Botulinum Toxin Injection to Treat Diffuse Esophageal Spasm (Botox)

Prospective Randomised Double-blind Sham Study on the Use of Botox for the Treatment of Diffuse Esophageal Spasm

This study evaluates the effect of botulinum toxin 100 U or saline, injected endoscopically in the distal esophagus, on symptoms and manometry pattern in patients with diffuse esophageal spasm.

Study Overview

• Status: Unknown
• Conditions: Esophageal Spasm, Diffuse
• Intervention / Treatment: Drug: Placebo; Drug: Botulinum Toxin Type A

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Belgium
  • Vlaanderen
    • Leuven, Vlaanderen, Belgium, 3000
      • Recruiting
      • University Hospitals Leuven
      • Contact: Jan Tack, M.D.; Phone Number: +3216344225; Email: jan.tack@med.kuleuven.be

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Spastic motor disorder in the upper GI tract

Exclusion Criteria:

-

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Placebo arm: per-endoscopic injection of saline	Drug: Placebo; 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 Names: Botox

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dysphagia severity score	the primary outcome variable is the symptom severity score in patients with diffuse esophageal spasm.	One month after treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Manometry pattern	Pattern of contractions on stationary esophageal manometry	One month after treatment

Collaborators and Investigators

• Sponsor: Universitaire Ziekenhuizen KU Leuven

Study record dates

Study Major Dates

• Study Start: February 1, 2010
• Primary Completion (Anticipated): December 1, 2013
• Study Completion (Anticipated): December 1, 2013

Study Registration Dates

• First Submitted: September 9, 2011
• First Submitted That Met QC Criteria: September 9, 2011
• First Posted (Estimate): September 13, 2011

Study Record Updates

• Last Update Posted (Estimate): September 13, 2011
• Last Update Submitted That Met QC Criteria: September 9, 2011
• Last Verified: September 1, 2011

More Information

Terms related to this study

• Keywords: Dysphagia; Weight loss; Chest Pain; Esophageal hypermotility
• Additional Relevant MeSH Terms: Digestive System Diseases; Nervous System Diseases; Neurologic Manifestations; Gastrointestinal Diseases; Neuromuscular Manifestations; Deglutition Disorders; Esophageal Diseases; Spasm; Esophageal Motility Disorders; Esophageal Spasm, Diffuse; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Cholinergic Agents; Membrane Transport Modulators; Acetylcholine Release Inhibitors; Neuromuscular Agents; Botulinum Toxins; Botulinum Toxins, Type A; abobotulinumtoxinA
• Other Study ID Numbers: BotoxDES