Clinical Trials Register

Summary
EudraCT Number:	2018-000282-37
Sponsor's Protocol Code Number:	AIFA-2016-02365063
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:	2021-06-17
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

2018-000282-37

A.3

Full title of the trial

Riluzole (Glentek) in patients with SpinoCerebellar Ataxia type 7: a randomized, double-blind, placebo-controlled pilot trial with a lead in phase

Studio pilota randomizzato, doppio cieco, controllato secondo placebo con una fase iniziale di osservazione sull' utilizzo del riluzolo (Glentek) in pazienti affetti da Atassia Spinocerebellare di tipo 7

A.3.1

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

Riluzole in spinocerebellar ataxia type 7

Riluzolo in pazienti affetti da Atassia Spinocerebellare di tipo 7

A.3.2

Name or abbreviated title of the trial where available

RISCA7

A.4.1

Sponsor's protocol code number

AIFA-2016-02365063

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	DIPARTIMENTO DI NEUROSCIENZE SALUTE MENTALE E ORGANI DI SENSO - NESMOS - SAPIENZA UNIVERSITÀ DI ROMA
B.1.3.4	Country	Italy
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	AIFA - Italian Medicines Agency
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	Università La Sapienza
B.5.2	Functional name of contact point	Dipartimento NESMOS
B.5.3	Address:
B.5.3.1	Street Address	Via di Grottarossa, 1037
B.5.3.2	Town/ city	Roma
B.5.3.3	Post code	00189
B.5.3.4	Country	Italy
B.5.4	Telephone number	0633776044
B.5.5	Fax number	0633775076
B.5.6	E-mail	silvia.romano@uniroma1.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	Yes
D.2.1.1.1	Trade name	Glentek 50 mg film-coated tablets
D.2.1.1.2	Name of the Marketing Authorisation holder	Glenmark Pharmaceuticals Europe Limited
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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	Glentek 50 mg compresse rivestite con film
D.3.2	Product code	[Glentek 50 mg compresse rivestite con film]
D.3.4	Pharmaceutical form	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	Riluzolo
D.3.9.1	CAS number	1744-22-5
D.3.9.2	Current sponsor code	Riluzolo
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.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

Spinocerebellar ataxia type 7 (SCA7)

Atassia spinocerebellare di tipo 7 (SCA7)

E.1.1.1

Medical condition in easily understood language

Spinocerebellar ataxia type 7 (SCA7)

Atassia spinocerebellare di tipo 7 (SCA7)

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

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	10029205
E.1.2	Term	Nervous system disorders
E.1.2	System Organ Class	10029205 - Nervous system 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

To compare the two study arms for the proportion of patients who remain stable at SARA score and visual acuity at 18 months respect to run-in.

Confrontare i due bracci dello studio per la proporzione di pazienti che rimangono stabili al punteggio SARA e acuità visiva a 18 mesi rispetto al run-in

E.2.2

Secondary objectives of the trial

To evaluate the effect of riluzole on visual function using quantitative ophthalmologic assessments and on SARA score, as continuous values, assessing changes at 18 months compared to run-in.
To investigate the safety and tolerability of riluzole administered in SCA 7 patients.

Valutare l'effetto del riluzolo sulla funzione visiva valutata attraverso parametri neuroftalmologici quantitativi e sul punteggio SARA, come valori continui, valutando i cambiamenti a 18 mesi rispetto al run-in.
Valutare la sicurezza e tollerabilità del riluzolo nei soggetti affetti da atassia spinocerebellare di tipo 7.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1) Male and female of any race and > 6 years old; 2) Positive genetic test for SCA7; 3) Signed Informed Consent (in case of minors, written informed consent must be obtained by parents or legal representative).

1) Maschi e femmine di ogni razza e di età maggiore di 6 anni; 2) test genetico positivo per SCA 7; 3) firma del consenso informato (in caso di minori, consenso informato firmato dai genitori o dal rappresentante legale).

E.4

Principal exclusion criteria

1) Female subjects: pregnant or lactating women cannot participate in the study. Women of childbearing potential cannot participate unless willing to use highly effective contraception methods as combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable; intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomised partner; sexual abstinence. In case of use of oral contraception, women should have been stable on the same pill for a minimum of 3 months before taking study drug. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception. 2.) Subjects with a clinically significant or unstable medical or surgical condition that would preclude safe and complete study participation. Such conditions may include cardiovascular, pulmonary, hepatic, renal, severe systemic mycotic infections, metabolic diseases or malignancies; 3) Hepatic diseases with serum values of alanine aminotransferase, aspartate aminotransferase or bilirubin > 1·5 times above normal limit 4) Any medical or psychiatric condition that may affect the subject ability to give informed consent, or to complete the study, or if the subject is considered by the treating neurologist to be, for any other reason, an unsuitable candidate for this study; 5)Known hypersensitivity to any component of riluzole (Glentek).

1) Soggetti femminili: donne in gravidanza o in allattamento non possono partecipare allo studio. Le donne in età fertile devono assicurare l’uso di contraccezione ormonale combinata (estrogeno e progestinico) associata a inibizione dell'ovulazione (orale, intravaginale o transdermica); contraccezione ormonale progestinica associata a inibizione dell'ovulazione (orale, iniettabile o impiantabile, dispositivo intrauterino (IUD), sistema di rilascio ormonale intrauterino (IUS); chiusura tubarica bilaterale; partner vasectomizzato; astinenza sessuale per tutta la durata dello studio ; 2) qualsiasi condizione medica o chirurgica importante che a giudizio dello sperimentatore comprometta la partecipazione allo studio; 3) patologia epatica con valori dell’alanina aminotransferasi, aspartato aminotransferasi o bilirubina > 1,5 volte i valori normali; 4) Qualsiasi condizione medica o psichiatrica che possa influire sulla capacità del soggetto di dare il consenso informato, o di completare lo studio, o se il soggetto è considerato dal neurologo, per qualsiasi altro motivo, un candidato non idoneo per questo studio; 5) ipersensibilità o allergia ai componenti del riluzolo (Glentek).

E.5 End points

E.5.1

Primary end point(s)

The primary endpoints will be the proportion of patients with stable SARA score and visual acuity expressed as log MAR units at 18 months, in comparison with the same parameters as mean of t0-t3-t6 evaluations .

Gli endpoint primari saranno la proporzione di pazienti con stabilità del punteggio SARA e dell’acuità visiva (in unità MAR log) a 18 mesi, rispetto ai valori medi delle valutazioni a 0-3-6 mesi.

E.5.1.1

Timepoint(s) of evaluation of this end point

Months: 0, 3, 6, 18

Mesi: 0, 3, 6, 18

E.5.2

Secondary end point(s)

The secondary endpoint will be quantitative ophthalmologic assessments (via a Farnsworth D15 Arrangement Test, Visual evoked, Electroretinography, Optical Coherence tomography, Computerized visual field examination) and SARA score as continuous values at 18 months, in comparison with the same parameters calculated for each patient as mean of t0-t3-t6 evaluations; The safety profile will be assessed through the recording, reporting and analyzing of baseline medical conditions, adverse events, physical examination findings including laboratory tests.

Gli end point secondari saranno le valutazioni quantitative neuroftalmologiche (Farnsworth D15 Arrangement Test, potenziali evocati visivi, elettroretinogramma, OCT, campo visivo computerizzato) e i punteggi della scala SARA come variabili continue a 18 mesi confrontati con gli stessi parametri calcolati per ogni paziente come medie dei punteggi a t0-t3-t6.; Il profilo di sicurezza sarà valutato attraverso la registrazione, la segnalazione e l'analisi delle condizioni mediche di base, gli eventi avversi, i risultati dell'esame fisico compresi i test di laboratorio.

E.5.2.1

Timepoint(s) of evaluation of this end point

Months: 0, 3, 6, 18; Months: 3, 6, 9, 12, 15, 18

Mesi: 0, 3, 6, 18; Mesi: 3, 6, 9, 12, 15, 18

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

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

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

United States

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

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)

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Subjects under age

Minori

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

Nessuno

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	Centro Nazionale Malattie Rare - ISS
G.4.3.4	Network Country	Italy

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2019-10-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-02-26

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

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