Clinical Trials Register

Summary
EudraCT Number:	2020-005106-25
Sponsor's Protocol Code Number:	D8241C00001
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-11-03
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

2020-005106-25

A.3

Full title of the trial

A Modular Phase I/II, Open-Label, Multi-Centre Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of AZD0466 Monotherapy or in Combination in Patients with Advanced Haematological Malignancies

Studio modulare di fase I/II, in aperto, multicentrico per valutare la sicurezza, la tollerabilità, la farmacocinetica e l’efficacia preliminare di AZD0466 in monoterapia o in combinazione in pazienti con tumori maligni ematologici in stadio avanzato

A.3.1

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

A Study to Assess the Safety, Tolerability, Pharmacokinetics of AZD0466 in Patients with Advanced Haematological Malignancies

Studio di fase I/II di AZD0466 in monoterapia o in combinazione in pazienti con tumori maligni ematologici in stadio avanzato

A.3.2

Name or abbreviated title of the trial where available

NIMBLE

A.4.1

Sponsor's protocol code number

D8241C00001

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04865419

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	ASTRAZENECA AB
B.1.3.4	Country	Sweden
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	AstraZeneca AB
B.4.2	Country	Sweden
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	AstraZeneca AB
B.5.2	Functional name of contact point	Information Center
B.5.3	Address:
B.5.3.1	Street Address	NA
B.5.3.2	Town/ city	NA
B.5.3.3	Post code	NA
B.5.3.4	Country	United States
B.5.6	E-mail	information.center@astrazeneca.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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	AZD0466 polvere per concentrato per soluzione per infusione
D.3.2	Product code	[AZD0466]
D.3.4	Pharmaceutical form	Powder and solvent for 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	AZD0466
D.3.9.2	Current sponsor code	AZD0466
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	500
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	Yes
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	Rasburicase
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Powder and solvent for 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	RASBURICASE
D.3.9.2	Current sponsor code	NA
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	1500
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	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	Yes
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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	Allopurinolo
D.3.2	Product code	[NA]
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	ALLOPURINOLO
D.3.9.2	Current sponsor code	NA
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.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.2.5	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	Allopurinolo
D.3.2	Product code	[NA]
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	ALLOPURINOLO
D.3.9.2	Current sponsor code	NA
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	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

Advanced Haematological Malignancies

Tumori maligni ematologici in stadio avanzato

E.1.1.1

Medical condition in easily understood language

Blood cancer

Tumori ematologici

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	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10007051
E.1.2	Term	Cancer (NOS)
E.1.2	System Organ Class	100000004864

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 the safety and tolerability of AZD0466 in patients with advanced haematological malignancies

Valutare la sicurezza e la tollerabilità di AZD0466 in pazienti con tumori maligni ematologici in stadio avanzato

E.2.2

Secondary objectives of the trial

To characterize the pharmacokinetic (PK) profile of AZD0466 following intravenous administration (via PK profiles of the active moiety AZD4320 in plasma) [Core Module]

To estimate the preliminary antitumor activity of AZD0466 [Module 1]

Caratterizzare il profilo faramacocinetico (PK) di AZD0466 in seguito a somministrazione endovenosa (tramite profili PK della frazione attiva AZD4320 nel plasma) [Modulo Core]

Stimare l'attività preliminare antitumorale di AZD0466 [Module 1]

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

-Patient must be aged >=18 years inclusive, at the time of signing the informed consent. In some countries parental consent may be required in addition to an assent form for patients who are 18 years of age.
-Histologically confirmed acute myeloid leukaemia (AML) or acute lymphoblastic leukaemia (ALL) for which there are limited treatment options known to provide clinical benefit.
-Subjects must have received at least one prior line of therapy, and an established standard of care with proven benefit, and for which the patient is eligible, must not be available at the time of enrolment.
-Eastern Cooperative Oncology Group performance status <=2. Performance status must not have deteriorated by >=2 levels within 2 weeks after providing informed consent.
-Predicted life expectancy >= 8 weeks.
-White blood cell count must be <10 x 10^9/L. Treatment with hydroxyurea during screening and Cycle 1 to achieve this level is permitted.
-Adequate organ function at screening as per the protocol defined criteria

PLEASE REFER TO THE PROTOCOL FOR FURTHER INCLUSION CRITERIA

-I pazienti devono avere un’età >=18 anni al momento della firma del consenso informato. In alcuni Paesi potrebbe essere richiesto il consenso dei genitori in aggiunta al modulo di assenso per i pazienti di 18 anni di età.
- Leucemia mieloide acuta (LMA) o leucemia linfoblastica acuta (LLA) istologicamente confermata per la quale esistono opzioni terapeutiche limitate note per fornire un beneficio clinico.
- I soggetti devono aver ricevuto almeno una precedente linea di terapia e uno standard
di cura consolidato con beneficio dimostrato e per il quale il paziente è idoneo; non devono essere disponibili al
momento dell’arruolamento.
- Stato di validità secondo l’Eastern Cooperative Oncology Group (gruppo orientale cooperativo di oncologia) <=2. Lo stato di validità non deve essere peggiorato di >=2 livelli entro 2 settimane dopo aver fornito il consenso informato.
- Aspettativa di vita prevista >=8 settimane.
- La conta leucocitaria deve essere <10 x 10^9/l. È consentito il trattamento con idrossiurea durante lo screening e il Ciclo 1 per raggiungere questo livello.
-Adeguata funzionalità d’organo allo screening secondo i criteri definiti dal protocollo.

SI PREGA DI FARE RIFERIMENTO AL PROTOCOLLO PER ULTERIORI CRITERI DI INCLUSIONE

E.4

Principal exclusion criteria

-Unresolved toxicity from prior anticancer therapy of Common Terminology Criteria for Adverse Events Grade >=2. Patients with Grade 2 neuropathy or Grade 2 alopecia are eligible.
-Active idiopathic thrombocytopenic purpura.
-Haemopoietic Stem cell transplant < 100 days prior to the first dose of study treatment.
-Immunosuppression for graft versus host disease (GVHD) or GVHD prophylaxis within 4 weeks prior to the first dose of study treatment. The following are permitted: (a) topical steroids for GVHD; (b) systemic steroids for GVHD up to 2 weeks prior to the first dose of study treatment; (c) if systemic steroids are being used, they may be continued up until 2 days prior to the first dose of study treatment.
-Active central nervous system (CNS) leukaemia/leptomeningeal disease/spinal cord compression. Patients who have a history of CNS leukaemia must be free of CNS leukaemia for >30 days prior to the first dose of study treatment, and the most recent 2 lumbar punctures must be negative for leukaemic cells, to be eligible. •Known uncontrolled infection with cytomegalovirus.
-Active infection including human immunodeficiency virus, Hepatitis B,Hepatitis C, or severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2).
-As judged by the Investigator: any evidence of severe or uncontrolledsystemic diseases; current unstable or uncompensated respiratory or cardiac conditions; uncontrolled hypertension; history of, or active, bleeding diatheses (eg, haemophilia or von Willebrand disease); uncontrolled active systemic fungal, bacterial, or other infection.

PLEASE REFER TO THE PROTOCOL FOR FURTHER EXCLUSION CRITERIA

-Tossicità non risolta da precedente terapia antitumorale dei Criteri terminologici comuni per gli eventi avversi di grado >=2. I pazienti con neuropatia di grado 2 o alopecia di grado 2 sono idonei.
-Porpora trombocitopenica idiopatica attiva.
-Trapianto di cellule staminali emopoietiche <100 giorni prima della prima dose di trattamento dello studio.
-Immunosoppressione dovuta a malattia del trapianto contro l’ospite (GVHD) o profilassi della GVHD nelle 4 settimane precedenti la prima dose del trattamento dello studio. È consentito quanto segue: (a) steroidi topici per la GVHD; (b) steroidi sistemici per la GVHD fino a 2 settimane prima della prima dose del trattamento dello studio; (c) se vengono utilizzati steroidi sistemici, possono essere proseguiti fino a 2 giorni prima della prima dose del trattamento dello studio.
-Leucemia attiva del sistema nervoso centrale (SNC)/malattia leptomeningea/compressione del midollo spinale. Per essere idonei, i pazienti che presentano un’anamnesi di leucemia del SNC devono essere liberi da leucemia del SNC da >30 giorni prima della prima dose di trattamento dello studio e le 2 punture lombari più recenti devono essere negative per le cellule leucemiche.
-Nota infezione non controllata da citomegalovirus.
-Infezione attiva, tra cui virus dell’immunodeficienza umana, epatite B, epatite C o sindrome respiratoria acuta grave-coronavirus-2 (SARS-CoV-2).
-Secondo il giudizio dello sperimentatore: qualsiasi evidenza di malattie sistemiche gravi o non controllate, attuali condizioni respiratorie o cardiache instabili o non compensate, ipertensione non controllata, anamnesi o presenza di diatesi emorragiche (ad es., emofilia o malattia di von Willebrand) attive, infezione fungina, batterica o di altro tipo sistemica attiva non controllata.

SI PREGA DI FARE RIFERIMENTO AL PROTOCOLLO PER ULTERIORI CRITERI DI ESCLUSIONE

E.5 End points

E.5.1

Primary end point(s)

• Number of subjects with adverse events (AEs) and serious adverse events (SAEs) [Core Module]
• Changes from baseline in laboratory findings, physical examinations, performance status, electrocardiograms and vital signs [Core Module]
• Dose-limiting toxicity [Module 1]
• Maximum tolerated dose [Module 1]
• Recommended Phase II dose [Module 1]

• Incidenza di EA e SAE
• Variazioni rispetto al basale nei risultati di laboratorio, negli esami obiettivi, nello stato di validità, negli elettrocardiogrammi e nei segni vitali
• Tossicità limitante la dose [Modulo 1]
• Massima dose tollerata [Modulo 1]
• Dose raccomandata Fase II [Modulo 1]

E.5.1.1

Timepoint(s) of evaluation of this end point

• Module 1 and Module 2: From Screening until post treatment follow-up (28 days after last dose);
• Module 1: From Screening until post treatment follow-up (28 days after last dose); Module 2: From Screening until Day 19
• Day 1 to Day 35 of Cycle 1 (35-day Cycle)
• Day 1 to Day 35 of Cycle 1 (35-day Cycle)
• Day 1 to Day 35 of Cycle 1 (35-day Cycle)

• Modulo 1 e Modulo 2: Dallo Screening fino al follow-up post trattamento (28 giorni dopo l'ultima dose);
• Modulo 1: Dallo Screening fino al follow-up post trattamento (28 giorni dopo l'ultima dose); Modulo 2: dallo screening fino al giorno 19
• Dal giorno 1 al giorno 35 del ciclo 1 (ciclo di 35 giorni)
• Dal giorno 1 al giorno 35 del ciclo 1 (ciclo di 35 giorni)
• Dal giorno 1 al giorno 35 del ciclo 1 (ciclo di 35 giorni)

E.5.2

Secondary end point(s)

• Plasma concentrations and derived PK parameters for total and released AZD4320
• Complete Response Rate (CR+CRi); Time to Response (TTR); Duration of Response (DoR); Overall Survival (OS) [Module 1]
• Area under the plasma concentration-curve (AUC) of AZD4320 after administration of AZD0466 alone and in combination with voriconazole [Module 2]; Maximum observed plasma (peak) drug concentration (Cmax) of AZD4320 after administration of AZD0466 alone and in combination with voriconazole [Module 2]

• Concentrazioni plasmatiche e parametri PK derivati ¿¿per AZD4320 totale e rilasciato
• Tasso di risposta completa (CR + CRi); Tempo di risposta (TTR); Durata della risposta (DoR); Sopravvivenza globale (OS) [Modulo 1]
• Area sotto la curva di concentrazione plasmatica (AUC) di AZD4320 dopo somministrazione di AZD0466 da solo e in combinazione con voriconazolo [Modulo 2]; Massima concentrazione plasmatica (picco) del farmaco (Cmax) osservata di AZD4320 dopo la somministrazione di AZD0466 da solo e in combinazione con voriconazolo [Modulo 2]

E.5.2.1

Timepoint(s) of evaluation of this end point

• Module 1: Days 1 and 4, and days 8, 9, 10, 11 of Cycle 1;
• Module 2: Days 1, 2, 3, 4, and 8 (Period 1) and days 15, 16, 17, 18, 19 (Period 3) during Cycle 1 (21 days)
• Module 1: Day 1 until post treatment follow-up (28 days after last dose)
• Module 2: Days 1, 2, 3, 4, and 8 (Period 1) and days 15, 16, 17, 18, 19 (Period 3) during Cycle 1 (21 days)

• Modulo 1: giorni 1 e 4 e giorni 8, 9, 10, 11 del ciclo 1;
• Modulo 2: giorni 1, 2, 3, 4 e 8 (periodo 1) e giorni 15, 16, 17, 18, 19 (periodo 3) durante il ciclo 1 (21 giorni)
• Modulo 1: giorno 1 fino al follow-up post trattamento (28 giorni dopo l'ultima dose)
• Modulo 2: giorni 1, 2, 3, 4 e 8 (periodo 1) e giorni 15, 16, 17, 18, 19 (periodo 3) durante il ciclo 1 (21 giorni)

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

Yes

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

Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy

Sicurezza, Tollerabilità, Farmacocinetica ed Efficacia Preliminare

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

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

Korea, Republic of

United States

France

Germany

Italy

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

The end of the study is defined as the date of the last visit of the last subject in the study or last scheduled procedure shown in the Schedule of Activity for the last subject in the study globally.

La fine della sperimentazione è definita come la data dell'ultima visita dell'ultimo paziente nello studio o l'ultima procedura programmata mostrata nella scheda di attività dell'ultimo paziente nello studio a livello globale

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)

Yes

F.1.2.1

Number of subjects for this age range:

152

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

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

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

160

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)

Not applicable

Non applicabile

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

2022-01-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-01-21

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2023-07-24

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