Clinical Trials Register

Summary
EudraCT Number:	2021-003177-58
Sponsor's Protocol Code Number:	SPIOMET4HEALTH
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:	2022-01-17
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

2021-003177-58

A.3

Full title of the trial

A Phase II, randomised, multi-centric, multi-national clinical trial to evaluate the efficacy, tolerability, and safety of a fixed dose combination of Spironolactone, Pioglitazone & Metformin (SPIOMET) for adolescent girls and young adult women (AYAs) with polycystic ovary syndrome (PCOS)

Studio clinico di fase II, randomizzato, multicentrico e multinazionale per valutare l'efficacia, la tollerabilità e la sicurezza di una combinazione a dose fissa di Spironolattone, Pioglitazone e Metformina (SPIOMET) per adolescenti e giovani adulte (AYA) con sindrome dell'ovaio policistico (PCOS)

A.3.1

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

A.3.2

Name or abbreviated title of the trial where available

The SPIOMET4HEALTH Trial

A.4.1

Sponsor's protocol code number

SPIOMET4HEALTH

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/150/2021

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Fundació Privada per a la Recerca y la Docencia Sant Joan de Déu
B.1.3.4	Country	Spain
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	European Commission
B.4.2	Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Fundació Sant Joan de Déu (FSJD)
B.5.2	Functional name of contact point	Coordinator
B.5.3	Address:
B.5.3.1	Street Address	Esplugués de Llobregat
B.5.3.2	Town/ city	Barcelona
B.5.3.3	Post code	08950
B.5.3.4	Country	Spain
B.5.4	Telephone number	0034936009751
B.5.5	Fax number	0034936009771
B.5.6	E-mail	lourdes.ibanez@sjd.es

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	SPIOMET
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Film-coated 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	SPIRONOLATTONE
D.3.9.1	CAS number	52-01-7
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB10631MIG
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	50
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	PIOGLITAZONE
D.3.9.1	CAS number	111025-46-8
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB09857MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	7500
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METFORMINA
D.3.9.1	CAS number	657-24-9
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB08831MIG
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	850
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	SPIO
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Film-coated 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	PIOGLITAZONE
D.3.9.1	CAS number	111025-46-8
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB09857MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	7500
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	SPIRONOLATTONE
D.3.9.1	CAS number	52-01-7
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB10631MIG
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	50
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	PIO
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Film-coated 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	PIOGLITAZONE
D.3.9.1	CAS number	111025-46-8
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB09857MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	7500
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Polycystic Ovary Syndrome (PCOS)

Sindrome dell'Ovaio Policistico (PCOS)

E.1.1.1

Medical condition in easily understood language

Polycystic Ovary Syndrome (PCOS)

Sindrome dell'Ovaio Policistico (PCOS)

E.1.1.2

Therapeutic area

Diseases [C] - Female diseases of the urinary and reproductive systems and pregancy complications [C13]

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	10036049
E.1.2	Term	Polycystic ovaries
E.1.2	System Organ Class	10038604 - Reproductive system and breast disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To test the efficacy of SPIOMET in normalising ovulation in adolescents and young adult women with PCOS.

Testare l'efficacia di SPIOMET nel normalizzare il tasso di ovulazione in adolescenti e giovani adulte con PCOS.

E.2.2

Secondary objectives of the trial

To test the efficacy of SPIOMET in normalising the endocrine-metabolic status, body composition and abdominal fat distribution, on-treatment and post-treatment; to assess the safety of SPIOMET and the adherence and subjective acceptability, and the quality of life of participating subjects.

Testare l’efficacia di SPIOMET nel normalizzare lo stato endocrino-metabolico, descrivere il profilo di sicurezza del farmaco e valutare l'aderenza e l'accettabilità soggettiva, nonché la qualità della vita dei soggetti partecipanti.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Age range within the AYAs category (> 12.0 years and <= 23.9 years at study start);
2. Gynaecological age of 2 years or more;
3. Clinical androgen excess, as defined by the presence of hirsutism (modified Ferriman-Gallwey score >= 4) and/or inflammatory acne (Leeds scale) unresponsive to medications. The scarce normative data existing in adolescents suggest that an adult level of hirsutism is reached around 2 years after menarche;
4. Biochemical androgen excess, as defined by increased total testosterone (>=50 ng/dL), and/or a FAI higher than 3.5 [FAI, total testosterone (nmol/L) x 100/SHBG (nmol/L)], in the follicular phase of the cycle (days 3–7) or after 2 months of amenorrhea;
5. Menstrual irregularity, as defined by <= 8 menses per year corresponding to an average intermenstrual time of >=45 days;
6. Written informed consent obtained from the patient, or assent from the patient and consent by the parents or the legally acceptable representative if she is a minor

1. Fascia di età all'interno della categoria adolescente e giovane adulto (> 12,0 anni e <= 23,9 anni all'inizio dello studio);
2. Età ginecologica di 2 anni o più;
3. Eccesso clinico di androgeni, definito dalla presenza di irsutismo (punteggio Ferriman-Gallwey modificato >= 4) e/o acne infiammatoria (scala di Leeds) che non risponde ai farmaci. Gli scarsi dati normativi esistenti negli adolescenti suggeriscono che un livello di irsutismo da adulto si raggiunga circa 2 anni dopo il menarca;
4. Eccesso di androgeni biochimici, definito come un aumento del testosterone totale (>=50 ng/dL) e/o un FAI superiore a 3,5 [FAI, testosterone totale (nmol/L) x 100/SHBG (nmol/L)], durante la fase follicolare del ciclo (giorni 3-7) o dopo 2 mesi di amenorrea;
5. Irregolarità mestruale, definita come <= 8 mestruazioni all'anno corrispondenti ad un tempo intermestruale medio di >=45 giorni;
6. Consenso informato scritto ottenuto dal paziente, o assenso del paziente e consenso dei genitori o del rappresentante legalmente riconosciuto se è minorenne

E.4

Principal exclusion criteria

1. Class II obesity or morbid obesity (BMI of 35 kg/m2 or higher) according to published international standard charts for both adolescents and young women;
2. Underweight (BMI <18.5 kg/m2) and eating disorders (anorexia nervosa);
3. Anaemia or bleeding disorder;
4. Evidence of thyroid, liver, or kidney dysfunction;
5. Precocious puberty [breast development before age 8 yr and/or precocious menarche (menarche before age 10.0 yr);
6. Cushing syndrome;
7. Late-onset adrenal hyperplasia due to 21-hydroxylase deficiency [17-hydroxyprogesterone
levels >200 ng/dL in the follicular phase of the cycle or after 2 months of amenorrhea];
8. Hyperprolactinaemia;
9. Glucose intolerance or diabetes mellitus;
10. Use of medications affecting gonadal or adrenal function, or carbohydrate or lipid metabolism in the previous three months (including OCs);
11. Gynaecological age < 2.0 years;
12. Positive pregnancy test;
13. Pregnancy risk (failure to guarantee the use of non-hormonal contraception in sexually active subjects).

1. Obesità di classe II o obesità patologica (BMI di 35 kg/m2 o superiore) secondo i diagrammi standard internazionali per adolescenti e giovani donne pubblicati;
2. Sottopeso (BMI <18,5 kg/m2) e disturbi del comportamento alimentare (anoressia nervosa);
3. Anemia o disturbo emorragico;
4. Evidenza di disfunzione tiroidea, epatica o renale;
5. Pubertà precoce [sviluppo del seno prima degli 8 anni e/o menarca precoce (menarca prima dei 10,0 anni)];
6. Sindrome di Cushing;
7. Iperplasia surrenalica a esordio tardivo da deficit di 21-idrossilasi [livelli di 17-idrossiprogesterone >200 ng/dL nella fase follicolare del ciclo o dopo 2 mesi di amenorrea];
8. Iperprolattinemia;
9. Intolleranza al glucosio o diabete mellito;
10. Uso di farmaci che influiscono sulla funzione gonadica o surrenalica, o sul metabolismo dei carboidrati o dei lipidi nei tre mesi precedenti (compresi i contraccettivi orali);
11. Età ginecologica < 2,0 anni;
12. Test di gravidanza positivo;
13. Rischio gravidanza (mancata garanzia dell'uso di contraccettivi non ormonali nei soggetti sessualmente attivi).

E.5 End points

E.5.1

Primary end point(s)

On-treatment and post-treatment ovulation rate.

Tasso di ovulazione al trattamento e post-trattamento.

E.5.1.1

Timepoint(s) of evaluation of this end point

Following end of each two 12-week on-treatment periods (month 0-3 and month 9-12), and following the end of post-treatment period (month 12-15).

Alla fine di ciascuno dei due periodi di trattamento di 12 settimane (mesi 0-3 e mesi 9-12) e alla fine del periodo di post-trattamento (mesi 12-15).

E.5.2

Secondary end point(s)

Endocrine-metabolic variables:
- Circulating androgens: total testosterone, SHBG, free androgen index (FAI), androstenedione
- Lipids - total cholesterol, low-density lipoprotein (LDL-cholesterol), high-density lipoprotein (HDL- cholesterol), triglycerides
- Insulinaemia: fasting and 2 hours after a 75-gr oral glucose load [oral glucose tolerance test (oGTT). Estimation of insulin resistance from fasting insulin and glucose levels using the homeostasis model
assessment (HOMA)
- Markers of inflammation & insulin sensitivity: ultra-sensitive C-reactive protein (us-CRP); growth-and- differentiation factor-15 (GDF15); high molecular weight adiponectin (HMW-adip), C-X-C motif chemokine ligand
14 (CXCL14); Epigenetic variable: circulating microRNA 451-a (miR-451a) concentrations; Imaging variables:
- Cardiovascular risk – cIMT (ultrasound)
- Body composition: dual-energy X-ray absorptiometry (DXA)
- Abdominal fat distribution (subcutaneous and visceral) and hepatic fat (MRI);; Lifestyle assessment:
- Body composition, abdominal fat distribution (waist circumference, Waist to hip ratio (WHR), and hepatic fat by MRI) and weight status
- Improvement of co-morbidities and health behaviour (SF-36, PCOSQ, modified self reported health behaviour in school-aged children questionnaire (HBSC-Q)
- Minimisation of adverse side effects (i.e., "Sick-Control-One-Fat-Food" (SCOFF) + Binge Eating Disorder Screener 7 (BEDS-7) questionnaires to assess the risk of eating disorders; of risk confirmed, then, the EDE-Q will be used)
- Improvement of HRQoL; Safety variables:
- Blood count, electrolyte panel, urea, alanine transaminase (ALT), aspartate transaminase (AST), gamma-glutamyltransferase (GGT), creatinine, vitamin B12 and folic acid
- Report of AEs; Adherence and acceptability:
- Adherence will be calculated as the ratio between the number of tablets prescribed and dispensed for the period between hospital appointments and the number of tablets returned by the patient at the following appointment
- Acceptability of the tablet by the study patients; PROMs & HRQoL: generic (SF-36) and specific (PCOSQ) questionnaires; Clinical variables:
- hirsutism (modified Ferriman & Gallwey score)
- Menstrual regularity
- Acne (evaluated using the Leeds Acne Grading Scale)

Variabili endocrino-metaboliche:
- Androgeni circolanti: testosterone totale, SHBG, indice degli androgeni liberi (FAI), androstenedione
- Lipidi: colesterolo totale, lipoproteine a bassa densità (LDL), lipoproteine ad alta densità (HDL), trigliceridi
- Insulinemia: a digiuno e 2 ore dopo un carico orale di glucosio [test orale di tolleranza al glucosio (oGTT)]. Stima della resistenza all’insulina derivante dai livelli di insulina e glucosio a digiuno utilizzando il modello omeostatico di valutazione della resistenza all'insulina (IR-HOMA)
- Marcatori di infiammazione e sensibilità all’insulina: proteina C-reattiva ultrasensibile (us-CRP); fattore di crescita e differenziazione-15 (GDF15); adiponectina ad alto peso molecolare (HMW-adip), C-X-C motif chemokine ligand 14 (CXCL14); Variabile epigenetica: concentrazioni di MicroRNA 451a (miR-451a) circolante; Variabili di imaging:
- Rischio cardiovascolare; spessore intima-media della carotide (cIMT) (ecografia)
- Composizione corporea: densiometria ossea a raggi X (DEXA)
- Distribuzione del grasso addominale (sottocutaneo e viscerale) e grasso epatico (risonanza magnetica, RM); Valutazione dello stile di vita:
- Composizione corporea, distribuzione del grasso addominale (circonferenza della vita, rapporto vita-fianchi e grasso epatico tramite risonanza magnetica) e stato del peso
- Miglioramento delle comorbidità e del comportamento della salute
- Minimizzazione degli effetti collaterali avversi (per esempio, disturbi alimentari)
- Miglioramento della qualità della vita relativa alla salute (HRQoL); Variabili di sicurezza:
- Emocromo, pannello elettrolitico, urea, alanina aminotransferasi (ALT), aspartato aminotransferasi (AST), gamma-glutamiltransferasi (GGT), creatinina, vitamina B12 e acido folico
- Segnalazione di AEs; Aderenza e accettabilità:
- L’aderenza, calcolata come il rapporto tra il numero di compresse prescritte e dispensate per il periodo tra gli appuntamenti in ospedale e il numero di compresse restituite dal paziente all’appuntamento successivo
- Accettabilità della compressa da parte dei pazienti dello studio; PROMs su HRQoL: sondaggio generico sulla salute in forma breve 36 (SF-36) e questionari sulla qualità della vita relativa alla salute PCOS (PCOSQ); Variabili cliniche:
- Irsutismo (scala Ferriman & Gallwey modificata)
- Regolarità mestruale
- Acne (valutata utilizzando la Leeds Acne Grading Scale)

E.5.2.1

Timepoint(s) of evaluation of this end point

As defined per protocol.; As defined per protocol.; As defined per protocol.; As defined per protocol.; As defined per protocol.; As defined per protocol.; As defined per protocol.; As defined per protocol.

Come definito da protocollo.; Come definito da protocollo.; Come definito da protocollo.; Come definito da protocollo.; Come definito da protocollo.; Come definito da protocollo.; Come definito da protocollo.; Come definito da protocollo.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

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)

E.8.2.2

Placebo

Yes

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

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

Turkey

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

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

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1.1

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

180

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

184

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

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

Paediatric patients

Pazienti pediatrici

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

322

F.4.2.2

In the whole clinical trial

364

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

2022-03-28

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-05-18

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

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Orphan Designation Number:
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