Clinical Trials Register

Summary
EudraCT Number:	2015-004448-20
Sponsor's Protocol Code Number:	Sarizotan/001/II/2015
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:
Date on which this record was first entered in the EudraCT database:	2020-12-14
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

2015-004448-20

A.3

Full title of the trial

A Randomized, Double-Blind, Placebo-Controlled, Six-Month Study to Evaluate the Efficacy, Safety and Tolerability of Sarizotan in Patients with Rett Syndrome with Respiratory Symptoms

Studio randomizzato, in doppio cieco, controllato con placebo, della durata di sei mesi per valutare l¿efficacia, la sicurezza e la tollerabilit¿ di sarizotan in pazienti affetti dalla sindrome di Rett con sintomi respiratori

A.3.1

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

Sarizotan: Treatment in patients with Rett Syndrome with Respiratory Symptoms.

Sarizotan:Trattamento nei pazienti con sindrome di Rett con sintomi respiratori

A.3.2

Name or abbreviated title of the trial where available

Sarizotan: Treatment in patients with Rett Syndrome with Respiratory Symptoms.

Sarizotan: trattamento in pazienti con Sindrome di Rett con sintomi respiratori.

A.4.1

Sponsor's protocol code number

Sarizotan/001/II/2015

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	NEWRON PHARMACEUTICALS SPA
B.1.3.4	Country	Italy
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	Newron Pharmaceuticals S.p.A.
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Newron Pharmaceuticals S.p.A.
B.5.2	Functional name of contact point	Ravi Anand
B.5.3	Address:
B.5.3.1	Street Address	Via Ludovico Ariosto 21
B.5.3.2	Town/ city	Bresso (MI)
B.5.3.3	Post code	20091
B.5.3.4	Country	Italy
B.5.4	Telephone number	0041793741364
B.5.5	Fax number	0041793741364
B.5.6	E-mail	ravi@anand.ch

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	EMA/430803/2015
D.3 Description of the IMP
D.3.1	Product name	Sarizotan hydrochloride
D.3.2	Product code	EMD 128130
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Gastroenteral use Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sarizotan Hydrochloride
D.3.9.1	CAS number	195068-07-6
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	SARIZOTAN HYDROCHLORIDE
D.3.9.4	EV Substance Code	SUB21592
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	5
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	EMA/430803/2015
D.3 Description of the IMP
D.3.1	Product name	sarizotan hydrochloride
D.3.2	Product code	EMD 128130
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Gastroenteral use Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	sarizotan
D.3.9.1	CAS number	195068-07-6
D.3.9.2	Current sponsor code	SARIZOTAN
D.3.9.3	Other descriptive name	SARIZOTAN HYDROCHLORIDE
D.3.9.4	EV Substance Code	SUB21592
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	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
D.3 Description of the IMP
D.3.1	Product name	Sarizotan Cloridrato
D.3.2	Product code	[EMD 128130]
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Gastroenteral use Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sarizotan Cloridrato
D.3.9.1	CAS number	195068-07-6
D.3.9.4	EV Substance Code	SUB21592
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	2
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	Capsule, hard
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

Rett syndrome

Sindrome di Rett

E.1.1.1

Medical condition in easily understood language

Rett syndrome

Sindrome di Rett

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	LLT
E.1.2	Classification code	10039000
E.1.2	Term	Rett's disorder
E.1.2	System Organ Class	100000004850

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 evaluate the effect of sarizotan (2 to 10 mg bid), compared to placebo, on reducing the number of apnea episodes, during awake time, in patients with RTT with respiratory abnormalities.

valutare l'effetto di sarizotan (da 2 a 10 mg bid), rispetto al placebo, sulla riduzione del numero di episodi di apnea, durante il tempo di veglia,
nei pazienti con RTT e con anomalie respiratorie.

E.2.2

Secondary objectives of the trial

Evaluate the effect of sarizotan (2 to 10 mg bid), compared to placebo, on Caregiver-rated Change (CIC) impression from baseline.
Safety: to assess the safety and tolerability of sarizotan (2 to 10 mg).
Efficacy: to evaluate the efficacy of the dosage range of sarizotan, compared to placebo, on the following:
- respiratory symptoms: percentage of time spent with respiratory dysrhythmia per hour; Number of hyperventilation episodes (=10 sec each) per hour; Oxygen saturation; Indication of respiratory distress; Incidence of episodes of dysrhythmia.
- Motor behavior;
- Global change from baseline;
- Cargiver load;
- overall assessment of the symptoms of RTT.
To determine the pharmacokinetic (PK) profile of sarizotan at the doses tested and compare it with the PK profile in adults.

Valutare l'effetto della sarizotan (2 a 10 mg bid), rispetto al placebo, sull'impressione Caregiver-rated di Change (CIC) rispetto al basale.
Sicurezza: per valutare la sicurezza e la tollerabilità di sarizotan (da 2 a 10 mg).
Efficacia: per valutare l'efficacia del range di dosaggio di sarizotan, rispetto al placebo, sui seguenti:
- sintomi respiratori: percentuale di tempo speso con disritmia respiratoria all'ora; Numero di episodi di iperventilazione (=10 sec ciascuno) per ora; Saturazione di ossigeno; Indicidenza di angoscia respiratoria; Incidenza di episodi di disritmia.
- Il comportamento motorio;
- Cambiamento globale rispetto al basale;
- Carico del caregiver;
- valutazione complessiva dei sintomi di RTT.
Per determinare il profilo farmacocinetico (PK) di sarizotan alle dosi testate e confrontarlo con il profilo PK negli adulti.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

The patient must meet all the following inclusion criteria in order to be eligible for study enrollment:
demography
1. Female or male = 4 years of age.
2. body weight = 10 kg, and within the expected range for a patient with RTT syndrome, based on age and height.
Diagnostic
3.Diagnosis of Rett syndrome based on clinical criteria of consent; a test for MECP2 mutations (Xq28), will be performed at the time of screening if the results
from an accredited laboratory are not available; the selection for the process is non-contiguous on the test result of MECP2. Patients with known duplications of MECP2 will not be eligible.
4.One or more of the following respiratory dysfunctions: periodic apnea during waking; intermittent hyperventilation; affective spasms; swallowing of the air; forced expulsion of air or saliva.
5.the patient meets all the following criteria related to abnormal breathing:
to. report of 10 or more episodes of abnormality per day while breathing during the vigil in the week preceding the screening visit;
b.The time spent on normal breathing is less than 90% of the total
Time per waking time (ie =10% of the time should be characterized by abnormal breathing);
c.He has at least 10 episodes of respiratory adisritmia, defined by episodes =10 seconds of breath holding (apnea), for now during cardiorespiratory monitoring (performed with home / ambulatory monitoring system during the screening period).
6. Therapeutic regime Stable for 4 weeks before starting the study (if the services they receive - physical, occupational or speech therapy - the subjects
they must be in a stable regime of these services for 3 months before starting the study). Female patients of childbearing potential must use an appropriate method of contraception, as recommended by their health care provider.
Procedural
7.genitor / legal guardian / representative provided written consent
before the patient can participate in the study. Where possible, consent or
assent for patients under the age of 18, was also provided by the patient.
8. Ability to take the study medication provided either in capsules or combined with food / drink.
9.the patient cooperates, has the will to complete all aspects of the study, and is able to do so with the assistance of a caregiver.
10.The Caregiver is able to understand the instructions and to participate fully.

E.3 Criteri di inclusione principali(elencare i più importanti): Il paziente deve soddisfare tutti i seguenti criteri di inclusione per poter beneficiare dell'arruolamento nello studio:
demografia
1.Femmina o maschio = 4 anni di età.
2. peso corporeo = 10 kg, e entro il range aspettato per un paziente affetto da sindrome di RTT, sulla base di età e altezza.
Diagnostico
3.Diagnosis della sindrome di Rett basa su criteri clinici di consenso; un test per le mutazioni MECP2 (Xq28), sarà eseguito al momento dello screening se i risultati
da un laboratorio accreditato non sono disponibili; la selezione per il processo è non contigente sul risultato di test di MECP2. I pazienti con note duplicazioni di MECP2non saranno ammissibili.
4.One o più delle seguenti disfunzioni respiratorie: apnea periodicadurante la veglia; iperventilazione intermittente; spasmi affettivi; deglutizione dell'aria; espulsione forzata di aria o saliva.
5.il paziente soddisfa tutti i seguenti criteri relativi alle anomalie della respirazione:
a. rapporto di 10 o più episodi di anomalia al giorno respirando durante la veglia nella settimana precedente la visita di screening;
b.il tempo all'ora speso per la respirazione normale è inferiore al 90% del totale
Tempo per ora di veglia (cioè =10% del tempo dovrebbe essere caratterizzato da respirazione anormale);
c.Ha almeno 10 episodi di adisritmia respiratoria, definiti da episodi =10 secondi di respiro tenuta (apnea), per ora durante il monitoraggio cardiorespiratorio (eseguita con sistema di monitoraggio casa / ambulatoriale durante il periodo di screening).
6. Regime terapeutico Stabile per 4 settimane prima di iniziare lo studio (se i servizi che ricevono – fisici, occupazionali o terapia del linguaggio - i soggetti
devono essere in un regime stabile di questi servizi per 3 mesi prima di cominciare lo studio). Le pazienti di sesso femminile in età fertile devono usare un adeguato metodo contraccettivo, come raccomandato dal loro fornitore di assistenza sanitaria.
Procedurale
7.genitore / tutore legale / rappresentante ha fornito il consenso scritto
prima che il paziente possa partecipare allo studio. Ove possibile, il consenso o
assenso per pazienti con meno di 18 anni, è stato anche fornito dal paziente.
8.Abilità di prendere il farmaco in studio fornito o in capsule o combinato con cibo / bevande.
9.il paziente coopera, ha volontà di completare tutti gli aspetti dello studio, ed è in grado di farlo con l'assistenza di un caregiver.
10.Il Caregiver è in grado di comprendere le istruzioni e di partecipare pienamente.

E.4

Principal exclusion criteria

The presence of one of the following will exclude a patient from the study
1. To meet any of the diagnostic exclusion criteria for Rett syndrome,
typical;
2.The patient participates in a clinical trial with another investigational drug or has taken an experimental drug within a month or 5 half-lives
(Depending on which is longer) before screening;
3. hypersensitivity to saritozan or other 5-HT1a agonists;
4. Clinically significant current (as determined by the Investigator) a) cardiovascular, respiratory (eg severe asthma), gastrointestinal, renal, hepatic, hematological or other medical disorders, in addition to those directly related to the patient's Rett syndrome;
5. QTcF interval to ECG is greater than 450 msec.
6. Surgery scheduled during the study (except for the insertion of gastrostomy)
7. severe diabetes mellitus or fatty acid oxidation disorders.
8. Ophthalmologic history including any of the following conditions: albino patients, family history of inherited diseases of the retina, retinitis pigmentosa, any active retinopathy or severe diabetic retinopathy.
9. Women who are pregnant, breastfeeding, or of child-bearing age who do not use adequate contraception, as recommended by their health care provider.
10. Evidence of clinically significant malnutrition.

E.4 Criteri di esclusione principali(elencare i più importanti): La presenza di una delle seguenti escluderà un paziente dallo studio
1.Incontrare nessuno dei criteri di esclusione diagnostici per la sindrome di Rett,
Tipici;
2.Il paziente partecipa a una sperimentazione clinica con un altro farmaco in fase di sperimentazione o ha assunto un farmaco sperimentale entro un mese o 5 emivite
(A seconda di quale è più lungo) prima dello screening;
3. ipersensibilità al saritozan o ad altri agonisti 5-HT1a;
4. Corrente clinicamente significativo (come determinato dall’Investigator) a) cardiovascolare, respiratoria (ad esempio asma grave), gastrointestinali, renali, epatiche, ematologiche o di altri disturbi medici, oltre a quelli direttamente legati alla sindrome di Rett del paziente;
5. Intervallo QTcF all'ECG è maggiore di 450 msec.
6.Chirurgia prevista durante lo studio (ad eccezione per l'inserimento di gastrostomia)
7. diabete mellito grave o disturbi di ossidazione degli acidi grassi.
8. storia oftalmologica inclusa qualsiasi delle seguenti condizioni: pazienti albini, storia familiare di malattie ereditarie della retina, la retinite pigmentosa, qualsiasi retinopatia attiva o grave retinopatia diabetica.
9.Donne in gravidanza, allattamento, o in età potenzialmente fertile che non utilizzano un adeguato contraccettivo, come raccomandato dal loro health care provider.
10. Evidenza di malnutrizione significante clinicamente.

E.5 End points

E.5.1

Primary end point(s)

Percent reduction in the number of apnea episodes (each =10 seconds in
duration) per hour, during awake time.

Riduzione percentuale del numero di episodi di apnea (>=10 sec. di durata ciascuno) all’ora, durante la veglia

E.5.1.1

Timepoint(s) of evaluation of this end point

Throughout all the study duration

Durante tutta la durata dello studio

E.5.2

Secondary end point(s)

Secondary Efficacy Outcome - global assessment performed by the
caregiver
Other Secondary Efficacy Outcome - Respiratory Measures; Heart beat
variables; Motor-Behavioral Assessment Scale; Clinical Global
Impression of Change (CGI-C); Caregiver Top 3 Concerns; Rett
Syndrome Clinical Severity Scale (RCSS)

Endpoint di Efficacia secondario - valutazione globale effettuata dal caregiver
Altri Endpoint di Efficacia secondari- Misure respiratorie; variabili di battito cardiaco; Scala di valutazione motorio-comportamentale (Motor-Behavioral Assessment Scale); Impressione clinica globale di cambiamento (Clinical Global
Impression of Change, CGI-C); 3 principali difficolt¿ del caregiver (Caregiver Top 3 Concerns); scala di gravit¿ clinica della sindrome di Rett (Rett
Sindrome clinica Severity Scale, RCSS)

E.5.2.1

Timepoint(s) of evaluation of this end point

For efficacy outcome: Throughout the entire study duration
For PK: prior dosing and at approximately 1 and 4 hr post-dose on Day 1
and on Day 15

Per risultati di efficacia: per l'intera durata di studio
Per PK: dosaggio precedente e circa 1 e 4 ore dopo la somministrazione al giorno 1 e al giorno 15

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

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

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

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

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

India

United Kingdom

United States

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:

100

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

incapacitated subjects due to their clinical condition

soggetti inabili a causa della loro condizione clinica

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

129

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)

Patients who complete 12 months (48 weeks) of double-blind treatment and are doing well on the study medication will be eligible to continue treatment with sarizotan in a separate open-label extension study.

I pazienti che completano 12 mesi (48 settimane) di trattamento in doppio cieco e stanno bene con farmaco in studio avranno diritto di continuare il trattamento con sarizotan in uno studio separato di estensione in aperto.

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

2016-09-07

Ethics Committee Opinion of the trial application

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

P. End of Trial
P.	End of Trial Status

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