NCT03654066

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P25-P50 for phase_4

Timeline
1mo left

Started May 2019

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress99%
May 2019Jun 2026

First Submitted

Initial submission to the registry

August 28, 2018

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 31, 2018

Completed
9 months until next milestone

Study Start

First participant enrolled

May 13, 2019

Completed
7.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2026

Last Updated

May 20, 2025

Status Verified

May 1, 2025

Enrollment Period

7.1 years

First QC Date

August 28, 2018

Last Update Submit

May 14, 2025

Conditions

Achalasia

Outcome Measures

Primary Outcomes (1)

  • 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).

    12 months

Study Arms (2)

Botulinum toxin

ACTIVE COMPARATOR

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.

Drug: Botulinum toxin type AOther: Patient reported outcomesDiagnostic Test: Barium esophagram

Botulinum toxin and dilation

ACTIVE COMPARATOR

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.

Drug: Botulinum toxin type ADevice: Endoscope balloon dilatorOther: Patient reported outcomesDiagnostic Test: Barium esophagram

Interventions

Botulinum toxin type ADRUG

Botox injection in the LES

Also known as: Botox
Botulinum toxinBotulinum toxin and dilation
Endoscope balloon dilatorDEVICE

Distal esophageal dilation

Botulinum toxin and dilation
Patient reported outcomesOTHER

Subjects will complete two patient reported outcome measures (Eckardt and MADS).

Botulinum toxinBotulinum toxin and dilation
Barium esophagramDIAGNOSTIC_TEST

Assess for radiologic severity of achalasia with barium column height measured 1 minute and 5 minutes after upright ingestion of barium

Botulinum toxinBotulinum toxin and dilation

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Vanderbilt University Medical Center Endoscopy Laboratory

Nashville, Tennessee, 37232, United States

Location

MeSH Terms

Conditions

Esophageal Achalasia

Interventions

Botulinum Toxins, Type APatient Reported Outcome Measures

Condition Hierarchy (Ancestors)

Esophageal Motility DisordersDeglutition DisordersEsophageal DiseasesGastrointestinal DiseasesDigestive System Diseases

Intervention Hierarchy (Ancestors)

Botulinum ToxinsMetalloendopeptidasesEndopeptidasesPeptide HydrolasesHydrolasesEnzymesEnzymes and CoenzymesMetalloproteasesBacterial ProteinsProteinsAmino Acids, Peptides, and ProteinsBacterial ToxinsToxins, BiologicalBiological FactorsHealth Care SurveysSurveys and QuestionnairesData CollectionEpidemiologic MethodsInvestigative TechniquesHealth Services ResearchHealth PlanningHealth Care Economics and OrganizationsPatient Outcome AssessmentOutcome Assessment, Health CareOutcome and Process Assessment, Health CareQuality of Health CareHealth Services AdministrationHealth Care Quality, Access, and EvaluationHealth Care Evaluation MechanismsPublic HealthEnvironment and Public Health

Study Officials

  • Dhyanesh Patel, MD

    Vanderbilt University Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

August 28, 2018

First Posted

August 31, 2018

Study Start

May 13, 2019

Primary Completion (Estimated)

June 1, 2026

Study Completion (Estimated)

June 1, 2026

Last Updated

May 20, 2025

Record last verified: 2025-05

Locations

US(1)