), or Response Assessment in Neuro-Oncology (RANO) criteria, as appropriate.

(ripetere quando necessario)
Fase I: sicurezza di larotrectinib (LOXO-101) per via orale, inclusa la DLT (Dose-Limiting Toxicity [tossicità dose-limitante]) finché non è stata raggiunta la Dose massima tollerata o una dose idonea sulla base dell'esposizione PK (Phamacokinetics [Farmacocinetica]) o una DLT inaccettabile. Fase II: ORR (Overall Response Rate [tasso di risposta globale]) determinato da un comitato di revisione radiologica indipendente sulla base della percentuale di soggetti che hanno ottenuto la migliore risposta globale confermata di CR (Complete Response [risposta completa]) o PR (Partial Response [risposta parziale]) rispetto ai RECIST 1.1 (Response Evaluation Criteria in Solid Tumors, version 1.1 [Criteri di Valutazione della Risposta nei Tumori Solidi, versione 1.1]), oppure ai criteri RANO (Response Assessment in Neuro-Oncology [Valutazione della Risposta in Neuro-Oncologia]), ove appropriato.

E.5.1.1

Timepoint(s) of evaluation of this end point

Every cohort from baseline until disease progression, unacceptable toxicity, patient’s withdrawal of consent, or death

Ogni coorte dal valore basale fino alla progressione della malattia, tossicità inaccettabile, ritiro del consenso da parte del/la paziente o decesso

E.5.2

Secondary end point(s)

(Repeat as necessary)
PK analysis: Plasm and CSF concentrations of larotrectinib (LOXO-101) will be determined with a validtaed bioanalytical assay. Pain and Health related QoL: in patients 3 years age or older pain will be assessed by the Wong-Baker Faces scale.

(Ripetere quando necessario)
Analisi PK: le concentrazioni plasmatiche e di LCS di larotrectinib (LOXO-101) saranno determinate con un saggio bioanalitico validato. Dolore e QoL (Quality of Life [Qualit¿ della vita]) correlata alla salute: nei/lle pazienti di almeno 3 anni di et¿, il dolore sar¿ valutato sulla base della scala di Wong-Baker (FACES).

E.5.2.1

Timepoint(s) of evaluation of this end point

The PK parameters will be calculated from the C1D1 and C2D1 (cohorts 1&2)and C4D1 and every 4 cycles thereafter (cohortes 3 and higher) data. Pain and Health related QoL: Mean changes over time will be analyzed descriptively by time-point.

I parametri PK saranno calcolati dai dati di C1D1 e C2D1 (coorti 1 e 2) e C4D1, dopodich¿ ogni 4 cicli (coorti 3 e superiori). Dolore e QoL correlata alla salute: variazioni medie nel tempo saranno analizzate in maniera descrittiva per punto temporale.

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

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

Yes

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)

Yes

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

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

Yes

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.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

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

Information not present in EudraCT

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

Denmark

France

Germany

Italy

Netherlands

Spain

Sweden

Switzerland

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

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

Last Patient Last Visit

Ultima visita dell'ultimo paziente

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

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)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

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

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Patients newborn to their legal majority age, with a documented NTRK fusion
Infants from birth with a diagnosis malignancy and for patients with a locally advanced or metastatic infantile fibrosarcoma

I/le pazienti da neonati fino alla maggiore et¿ con documentata presenza del gene di fusione NTRK
Neonati con una diagnosi di tumore e per i/le pazienti con fibrosarcoma infantile localmente avanzato o metastatico

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

2017-05-31

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-03-23

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

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