Clinical Trials Register

Summary
EudraCT Number:	2016-000558-37
Sponsor's Protocol Code Number:	ARQ092-103
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-01-03
Trial results	View 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

2016-000558-37

A.3

Full title of the trial

A Phase 1/2 Study of ARQ 092 in Patients with Overgrowth Diseases and Vascular Anomalies with Genetic Alterations of the PI3K/AKT Pathway

Studio di fase 1/2 su ARQ 092 in pazienti affetti da malattie da iperaccrescimento e anomalie vascolari con alterazioni genetiche del pathway PI3K/AKT

A.3.1

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

Study of patients with an increase in the size of certain parts of their body and circulatory abnormalities.

Studio su pazienti che presentano un aumento della dimensione di alcune parti del proprio corpo e anomalie dell’apparato circolatorio.

A.3.2

Name or abbreviated title of the trial where available

ARQ 092-103

A.4.1

Sponsor's protocol code number

ARQ092-103

A.5.1

ISRCTN (International Standard Randomised Controlled Trial) Number

ISRCTN00000000

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT00000000

A.5.3

WHO Universal Trial Reference Number (UTRN)

U0000-0000-0000

A.5.4

Other Identifiers

Name:

IND

Number:

130784

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	ArQule, Inc.
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	ArQule 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	Innopharma Srl
B.5.2	Functional name of contact point	Paola Strada
B.5.3	Address:
B.5.3.1	Street Address	Via Lavoratori Autobianchi 1
B.5.3.2	Town/ city	Desio (MB)
B.5.3.3	Post code	20832
B.5.3.4	Country	Italy
B.5.4	Telephone number	00390362573128
B.5.5	Fax number	00390362544211
B.5.6	E-mail	p.strada@innopharma.it

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	ARQ 092
D.3.2	Product code	ARQ 092 -2 MSA
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.3.11 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

subjects (at least 6 years) suffering from diseases overgrowth and vascular anomalies with genetic alterations of the PI3K / AKT pathway.

soggetti (di almeno 6 anni) affetti da malattie da iperaccrescimento e anomalie vascolari con alterazioni genetiche del pathway PI3K/AKT.

E.1.1.1

Medical condition in easily understood language

patients at least 6 years with diseases of overgrowth and abnormal blood vessels due to genetic causes

pazienti di almeno 6 anni affetti da malattie di iperaccrescimento e anomalie dei vasi sanguigni dovute a cause genetiche

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	SOC
E.1.2	Classification code	10010331
E.1.2	Term	Congenital, familial and genetic disorders
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

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

The primary objective of this study is to assess the safety of ARQ 092 in subjects with overgrowth diseases and vascular anomalies with genetic alterations of the PI3K/AKT pathway.

L'obiettivo primario di questo studio è valutare la sicurezza di ARQ 092 in soggetti affetti da malattie da iperaccrescimento e anomalie vascolari con alterazioni genetiche del pathway PI3K/AKT.

E.2.2

Secondary objectives of the trial

To determine the clinical and biological activity of ARQ 092 in subjects with overgrowth diseases and vascular anomalies
• To determine the recommended dose of ARQ 092 in subjects with overgrowth diseases and vascular anomalies
• To evaluate the association between markers of the PI3K/AKT signaling pathway, their pharmacodynamic changes, and clinical activity in subjects treated with ARQ 092
• To assess the pharmacokinetic profile of ARQ 092
• To assess the changes while on therapy (change from baseline after 3, 6, and possibly 9 cycles on therapy) of the excess lesion volume at the affected site(s) by imaging (e.g., bi-dimensional/volumetric magnetic resonance imaging [MRI], computed tomography [CT] scan, ultrasound) and/or circumferential measurements including photographs, where applicable
• To measure the changes in clinical function assessment while on therapy (change from baseline after 3, 6, and possibly 9 cycles on therapy)

• Determinare: - l'attività clinica e biologica di ARQ 092 in soggetti affetti da malattie da iperaccrescimento e anomalie vascolari -la dose raccomandata di ARQ 092 in soggetti affetti da malattie da iperaccrescimento e anomalie vascolari • Valutare:- l’associazione tra i marcatori della via di segnalazione PI3K/AKT, le rispettive modifiche farmacodinamiche e l’attività clinica nei soggetti trattati con ARQ 092 -il profilo farmacocinetico di ARQ 092 - i cambiamenti nel corso della terapia (rispetto al basale dopo 3, 6 e possibilmente 9 cicli di terapia) del volume in eccesso della lesione nei siti interessati per mezzo di diagnostica per immagini (ad es. risonanza magnetica bidimensionale/volumetrica [MRI], tomografia computerizzata [CT], ultrasuoni) e/o misurazioni della circonferenza, incluse fotografie, ove applicabile.• Misurare i cambiamenti nella valutazione della funzionalità clinica nel corso della terapia (rispetto al basale dopo 3, 6 e possibilmente 9 cicli di terapia)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Each prospective subject must meet ALL of the following inclusion criteria in order to be eligible for this study:
1. Male or female subjects ≥ 6 years old
2. Overgrowth diseases or vascular anomalies with documented and/or confirmed somatic genetic alterations of PIK3CA, AKT, or PTEN defined/assessed as:
 Measureable segmental overgrowth, currently experiencing growth, or with clinical history of overgrowth progression
 Vascular and/or lymphatic overgrowth diseases as determined by clinical (such as dermatological), imaging (e.g., bi-dimensional/volumetric MRI, CT, ultrasound), and histological/cytological criteria
3. Subjects with significant morbidity, poor quality of life, or with disease characterized by poor prognosis
4. No standard systemic therapeutic option available or no satisfactory response to prior experimental or local therapies
5. Signed informed consent and, when applicable, signed assent
6. Hemoglobin (Hgb) depending on age:
 6-9 years male and female: ≥ 11.5 g/dL
 10-17 years female: ≥ 12.0 g/dL
 10-17 years male: ≥ 12.5 g/dL
 > 17 years male and female: ≥ 10.0 g/dL
7. Absolute neutrophil count (ANC): ≥ 1.5 x 109/L
8. Platelet count ≥ 150 x 109/L (for subjects with KHE or MLT, platelet count must be ≥ 75 x 109/L)
9. Total bilirubin ≤ 1.5 x upper limit of normal (ULN)/L
10. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x ULN
11. Serum creatinine depending on age:
 6-10 years male and female: maximum 0.59 mg/dL
 11-15 years male and female: maximum 1.2 mg/dL
 > 15 years male and female: maximum 1.5 mg/dL
12. If a female is of child-bearing potential, documentation of a negative pregnancy test is required prior to enrollment. Sexually active subjects (male and female) must use adequate contraceptive measures while on study and for up to 90 days after ending treatment

1. Soggetti di sesso maschile o femminile ≥ 6 anni di età
2. Malattie da iperaccrescimento e anomalie vascolari con alterazioni genetiche somatiche confermate e/o documentate di PIK3CA, AKT o PTEN definite/valutate come:
• Crescita segmentaria eccessiva misurabile, con crescita attualmente in corso o con una storia clinica di progressione della crescita eccessiva.
• Malattie da iperaccrescimento vascolari e/o linfatiche, determinate per mezzo di criteri clinici (dermatologici, ad esempio), di imaging (ad es. MRI bidimensionale/volumetrica, CT, ultrasuoni) e istologici/citologici.
3. Soggetti con morbilità significativa, scarsa qualità di vita o affetti da malattie caratterizzate da prognosi infausta.
4. Nessuna opzione terapeutica sistemica standard disponibile o nessuna risposta soddisfacente alle precedenti terapie sperimentali o locali.
5. Consenso informato firmato e, ove applicabile, assenso firmato.
6. Emoglobina (Hgb), a seconda dell’età:
• maschi e femmine 6-9 anni: ≥ 11,5 g/dL
• femmine 10-17 anni: ≥ 12,0 g/dL
• maschi 10-17 anni: ≥ 12,5 g/dL
• maschi e femmine > 17 anni: ≥ 10,0 g/dL
7. Conta assoluta dei neutrofili (CAN): ≥ 1,5 x 109/L
8. Conta piastrinica ≥ 150 x 109/L (per soggetti affetti da KHE o MLT, la conta piastrinica deve essere ≥ 75 x 109/L)
9. Bilirubina totale ≤ 1,5 x limite superiore della norma (ULN)/L
10. Aspartato aminotransferasi (AST) e alanina aminotransferasi (ALT) ≤ 3 x ULN
11. Creatinina sierica a seconda dell’età:
• maschi e femmine 6-10 anni: 0,59 mg/dL max.
• maschi e femmine 11-15 anni: 1,2 mg/dL max.
• maschi e femmine > 15 anni: 1,5 mg/dL max.
12. Le donne in età fertile dovranno presentare, prima di essere inserito nello studio, un documentato risultato negativo al test di gravidanza. I soggetti sessualmente attivi (sia di sesso maschile che femminile) devono adottare misure contraccettive adeguate durante lo studio e nei 90 giorni successivi al trattamento finale.

Ogni soggetto potenziale deve soddisfare TUTTI i criteri di inclusione per essere idoneo al presente studio.

E.4

Principal exclusion criteria

Potential subjects who meet ANY of the following exclusion criteria are not eligible for enrollment into this study:
1. History of Type 1 or 2 uncontrolled diabetes mellitus requiring regular medication (other than metformin or other oral hypoglycemic agents) or fasting glucose ≥ 160 mg/dL (if > 12 years) and ≥ 180 mg/dL (if ≤ 12 years) at the screening visit
2. Grade ≥ 2 (per National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE version 4.03]) hypercholesterolemia or hypertriglyceridemia or > 8% glycated Hgb (HbA1C)
3. Malabsorption syndrome
4. History of myocardial infarction (MI) or New York Heart Association (NYHA) Class II-IV congestive heart failure within 6 months of the administration of the first dose of ARQ 092 (MI occurring > 6 months of the first dose of ARQ 092 will be permitted); Grade 2 (per NCI CTCAE version 4.03) or worse conduction defect (e.g., right or left bundle branch block); left ventricular ejection fraction (LVEF) < 50% assessed by echocardiogram/multigated acquisition (MUGA) scan
5. Major surgery, chemotherapy, radiotherapy, or immunotherapy within four weeks of the first dose of ARQ 092
6. No experimental systemic therapy for the purpose of treating PIK3CA Related Overgrowth Spectrum (PROS) (e.g., sirolimus, everolimus, high dose steroids) within two weeks of first dose of ARQ 092
7. Previous treatment with AKT inhibitors
8. Concurrent severe uncontrolled illness not related to overgrowth diseases
9. Ongoing or active known infection, including human immunodeficiency virus (HIV) infection or bleeding
10. Psychiatric illness/substance abuse/social situation that would limit compliance with study requirements
11. Pregnant or breastfeeding
12. Severe hypersensitivity reactions to mTOR inhibitors (e.g., sirolimus, everolimus)
13. Insufficient language proficiency of the subject (or legal guardian) to complete the informed consent and quality of life questionnaires
14. Inability to swallow oral medications

1. Storia di diabete mellito di tipo 1 o 2 che richiede l’assunzione regolare di farmaci (che non siano metformina o altri agenti ipoglicemici orali) o glicemia a digiuno ≥ 160 mg/dL (se > 12 anni) e ≥ 180 mg/dL (se ≤ 12 anni) alla visita di screening
2. Ipercolesterolemia e ipertrigliceridemia di grado ≥ 2 (secondo i Criteri di terminologia comune per gli eventi avversi [CTCAE versione 4.03] dell’Istituto nazionale dei tumori [NCI]) o Emoglobina glicata (HbA1C) > 8%
3. Sindrome da malassorbimento
4. Storia di infarto del miocardio (MI) o insufficienza cardiaca congestizia di classe II-IV per la New York Heart Association (NYHA) verificatosi entro 6 mesi dalla somministrazione della prima dose di ARQ 092 (verranno accettati i casi di IM verificatisi > 6 mesi dalla prima dose di ARQ 092); disturbo di conduzione di grado 2 (secondo CTCAE versione 4.03 del NCI) o più grave (ad es. blocco di branca destra o sinistra); frazione di eiezione del ventricolo sinistro (LVEF) < 50% valutata per mezzo di ecocardiogramma/scansione con acquisizione a gate multipli (MUGA)
5. Intervento chirurgico maggiore, chemioterapia, radioterapia o immunoterapia entro quattro settimane dalla prima dose di ARQ 092
6. Nessuna terapia sistemica sperimentale per trattare il PIK3CA Related Overgrowth Spectrum (PROS) (ad es. sirolimus, everolimus, elevate dosi di steroidi) entro due settimane dalla prima dose di ARQ 092
7. Precedente trattamento con inibitori di AKT
8. Malattia grave non controllata concomitante non correlata a malattie da iperaccrescimento
9. Infezione nota attiva o in atto, inclusa infezione causata dal virus da immunodeficienza umana (HIV) o emorragia
10. Malattia psichiatrica/abuso di sostanze/condizione sociale che potrebbe limitare la conformità ai requisiti dello studio
11. Gravidanza o allattamento
12. Gravi reazioni di ipersensibilità agli inibitori di mTOR (ad es. sirolimus, everolimus)
13. Soggetto (o tutore legale) con conoscenze linguistiche insufficienti per completare il consenso informato o i questionari sulla qualità della vita
14. Incapacità di assumere farmaci per via orale

I soggetti potenziali che soddisfano UNO QUALSIASI dei criteri di esclusione non sono idonei a essere inseriti nel presente studio.

E.5 End points

E.5.1

Primary end point(s)

The primary endpoint of this study is safety and it is measured by safety variables which include the reported adverse events (AEs), laboratory tests, vital signs, ECG, echocardiograms, and physical examination.

L’endpoint principale di questo studio è la sicurezza, che viene misurata per mezzo di variabili di sicurezza che includono eventi avversi (AE) rilevati, test di laboratorio, parametri vitali, ECG, ecocardiogrammi e visita medica.

E.5.1.1

Timepoint(s) of evaluation of this end point

for the entire duration of the study and in the three months following the end of the same

per tutta la durata dello studio e nei tre mesi successivi alla fine dello stesso

E.5.2

Secondary end point(s)

PK which are measured by maximum plasma drug concentration (Cmax), area under the curve (AUC), and elimination half-life
• Pharmacodynamic activity which is measured by evaluating changes in PI3K/AKT signaling pathway markers between baseline and post-dose tissue samples
• Pharmacodynamic activity which is measured by evaluating changes in insulin-like growth factor (IGF)-binding protein 2, fibrinogen, d-dimers and circulating DNA for AKT, PIK3CA and PTEN from blood samples collected at baseline and post-dose timepoints
• Recommended dose of ARQ 092 in subjects with overgrowth diseases and vascular anomalies will be determined following a review of all safety and pharmacodynamic data generated
• Efficacy, measured as evidence of changes to the excess lesion volume by comparing baseline imaging (such as MRI, CT, ultrasound) and/or circumferential measurements to post dose timepoints
• Efficacy, measured as changes in the degree of clinical impairment by evaluating responses to Clinical Function Assessment questions from baseline to post-dose timepoints

Exploratory endpoints include the following:

• Efficacy, measured as quality of life changes by evaluating responses to the PedsQL™ questionnaires and PedsQL™ Pediatric Pain Questionnaire/short-form McGill Pain Questionnaire from baseline to post dose timepoints
• Efficacy, measured as changes in performance status by comparing Karnofsky/Lansky scores at baseline to those post-dose

• Parametri PK, ottenuti misurando la concentrazione massima del farmaco nel plasma (Cmax), l’area sotto la curva (AUC) e l’emivita (di eliminazione)
• Attività farmacodinamica, misurata valutando i cambiamenti dei marcatori della via di segnalazione PI3K/AKT tra i campioni di tessuto al basale e post-dose
• Attività farmacodinamica, misurata valutando i cambiamenti di proteina 2 legante il fattore di crescita insulino-simile (IGF), fibrinogeno, d-dimeri e DNA circolante per AKT, PIK3CA e PTEN da campioni di sangue raccolti al basale e a diversi tempi stabiliti dopo la somministrazione (di ARQ 092 ).
• La dose raccomandata di ARQ 092 in soggetti affetti da malattie da iperaccrescimento e anomalie vascolari verrà determinata dopo aver rivisto tutti i dati farmacodinamici e di sicurezza generati
• Efficacia, misurata in termini di evidenza di cambiamenti del volume in eccesso della lesione confrontando le immagini (come ad esempio MRI, CT e ultrasuoni) e/o le misurazioni della circonferenza ottenute al basale con quelle ottenute a vari tempi stabiliti dopo la somministrazione.

Gli endpoint esplorativi includono quanto segue:

• Efficacia, misurata in termini di cambiamenti della qualità della vita valutando le risposte ai questionari PedsQL™ e al PedsQL™ Pediatric Pain Questionnaire (questionario di valutazione del dolore nel paziente pediatrico)/alla forma ridotta del McGill Pain Questionnaire (questionario del dolore di McGill) dal basale a vari tempi stabiliti dopo la somministrazione.
• Efficacia, misurata in termini di cambiamenti del performance status confrontando i punteggi ottenuti con la scala di Karnofsky/Lansky al basale con quelli a vari tempi stabiliti dopo la somministrazione.

E.5.2.1

Timepoint(s) of evaluation of this end point

PK: cycles from 1 to 8; pharmacodynamic activity: cycles 1, 4, 7, 10, 16 and at the end of treatment; effectiveness: screening, Cycles 4, 7, 10, 16 and at the end of treatment

exploratory endpoint: time points of cycles 1, 4, 7, 10, 16 and EOT

PK: cicli da 1 a 8; attività farmacodinamica: cicli 1, 4, 7, 10, 16 e a fine trattamento; efficacia: screening, cicli 4, 7, 10, 16 e a fine trattamento

Time point obiettivi esplorativi: cilci 1, 4, 7, 10, 16 e a fine trattamento

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

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

QUALITY OF LIFE

QUALITA' DELLA VITA

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 assessment and determination of the dose for the subsequent steps

valutazione di sicurezza e determinazione della dose per le fasi successive

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

Yes

E.8.1.7.1

Other trial design description

DOSE ESCALATION INTRA PAZIENTE

DOSE ESCALATION INTRA PATIENT

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

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

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

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)

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)

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)

Yes

F.1.2.1

Number of subjects for this age range:

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

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

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

2017-02-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-12-14

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2022-01-12

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IMP with orphan designation in the indication
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Clinical trials