search
Back to results

Toxin Treatment for Amyotrophic Lateral Sclerosis (ALS) Related Sialorrhea (ALS-TOX)

Primary Purpose

Sialorrhea, Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Botox injection
Placebo injection
Sponsored by
Hospices Civils de Lyon
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sialorrhea focused on measuring Sialorrhea, Amyotrophic lateral sclerosis, Botulinum toxin

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Age > 18 years
  • Obtaining of a written consent after information
  • Diagnosis of probable or certain ALS according to the El Escorial criteria of the World Federation and Neurology Committee on Neuromuscular Diseases
  • Patient having a follow-up in an ALS center
  • Sialorrhea with VAS functional embarrassment > or equal at 50/100.
  • Patient beneficiary of Social Security regime

Exclusion Criteria:

  • Evolving disease associated with predictable survival < 1 month
  • Patient having previously received an injection of botulinum toxin in the salivary glands
  • Patient taking the other medical treatments for sialorrhea in the 7 days before the inclusion in the study (scopoderm, trihexyph'nidyle, atropine, ipatropium, amitriptyline, clomipramine, oxybutinine, diphenhydramine, beta-blockers)
  • Patient having benefited from radiotherapy or from surgery on the salivary glands
  • Behavioral problems, dementia or other psychiatric problems
  • Myasthenia
  • Known Pregnancy or absence of contraception recognized as effective, breast feeding

Sites / Locations

  • Département de Neurologie, Hôpital de l'Hôtel-Dieu, CHU d'Angers
  • Centre SLA, Groupement Hospitalier Est, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon
  • Service de Neurologie Vastel, Hôpital Côte de Nacre, CHU de Caen
  • Service de Neurologie, Hôpital Gabriel Montpied, CHU de Clermont-Ferrand
  • Centre SLA, Service de Neurologie A et pathologies du mouvement, Hôpital Roger Salengro, CHU de Lille
  • Service de Neurologie, Hôpital Dupuytren, CHU de Limoges
  • Service de Neurologie, Hôpital de la Timone, CHU de Marseille
  • Service de Neurologie, Hôpital Central, CHU de Nancy
  • Centre de Référence maladies Neuromusculaires, Service de Médecine Physique et Réadaptation, Hôpital de l'Archet 1, CHU de Nice
  • Fédération des Maladies du Système Nerveux, Groupement Hospitalier Pitié Salpêtrière, AP-HP
  • Service de Neurologie, CHU de Saint-Etienne
  • Département de Neurologie, Hôpital de Hautepierre, CHRU de Strasbourg
  • Service d'Explorations Fonctionelles Neuro-Musculaires, Hôpital Rangueil, CHU de Toulouse

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Botox

Placebo

Arm Description

Botox injection : 100 UI of botulinum toxin type A (Botox®) diluted in 2.2 ml of NaCl 0.9 %

Placebo injection : NaCl 0.9 %

Outcomes

Primary Outcome Measures

Improvement of the functional embarrassment provoked by sialorrhea
Demonstrate after the injection of botulinum toxin type A an improvement of at least 25 % of the functional embarrassment provoked by sialorrhea in the ALS patient, evaluated with a horizontal visual analogue scale (VAS).

Secondary Outcome Measures

Decrease of the salivary secretion rate and a decrease of the embarrassment for the main caregiver
Demonstrate, one month after the injection of botulinum toxin type A, a decrease of the salivary secretion rate and a decrease of the embarrassment for the main caregiver by a horizontal visual analogue scale.
Improvement of the value of the hypersalivation item in ALSFRS-R scale
Demonstrate, one month after the injection of botulinum toxin type A, an improvement of the value of the hypersalivation item in ALSFRS-R scale.
Decrease of the score of severity and frequency of the drooling rating scale
Demonstrate, one month after the injection of botulinum toxin type A, a decrease of the score of severity and frequency of the drooling rating scale
Decrease of the cotton roll weight
Demonstrate, one month after the injection of botulinum toxin type A, a decrease of the cotton roll weight. Cotton rolls weight: production and quantity of saliva are verified by placing dental cotton rolls during 3 minutes in the mouth of the patient and by comparing the weight of rolls dry and soaked with saliva.
Decrease of the number of paper handkerchiefs used
Demonstrate, one month after the injection of botulinum toxin type A, a decrease of the number of paper handkerchiefs used. We ask the patient to count the number of handkerchiefs used a day.
Modification of the speech evaluation
Demonstrate, one month after the injection of botulinum toxin typeA, a modification of the speech evaluation (evaluation realized by speech evaluator)
Improvement of the quality of life
Demonstrate, one month after the injection of botulinum toxin type A, an improvement of the quality of life. Scale to estimate quality of life (ALSAQ-40), usually used with the patients affected by ALS
Description of patient cohort after the first injection
Describe cohort after the first injection: evolution of scores for every patient among Day0 and Month1, delay of appearance of the efficiency, the duration of effect of the treatment, the side effects, the modification of the consistency of the saliva and the possible necessity for re-injecting the patient at 3 months of follow-up.

Full Information

First Posted
March 9, 2012
Last Updated
August 13, 2015
Sponsor
Hospices Civils de Lyon
search

1. Study Identification

Unique Protocol Identification Number
NCT01551940
Brief Title
Toxin Treatment for Amyotrophic Lateral Sclerosis (ALS) Related Sialorrhea
Acronym
ALS-TOX
Official Title
Evaluation of Efficacy of Botulinum Toxin Type A in the Treatment of Sialorrhea in the Patient Affected by Amyotrophic Lateral Sclerosis (ALS)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2015
Overall Recruitment Status
Completed
Study Start Date
February 2012 (undefined)
Primary Completion Date
March 2014 (Actual)
Study Completion Date
March 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hospices Civils de Lyon

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Evaluation of the decrease of the secretion of saliva in patients with amyotrophic lateral sclerosis by a local ultrasound-guided bilateral injection of botulinum toxin type A in parotids and submandibular glands. The investigators want to demonstrate 1 month after the injection, by a multicenter French randomized double blind study, an improvement of at least 25 % of the functional embarrassment due to saliva, estimated with a visual analogue scale, a decrease of the quantity of saliva and a decrease of the embarrassment for the main caregiver.
Detailed Description
The patient will benefit from an ultrasound guided injection of botulinum toxin A (Botox®) or placebo (NaCl 0.9 %) and will be followed up in consultation at 4, 12, 16 (if reinjection) and 24 weeks. He will be contacted by telephone in 2 and in 8 weeks (percentage of decrease of functional embarrassment, percentage of decrease of salivary secretion rate). He can be able to benefit in the open label phase of a botulinum toxin type A injection at the 12-week follow up if he estimates that first injection was not effective or if the efficiency of the first injection began to become blurred. After the 6 months of the study, the patient will benefit again from the usual follow-up as advised by the French consensus conference in November, 2005.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sialorrhea, Amyotrophic Lateral Sclerosis
Keywords
Sialorrhea, Amyotrophic lateral sclerosis, Botulinum toxin

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Botox
Arm Type
Experimental
Arm Description
Botox injection : 100 UI of botulinum toxin type A (Botox®) diluted in 2.2 ml of NaCl 0.9 %
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo injection : NaCl 0.9 %
Intervention Type
Drug
Intervention Name(s)
Botox injection
Intervention Description
Local ultrasound-guided bilateral injection of botulinum toxin type A in parotid (35 UI/gland) and submandibular (15 UI/gland) glands.
Intervention Type
Drug
Intervention Name(s)
Placebo injection
Intervention Description
A local ultrasound-guided bilateral injection of placebo (NaCl 0.9 %) in parotid (0.7 ml/gland) and submandibular (0.3 ml/gland) glands.
Primary Outcome Measure Information:
Title
Improvement of the functional embarrassment provoked by sialorrhea
Description
Demonstrate after the injection of botulinum toxin type A an improvement of at least 25 % of the functional embarrassment provoked by sialorrhea in the ALS patient, evaluated with a horizontal visual analogue scale (VAS).
Time Frame
1 month after the injection
Secondary Outcome Measure Information:
Title
Decrease of the salivary secretion rate and a decrease of the embarrassment for the main caregiver
Description
Demonstrate, one month after the injection of botulinum toxin type A, a decrease of the salivary secretion rate and a decrease of the embarrassment for the main caregiver by a horizontal visual analogue scale.
Time Frame
1 month after the injection
Title
Improvement of the value of the hypersalivation item in ALSFRS-R scale
Description
Demonstrate, one month after the injection of botulinum toxin type A, an improvement of the value of the hypersalivation item in ALSFRS-R scale.
Time Frame
1 month after the injection
Title
Decrease of the score of severity and frequency of the drooling rating scale
Description
Demonstrate, one month after the injection of botulinum toxin type A, a decrease of the score of severity and frequency of the drooling rating scale
Time Frame
1 month after the injection
Title
Decrease of the cotton roll weight
Description
Demonstrate, one month after the injection of botulinum toxin type A, a decrease of the cotton roll weight. Cotton rolls weight: production and quantity of saliva are verified by placing dental cotton rolls during 3 minutes in the mouth of the patient and by comparing the weight of rolls dry and soaked with saliva.
Time Frame
1 month after the injection
Title
Decrease of the number of paper handkerchiefs used
Description
Demonstrate, one month after the injection of botulinum toxin type A, a decrease of the number of paper handkerchiefs used. We ask the patient to count the number of handkerchiefs used a day.
Time Frame
1 month after the injection
Title
Modification of the speech evaluation
Description
Demonstrate, one month after the injection of botulinum toxin typeA, a modification of the speech evaluation (evaluation realized by speech evaluator)
Time Frame
1 month after the injection
Title
Improvement of the quality of life
Description
Demonstrate, one month after the injection of botulinum toxin type A, an improvement of the quality of life. Scale to estimate quality of life (ALSAQ-40), usually used with the patients affected by ALS
Time Frame
1 month after the injection
Title
Description of patient cohort after the first injection
Description
Describe cohort after the first injection: evolution of scores for every patient among Day0 and Month1, delay of appearance of the efficiency, the duration of effect of the treatment, the side effects, the modification of the consistency of the saliva and the possible necessity for re-injecting the patient at 3 months of follow-up.
Time Frame
6 months after the injection

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age > 18 years Obtaining of a written consent after information Diagnosis of probable or certain ALS according to the El Escorial criteria of the World Federation and Neurology Committee on Neuromuscular Diseases Patient having a follow-up in an ALS center Sialorrhea with VAS functional embarrassment > or equal at 50/100. Patient beneficiary of Social Security regime Exclusion Criteria: Evolving disease associated with predictable survival < 1 month Patient having previously received an injection of botulinum toxin in the salivary glands Patient taking the other medical treatments for sialorrhea in the 7 days before the inclusion in the study (scopoderm, trihexyph'nidyle, atropine, ipatropium, amitriptyline, clomipramine, oxybutinine, diphenhydramine, beta-blockers) Patient having benefited from radiotherapy or from surgery on the salivary glands Behavioral problems, dementia or other psychiatric problems Myasthenia Known Pregnancy or absence of contraception recognized as effective, breast feeding
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Christophe VIAL, MD
Organizational Affiliation
Centre SLA, Groupement Hospitalier Est, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon
Official's Role
Principal Investigator
Facility Information:
Facility Name
Département de Neurologie, Hôpital de l'Hôtel-Dieu, CHU d'Angers
City
Angers cedex 9
ZIP/Postal Code
49933
Country
France
Facility Name
Centre SLA, Groupement Hospitalier Est, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon
City
Bron
ZIP/Postal Code
69677
Country
France
Facility Name
Service de Neurologie Vastel, Hôpital Côte de Nacre, CHU de Caen
City
Caen cedex 9
ZIP/Postal Code
14033
Country
France
Facility Name
Service de Neurologie, Hôpital Gabriel Montpied, CHU de Clermont-Ferrand
City
Clermont-Ferrand cedex 1
ZIP/Postal Code
63003
Country
France
Facility Name
Centre SLA, Service de Neurologie A et pathologies du mouvement, Hôpital Roger Salengro, CHU de Lille
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Service de Neurologie, Hôpital Dupuytren, CHU de Limoges
City
Limoges
ZIP/Postal Code
87042
Country
France
Facility Name
Service de Neurologie, Hôpital de la Timone, CHU de Marseille
City
Marseille cedex 5
ZIP/Postal Code
13385
Country
France
Facility Name
Service de Neurologie, Hôpital Central, CHU de Nancy
City
Nancy
ZIP/Postal Code
54035
Country
France
Facility Name
Centre de Référence maladies Neuromusculaires, Service de Médecine Physique et Réadaptation, Hôpital de l'Archet 1, CHU de Nice
City
Nice cedex 3
ZIP/Postal Code
06202
Country
France
Facility Name
Fédération des Maladies du Système Nerveux, Groupement Hospitalier Pitié Salpêtrière, AP-HP
City
Paris cedex 13
ZIP/Postal Code
75651
Country
France
Facility Name
Service de Neurologie, CHU de Saint-Etienne
City
Saint-Etienne cedex 2
ZIP/Postal Code
42055
Country
France
Facility Name
Département de Neurologie, Hôpital de Hautepierre, CHRU de Strasbourg
City
Strasbourg
ZIP/Postal Code
67091
Country
France
Facility Name
Service d'Explorations Fonctionelles Neuro-Musculaires, Hôpital Rangueil, CHU de Toulouse
City
Toulouse
ZIP/Postal Code
31059
Country
France

12. IPD Sharing Statement

Learn more about this trial

Toxin Treatment for Amyotrophic Lateral Sclerosis (ALS) Related Sialorrhea

We'll reach out to this number within 24 hrs