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Botox or Botox With Esophageal Dilation in Patients With Achalasia

Primary Purpose

Achalasia

Status
Enrolling by invitation
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Botulinum toxin type A
Endoscope balloon dilator
Patient reported outcomes
Barium esophagram
Sponsored by
Vanderbilt University Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Achalasia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Adults with known diagnosis of achalasia who are NOT candidates for definitive therapy with pneumatic dilation, surgical myotomy, or POEM
  • Patient undergoing routine care upper endoscopy for achalasia

Exclusion Criteria:

  • Less than 18 years old
  • Previous surgery for reflux or peptic ulcer disease
  • Significant medical conditions possibly placing subjects at risk to undergo endoscopy

Sites / Locations

  • Vanderbilt University Medical Center Endoscopy Laboratory

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

Botulinum toxin

Botulinum toxin and dilation

Arm Description

A one time dose of Botulinum toxin (Botox) is injected into the muscle of the LES leading to blockage of acetylcholine release from nerve endings resulting in increased relaxation.

A one time dose of Botulinum toxin (Botox) is injected into the muscle of the LES leading to blockage of acetylcholine release from nerve endings resulting in increased relaxation. Subjects will also undergo distal esophageal dilation using a 20mm through the scope balloon positioned across the LES.

Outcomes

Primary Outcome Measures

dysphagia score
Dysphagia score as measured by patient reported outcomes will be used to compare the two groups. The scale runs from 0 (no symptoms) to 100 (severe symptoms).

Secondary Outcome Measures

Full Information

First Posted
August 28, 2018
Last Updated
May 10, 2023
Sponsor
Vanderbilt University Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT03654066
Brief Title
Botox or Botox With Esophageal Dilation in Patients With Achalasia
Official Title
Prospective Single-Blinded Randomized Controlled Trial Comparing Botox or Botox With Esophageal Dilation in Patients With Achalasia
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 13, 2019 (Actual)
Primary Completion Date
June 2024 (Anticipated)
Study Completion Date
June 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Vanderbilt University Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
Yes

5. Study Description

Brief Summary
Achalasia is a rare esophageal motility disorder. Treatment of achalasia is aimed toward palliation of symptoms. These include botox injections to the lower esophageal sphincter (LES), pneumatic dilation, surgical myotomy, and per-oral endoscopic myotomy (POEM). Botox injections are frequently used for patients that have significant comorbidities. The primary aim of this study is to assess symptomatic response of patient with achalasia to esophageal dilation and botox injection to the LES compared to standard therapy of only botox injection.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Achalasia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
Participant
Allocation
Randomized
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Botulinum toxin
Arm Type
Active Comparator
Arm Description
A one time dose of Botulinum toxin (Botox) is injected into the muscle of the LES leading to blockage of acetylcholine release from nerve endings resulting in increased relaxation.
Arm Title
Botulinum toxin and dilation
Arm Type
Active Comparator
Arm Description
A one time dose of Botulinum toxin (Botox) is injected into the muscle of the LES leading to blockage of acetylcholine release from nerve endings resulting in increased relaxation. Subjects will also undergo distal esophageal dilation using a 20mm through the scope balloon positioned across the LES.
Intervention Type
Drug
Intervention Name(s)
Botulinum toxin type A
Other Intervention Name(s)
Botox
Intervention Description
Botox injection in the LES
Intervention Type
Device
Intervention Name(s)
Endoscope balloon dilator
Intervention Description
Distal esophageal dilation
Intervention Type
Other
Intervention Name(s)
Patient reported outcomes
Intervention Description
Subjects will complete two patient reported outcome measures (Eckardt and MADS).
Intervention Type
Diagnostic Test
Intervention Name(s)
Barium esophagram
Intervention Description
Assess for radiologic severity of achalasia with barium column height measured 1 minute and 5 minutes after upright ingestion of barium
Primary Outcome Measure Information:
Title
dysphagia score
Description
Dysphagia score as measured by patient reported outcomes will be used to compare the two groups. The scale runs from 0 (no symptoms) to 100 (severe symptoms).
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Adults with known diagnosis of achalasia who are NOT candidates for definitive therapy with pneumatic dilation, surgical myotomy, or POEM Patient undergoing routine care upper endoscopy for achalasia Exclusion Criteria: Less than 18 years old Previous surgery for reflux or peptic ulcer disease Significant medical conditions possibly placing subjects at risk to undergo endoscopy
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dhyanesh Patel, MD
Organizational Affiliation
Vanderbilt University Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Vanderbilt University Medical Center Endoscopy Laboratory
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Botox or Botox With Esophageal Dilation in Patients With Achalasia

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