Clinical trial NCT00125203: Study of Myobloc in the Treatment of Sialorrhea (Drooling) in Patients With Amyotrophic Lateral Sclerosis (ALS)
Official title: A Randomized, Double-Blind, Placebo-Controlled Study of Safety and Efficacy of Botulinum Toxin Type B (Myobloc) in Sialorrhea in Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Sialorrhea
Keywords: ALS
Lou Gehrig's Disease
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sialorrhea
Hypersalivation
Excessive Drooling
Myobloc
botulinum toxin type B
Clostridium botulinum B toxin
botulinum B toxin
botulinum neurotoxin B
NeuroBloc
Lead sponsor: The University of Texas Health Science Center at San Antonio
Sponsor class: OTHER
Collaborators: name: ALS Association
name: University of Kansas
name: Carolinas MDA Neuromuscular/ALS Center-Carolinas Medical System
First posted: 2005-07-29
Enrollment: 20
Sex: ALL
Minimum age (years): 21
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the safety and efficacy of Myobloc in ALS patients who are having excessive drooling.

The primary goal of the study is to determine if the patient perceives a benefit from the Myobloc in controlling excessive drooling.

Detailed description: The secondary goals of this study are to:

* determine the safety of bilateral injections of Myobloc into the parotid and submandibular glands as an effort to control sialorrhea;
* determine by objective measures if the Myobloc injection decreases the saliva produced;
* determine caregiver perceived benefit from Myobloc injection.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of probable or definite ALS based on current World Federation of Neurology criteria
* Between the ages of 21-85, inclusive
* Sialorrhea refractory to treatment with at least two anticholinergic medications OR has been intolerant of anticholinergic medications due to side effects
* Capable of giving informed consent
* Must be able to attend all study visits

Exclusion Criteria:

* Patient has any uncontrolled significant medical, psychiatric or neurological disease (other than ALS) over the past 30 days
* History of ongoing substance abuse
* History of non-compliance with treatment in other experimental protocols
* Cannot provide informed consent or comply with evaluation procedures
* Has received any form of botulinum toxin in the past for any indication
* Women who are pregnant, lactating, or of child bearing potential not using an adequate form of birth control
* Currently being treated with coumadin
* Forced vital capacity (FVC) \<40% of predicted unless the tidal volume is \> 600cc, as patients with significant bulbar weakness may show a falsely low FVC due to bulbar muscle spasticity

Primary outcomes:
measure: Global impression of change by subject at eight weeks post injection
Secondary outcomes:
measure: Patient's subjective assessment of benefit
measure: Change in volume of saliva produced over five minutes (measured with funnel and tube)
measure: ALS Functional Rating Scale (ALSFRS)
measure: Caregiver's subjective assessment of benefit
measure: Change in anticholinergic medication doses and number of times per day suction is used
measure: SEQOL-DW
measure: Duration of benefit
measure: Assessment of treatment assignment (final visit only)
measure: Global assessment of change by investigator
Other outcomes:
Locations:
city: Kansas City
country: United States
facility: University of Kansas Medical Center/Neurology, 1008 Wescoe
state: Kansas
city: Charlotte
country: United States
facility: Carolinas MDA Neuromuscular/ALS Center; Carolinas Medical System; 1540 Garden Terrace
state: North Carolina
Study officials:
affiliation: The University of Texas Health Science Center at San Antonio
name: Carlayne E Jackson, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: The University of Texas Health Science Center at San Antonio
name: Charles B Simpson, MD
role: PRINCIPAL_INVESTIGATOR
References: