Clinical Trials Register

Summary
EudraCT Number:	2010-023161-21
Sponsor's Protocol Code Number:	2010.607/11
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2011-09-06
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

France - ANSM

A.2

EudraCT number

2010-023161-21

A.3

Full title of the trial

Evaluation de la toxine botulinique dans le traitement du trouble salivaire chez le patient atteint de sclérose latérale amyotrophique

A.3.1

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

Sclérose Latérale Amyotrophique (SLA) – toxine botulinique A (Botox®)

A.3.2

Name or abbreviated title of the trial where available

SLA-TOX

A.4.1

Sponsor's protocol code number

2010.607/11

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	Hospices Civils de Lyon
B.1.3.4	Country	France
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	Association Française contre les Myopathies
B.4.2	Country	France
B.4.1	Name of organisation providing support	Association pour le Développement de la Neurogénétique
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Hospices Civils de Lyon
B.5.2	Functional name of contact point	PLATTNER valérie
B.5.3	Address:
B.5.3.1	Street Address	3 quai des Célestins
B.5.3.2	Town/ city	Lyon cedex 02
B.5.3.3	Post code	69229
B.5.3.4	Country	France
B.5.4	Telephone number	472 115 213+33
B.5.5	Fax number	472 115 190+33
B.5.6	E-mail	valerie.plattner@chu-lyon.fr

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	BOTOX 100 UNITES ALLERGAN (PR1)
D.2.1.1.2	Name of the Marketing Authorisation holder	ALLERGAN FRANCE
D.2.1.2	Country which granted the Marketing Authorisation	France
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.3.4	Pharmaceutical form	Powder for solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Other use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.1	CAS number	93384-43-1
D.3.9.3	Other descriptive name	BOTULINUM TOXIN TYPE A
D.3.9.4	EV Substance Code	SUB13117MIG
D.3.10	Strength
D.3.10.1	Concentration unit	U unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Other use (Noncurrent)

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Sclérose Latérale Amyotrophique (SLA)

E.1.1.1

Medical condition in easily understood language

Sclérose Latérale Amyotrophique (SLA)

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	14.0
E.1.2	Level	PT
E.1.2	Classification code	10002026
E.1.2	Term	Amyotrophic lateral sclerosis
E.1.2	System Organ Class	10029205 - Nervous system 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

Démontrer une amélioration de la gêne fonctionnelle induite par le trouble salivaire évalué par une EVA gêne présentée au patient, 1 mois après l’injection de Botox

E.2.2

Secondary objectives of the trial

- Démontrer une diminution de la quantité de salive et une amélioration de la gêne pour l’aidant principal 1 mois après l’injection de Botox.
- Démontrer une amélioration du score du critère hypersalivation dans l’échelle ALSFRS-R, une diminution du score de sévérité et de fréquence du trouble salivaire (score DRS), une diminution du poids des rouleaux dentaires imbibées de salive, une modification de l’évaluation orthophonique, une diminution du nombre de mouchoirs en papier utilisés par jour et une amélioration de la qualité de vie du patient mesurée par l’échelle ALSAQ-40 (Cf 4.1 critères dévaluation).
- Comparer le poids des rouleaux dentaires imbibés de salive et la gêne fonctionnelle mesurée par EVA pour évaluer sa place dans le suivi du trouble salivaire chez les patients avec une SLA.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Age > 18 ans
- Obtention d’un consentement éclairé écrit après information
- Diagnostic de SLA “ probable ” ou “ certaine ” selon les critères El Escorial de la “ World Fédération and Neurology Committee on Neuromuscular Diseases ”
- Patient faisant l’objet d’un suivi dans un centre SLA
- Trouble salivaire gênant avec une EVA gêne fonctionnelle > ou égale à 50/100 mm.
- Patient bénéficiaire d’un régime de sécurité sociale.

E.4

Principal exclusion criteria

- Maladie évolutive associée avec survie prévisible < 1 mois
- Patient ayant auparavant reçu une injection de toxine botulinique au niveau des glandes salivaires
- Mise en place récente (moins de 7 jours) d’un autre traitement médicamenteux pour le trouble salivaire (scopolamine, trihexyphénidyle, atropine, ipratropium, amitriptyline, clomipramine, oxybutinine, diphenhydramine, bêta-bloquants)
- Patient ayant bénéficié d’une radiothérapie ou d’une chirurgie au niveau des glandes salivaires accessoires
- Troubles du comportement, démence ou atteinte psychiatrique majeure
- Myasthénie
- Grossesse connue ou absence de contraception reconnue comme efficace, allaitement
- Patients majeurs protégés par la loi.

E.5 End points

E.5.1

Primary end point(s)

Gêne fonctionnelle due au trouble salivaire mesurée sur l’Echelle Visuo-Analogique horizontale (EVA) (mesure de 0 à 100 mm).

E.5.1.1

Timepoint(s) of evaluation of this end point

1 mois après l'injection de Botox

E.5.2

Secondary end point(s)

- Mesure des échelles :
○ EVA horizontale « Quantité de salive »
○ EVA horizontale « Gêne » aidant principal
- Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) : permet d’observer la variation du score de l’item hypersalivation
- Drooling rating scale (DRS) : évaluation de la sévérité et de la fréquence du trouble salivaire
- Pesée des rouleaux dentaires
- Evaluation orthophonique
- Nombre de mouchoirs en papier utilisés / jour
- Echelle de qualité de vie
- Tolérance

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

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

E.8.6.2

Trial being conducted completely outside of the EEA

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

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

F. Population of Trial Subjects
F.1 Age Range
F.1.1	Trial has subjects under 18	No
F.1.1.1	In Utero	No
F.1.1.2	Preterm newborn infants (up to gestational age < 37 weeks)	No
F.1.1.3	Newborns (0-27 days)	No
F.1.1.4	Infants and toddlers (28 days-23 months)	No
F.1.1.5	Children (2-11years)	No
F.1.1.6	Adolescents (12-17 years)	No
F.1.2	Adults (18-64 years)	Yes
F.1.2.1	Number of subjects for this age range:	70
F.1.3	Elderly (>=65 years)	Yes
F.1.3.1	Number of subjects for this age range:	20
F.2 Gender
F.2.1	Female	Yes
F.2.2	Male	Yes
F.3 Group of trial subjects
F.3.1	Healthy volunteers	No
F.3.2	Patients	Yes
F.3.3	Specific vulnerable populations	No
F.3.3.1	Women of childbearing potential not using contraception	No
F.3.3.2	Women of child-bearing potential using contraception	Yes
F.3.3.3	Pregnant women	No
F.3.3.4	Nursing women	No
F.3.3.5	Emergency situation	No
F.3.3.6	Subjects incapable of giving consent personally	No
F.3.3.7	Others	No
F.4 Planned number of subjects to be included
F.4.1	In the member state	90

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

2011-09-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-11-23

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

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