SialorrheaPhase 2ALS-TOXNCT01551940
Toxin Treatment for Amyotrophic Lateral Sclerosis (ALS) Related Sialorrhea
A Phase 2 study testing Botox injection in people with Sialorrhea and Amyotrophic Lateral Sclerosis, run by Hospices Civils de Lyon at 13 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sialorrhea, Amyotrophic Lateral Sclerosis
- What is being tested?
- Botox injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 13 sitesFrance
- Study dates
- Feb 2012to Mar 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
France13 sites
Département de Neurologie, Hôpital de l'Hôtel-Dieu, CHU d'Angers
Angers, 49933
Centre SLA, Groupement Hospitalier Est, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon
Bron, 69677
Service de Neurologie Vastel, Hôpital Côte de Nacre, CHU de Caen
Caen, 14033
Service de Neurologie, Hôpital Gabriel Montpied, CHU de Clermont-Ferrand
Clermont-Ferrand, 63003
Centre SLA, Service de Neurologie A et pathologies du mouvement, Hôpital Roger Salengro, CHU de Lille
Lille, 59037
Service de Neurologie, Hôpital Dupuytren, CHU de Limoges
Limoges, 87042
Service de Neurologie, Hôpital de la Timone, CHU de Marseille
Marseille, 13385
Service de Neurologie, Hôpital Central, CHU de Nancy
Nancy, 54035
Centre de Référence maladies Neuromusculaires, Service de Médecine Physique et Réadaptation, Hôpital de l'Archet 1, CHU de Nice
Nice, 06202
Fédération des Maladies du Système Nerveux, Groupement Hospitalier Pitié Salpêtrière, AP-HP
Paris, 75651
Service de Neurologie, CHU de Saint-Etienne
Saint-Etienne, 42055
Département de Neurologie, Hôpital de Hautepierre, CHRU de Strasbourg
Strasbourg, 67091
Service d'Explorations Fonctionelles Neuro-Musculaires, Hôpital Rangueil, CHU de Toulouse
Toulouse, 31059
About this study
Official title: Evaluation of Efficacy of Botulinum Toxin Type A in the Treatment of Sialorrhea in the Patient Affected by Amyotrophic Lateral Sclerosis (ALS)
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.
Read the 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.
- Conditions
- Sialorrhea, Amyotrophic Lateral Sclerosis
- Being tested
- Botox injection, Placebo injection
- Keywords
- Sialorrhea, Amyotrophic lateral sclerosis, Botulinum toxin
- Medical subject headings
- Sialorrhea, Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Botox injectionDrug
Local ultrasound-guided bilateral injection of botulinum toxin type A in parotid (35 UI/gland) and submandibular (15 UI/gland) glands.
Placebo injectionDrug
A local ultrasound-guided bilateral injection of placebo (NaCl 0.9 %) in parotid (0.7 ml/gland) and submandibular (0.3 ml/gland) glands.
| Study group | Role | What they receive |
|---|---|---|
| Botox | Experimental | Botox injection : 100 UI of botulinum toxin type A (Botox®) diluted in 2.2 ml of NaCl 0.9 % Receives: Drug: Botox injection |
| Placebo | Placebo comparator | Placebo injection : NaCl 0.9 % Receives: Drug: Placebo injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 1 month after the injection
Secondary outcomes (8)
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.
Measured: 1 month after the injection
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.
Measured: 1 month after the injection
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
Measured: 1 month after the injection
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.
Measured: 1 month after the injection
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.
Measured: 1 month after the injection
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)
Measured: 1 month after the injection
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
Measured: 1 month after the injection
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.
Measured: 6 months after the injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2012 (estimated)
- Study startFeb 2012
- Primary completionMar 2014
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SialorrheaStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christophe VIAL, MD, Principal investigator, Centre SLA, Groupement Hospitalier Est, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01551940
- Sponsor's protocol ID
- 2010.607/11
- Acronym
- ALS-TOX
- Status last verified
- Aug 2015
- First submitted
- 9 Mar 2012
- Last updated
- 14 Aug 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Efficacy of Botulinum Toxin Type A in the Treatment of Sialorrhea in the Patient Affected by Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT01551940
- Acronym / sponsor ID
- ALS-TOX / 2010.607/11
- Conditions
- Sialorrhea, Amyotrophic Lateral Sclerosis
- Keywords
- Sialorrhea, Amyotrophic lateral sclerosis, Botulinum toxin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- Feb 2012
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 13 Mar 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Botox | Experimental | Drug: Botox injectionBotox injection : 100 UI of botulinum toxin type A (Botox®) diluted in 2.2 ml of NaCl 0.9 % |
| Placebo | Placebo comparator | Drug: Placebo injectionPlacebo injection : NaCl 0.9 % |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Botox injectionLocal ultrasound-guided bilateral injection of botulinum toxin type A in parotid (35 UI/gland) and submandibular (15 UI/gland) glands. | Drug | Botox |
| Placebo injectionA local ultrasound-guided bilateral injection of placebo (NaCl 0.9 %) in parotid (0.7 ml/gland) and submandibular (0.3 ml/gland) glands. | Drug | Placebo |
Primary outcome
Improvement of the functional embarrassment provoked by sialorrhea
Time frame: 1 month after the injection
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).
8 secondary outcomes
Decrease of the salivary secretion rate and a decrease of the embarrassment for the main caregiver
Time frame: 1 month after the injection
Improvement of the value of the hypersalivation item in ALSFRS-R scale
Time frame: 1 month after the injection
Decrease of the score of severity and frequency of the drooling rating scale
Time frame: 1 month after the injection
Decrease of the cotton roll weight
Time frame: 1 month after the injection
Decrease of the number of paper handkerchiefs used
Time frame: 1 month after the injection
Modification of the speech evaluation
Time frame: 1 month after the injection
Improvement of the quality of life
Time frame: 1 month after the injection
Description of patient cohort after the first injection
Time frame: 6 months after the injection
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christophe VIAL, MD principal investigator
Centre SLA, Groupement Hospitalier Est, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Botox injection in people with Sialorrhea and Amyotrophic Lateral Sclerosis, run by Hospices Civils de Lyon at 13 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement of the functional embarrassment provoked by sialorrhea (time frame: 1 month after the injection). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2015.