Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12611001270909
Ethics application status
Approved
Date submitted
13/09/2011
Date registered
12/12/2011
Date last updated
12/12/2011
Type of registration
Retrospectively registered

Titles & IDs
Public title
BOTOX for vocal cord dysfunction in severe asthma
Scientific title
Botulinum Toxin to treat vocal cord dysfunction in severe asthma
Secondary ID [1] 263028 0
Nil
Universal Trial Number (UTN)
Trial acronym
VCD BOTOX
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Severe asthma 270755 0
Vocal cord Dysfunction 278898 0
Condition category
Condition code
Respiratory 270935 270935 0 0
Asthma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Botulinium Toxin 3 units (1.5 units to each thyro-arytenoid muscle = 0.1ml total volume) will be injected into the thyroarytenoid muscle (vocal cord) localised by using Electromyography.
Percutaneous transcricothyroid injection is the method of injection to be used.
Treatment will occur on one occasion only for the purpose of this study.
Intervention code [1] 269372 0
Treatment: Drugs
Comparator / control treatment
Placebo - normal saline = 0.1ml total volume, will be injected into the thyroarytenoid muscle (vocal cord) localised by using Electromyography.
Percutaneous transcricothyroid injection is the method of injection to be used.
Control group
Placebo

Outcomes
Primary outcome [1] 279604 0
Primary outcome measure for first hypothesis is RATIO (vocal cord diameter:tracheal diameter) using 320-slice CT scan of the larynx, adjusted for baseline
Timepoint [1] 279604 0
Measured at 4 weeks
Primary outcome [2] 279605 0
Asthma Control Test (ACT)
Timepoint [2] 279605 0
performed 4 weekly over 6 months, adjusted for baseline
Primary outcome [3] 279606 0
Pre-bronchodilator FEV1
Timepoint [3] 279606 0
Performed 4 weekly over 6 months adjusted for baseline
Secondary outcome [1] 294015 0
Changes in BORG score
Timepoint [1] 294015 0
Assessed weekly for 4 weeks, then monthly to 6 months
Secondary outcome [2] 294016 0
Asthma Control Questionnaire
Timepoint [2] 294016 0
Assessed weekly for 4 weeks, then monthly to 6 months
Secondary outcome [3] 294017 0
Daily Symptom scores and Peak Flow reading
Timepoint [3] 294017 0
Performed daily by the patient for 24 weeks
Secondary outcome [4] 294018 0
Changes in FEV1
Timepoint [4] 294018 0
Assessed weekly for 4 weeks, then monthly to 6 months
Secondary outcome [5] 294021 0
Voice related Quality of Life score (VRQoL)
Timepoint [5] 294021 0
Assessed weekly for 4 weeks, then monthly to 6 months

Eligibility
Key inclusion criteria
Asthma confirmed by airway reversibility (FEV1 >12% and 200mls) OR airway hyperreponsiveness;
Subjects with clinical features of severe refractory asthma;
Subjects with well documented requirement for regular treatment with high dose ICS;
Subjects with well documented requirement for controller medication in addition to high dose ICS;
Evidence of optimal medication use;
Sub-optimally controlled asthma, as indicated by ACT score <15;
Post-bronchodilator FEV1 >40% predicted;
VCD demonstrated by 320-slice CT larynx (RATIO below LLN during either inspiration or expiration;
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Other significant respiratory disorder;
Known vocal cord pathology or diagnosed voice condition other than VCD;
Known brain or brainstem cancer, or head and neck cancer;
Known neurological disorders or neuromuscular disorders;
Pregnancy

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Once patients are successfully screened and eligibility confirmed, they are randomised to receive medication/placebo assigned to the subject from a central computer-based randomisation program.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Randomisation to active (BOTOX) or control (saline) intervention will be performed by an unblinded pharmacist, using a computer-based minimisation algoithm (Woolcock Institute of Medical Research), with stratification according to whether the patient has or has not received speech therapy for VCD and FEV1 <80% predicted.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 4
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/11/2011
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
46
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 269964 0
Self funded/Unfunded
Name [1] 269964 0
Country [1] 269964 0
Australia
Primary sponsor type
Individual
Name
Prof Philip Bardin
Address
Southern Health, Monash Medical Centre, Respiratory and Sleep Medicine
246 Clayton Rd
Clayton
VIC, 3168
Country
Australia
Secondary sponsor category [1] 268862 0
None
Name [1] 268862 0
Address [1] 268862 0
Country [1] 268862 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 271799 0
Southern Health HREC A
Ethics committee address [1] 271799 0
Research Directorate
Monash Medical Centre
246 Clayton Rd
Clayton, VIC, 3168
Ethics committee country [1] 271799 0
Australia
Date submitted for ethics approval [1] 271799 0
Approval date [1] 271799 0
25/05/2011
Ethics approval number [1] 271799 0
11006A

Summary
Brief summary
Asthma is a common condition and severe and difficult-to-treat in up to 5-20% of patients. Vocal cord dysfunction (VCD) is a disorder characterised by inappropriate closure of the vocal cords during respiration. Numerous asthmatics have VCD co-existing with asthma. Recent studies have detected VCD in as many as 50% of cases with Difficult-To-Treat-Asthma. Botulinium Toxin has been shown to effectively reverse the detrimental effects of VCD.
This study looks at two hypotheses:
1. That in patients with VCD and DTTA, botulinium toxin treatment reduces VCD compared with saline control;
2. That in patients with VCD and DTTA, botulinium toxin treatment is associated with improvement in symptom-based, but not physiological, measures of asthma control, compared with saline control.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 33145 0
Address 33145 0
Country 33145 0
Phone 33145 0
Fax 33145 0
Email 33145 0
Contact person for public queries
Name 16392 0
Professor Philip Bardin
Address 16392 0
Monash Medical Centre
Department of Respiratory and Sleep Medicine
246 Clayton Rd
Clayton, VIC, 3168
Country 16392 0
Australia
Phone 16392 0
+61 3 9594 2281
Fax 16392 0
+61 3 9594 7877
Email 16392 0
[email protected]
Contact person for scientific queries
Name 7320 0
Prof Philip Bardin
Address 7320 0
Monash Medical Centre
Department of Respiratory and Sleep Medicine
246 Clayton Rd
Clayton, VIC, 3168
Country 7320 0
Australia
Phone 7320 0
+61 3 9594 2281
Fax 7320 0
Email 7320 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.