Botulinum Toxin Injection to Treat Diffuse Esophageal Spasm (Botox)
Recruitment status was: Recruiting
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Prospective Randomised Double-blind Sham Study on the Use of Botox for the Treatment of Diffuse Esophageal Spasm|
- Dysphagia severity score [ Time Frame: One month after treatment ] [ Designated as safety issue: No ]the primary outcome variable is the symptom severity score in patients with diffuse esophageal spasm.
- Manometry pattern [ Time Frame: One month after treatment ] [ Designated as safety issue: No ]Pattern of contractions on stationary esophageal manometry
|Study Start Date:||February 2010|
|Estimated Study Completion Date:||December 2013|
|Estimated Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Placebo Comparator: Placebo
Placebo arm: per-endoscopic injection of saline
Saline 4 ml
Active Comparator: Botulinum toxin
Botulinum toxin (Botox) 100 u in 4 ml, injected per-endoscopically
Drug: Botulinum Toxin Type A
per-endoscopic injection of 100 U of botulinum toxin dissolved in 4 ml of saline
Other Name: Botox
Patients are selected on the basis of a clinical diagnosis of diffuse esophageal spasm, based on endoscopy and manometry.
This is a sham-controlled cross-over study of injection of botulinum toxin or saline, in randomized order, in the distal esophagus.
All patients have undergone esophageal manometry which shows diffuse esophageal spasm, and a 24h pH monitoring/manometry to quantify acid reflux. All drugs prescribed to affect esophageal motility (nitrates, calcium antagonists) are stopped during the study. After randomisation, the patient will undergo standard esophageal manometry to assess baseline esophageal motility. On the same day the patient will be asked to fill out a set of symptom questionnaires including a dysphagia questionnaire. At endoscopy, the active treatment group receives 8 x 12.5 units, each in 0.5 ml, of botulinum toxin A which will be injected in the four quadrants of the esophagus at 2 and 5 cm above the lower esophageal sphincter. In the Sham group 8 * 0.5 ml of physiologic serum will be injected. The randomisation will be performed at the endoscopy unit by a nurse otherwise not involved in the protocol.
After 1 month a stationary manometry and a new esophageal pH/manometry monitoring will be performed, followed by a second endoscopy with injection of saline or botulinum toxin in a cross-over mode. At the time of the second endoscopy each patient will fill out a dysphagia questionnaire. One month later, the dysphagia questionnaire is repeated.
Subsequently, follow-up will occur at the outpatient clinic after 6 and 12 months, with follow-up dysphagia questionnaires but no additional manometries or endoscopies, unless the clinical status deteriorates.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01432782
|University Hospitals Leuven|
|Leuven, Vlaanderen, Belgium, 3000|
|Principal Investigator:||Jan Tack, M.D., Ph.D.||Universitaire Ziekenhuizen Leuven|