AchalasiaPhase 4NCT03654066
Botox or Botox With Esophageal Dilation in Patients With Achalasia
A Phase 4 study testing Botulinum toxin type A, Endoscope balloon dilator and Patient reported outcomes in people with Achalasia, run by Vanderbilt University Medical Center at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Achalasia
- What is being tested?
- Botulinum toxin type A, Endoscope balloon dilator and Patient reported outcomes
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- 13 May 2019to 1 Aug 2026
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Less than 18 years old
- Previous surgery for reflux or peptic ulcer disease
- Significant medical conditions possibly placing subjects at risk to undergo endoscopy
Trial locations and contacts
1 site in 1 country.
United States1 site
Vanderbilt University Medical Center Endoscopy Laboratory
Nashville, Tennessee, 37232
About this study
Official title: Prospective Single-Blinded Randomized Controlled Trial Comparing Botox or Botox With Esophageal Dilation in Patients With Achalasia
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.
- Conditions
- Achalasia
- Being tested
- Botulinum toxin type A, Endoscope balloon dilator, Patient reported outcomes, Barium esophagram
- Medical subject headings
- Esophageal Achalasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called Botox
Botox injection in the LES
Endoscope balloon dilatorDevice
Distal esophageal dilation
Patient reported outcomesOther
Subjects will complete two patient reported outcome measures (Eckardt and MADS).
Barium esophagramDiagnostic test
Assess for radiologic severity of achalasia with barium column height measured 1 minute and 5 minutes after upright ingestion of barium
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Botulinum toxin type A; Other: Patient reported outcomes; Diagnostic 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. Receives: Drug: Botulinum toxin type A; Device: Endoscope balloon dilator; Other: Patient reported outcomes; Diagnostic Test: Barium esophagram |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2018
- Study start13 May 2019
- Primary completion1 Jun 2026
- Study completion1 Aug 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchalasiaStudies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dhyanesh Patel, Vanderbilt University Medical Center
- Investigators
- Dhyanesh Patel, MD, Principal investigator, Vanderbilt University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03654066
- Sponsor's protocol ID
- 181420
- Status last verified
- Aug 2026
- First submitted
- 28 Aug 2018
- Last updated
- 26 Aug 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Single-Blinded Randomized Controlled Trial Comparing Botox or Botox With Esophageal Dilation in Patients With Achalasia
- ClinicalTrials.gov ID
- NCT03654066
- Acronym / sponsor ID
- 181420
- Conditions
- Achalasia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Study start
- 13 May 2019 (actual)
- Primary completion
- 1 Jun 2026 (actual)
- Study completion
- 1 Aug 2026 (actual)
- First posted
- 31 Aug 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Botulinum toxin | Active comparator | Drug: Botulinum toxin type A, Other: Patient reported outcomes, Diagnostic Test: Barium esophagramA 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. |
| Botulinum toxin and dilation | Active comparator | Drug: Botulinum toxin type A, Device: Endoscope balloon dilator, Other: Patient reported outcomes, Diagnostic Test: Barium esophagramA 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (3 listed)
- Less than 18 years old
- Previous surgery for reflux or peptic ulcer disease
- Significant medical conditions possibly placing subjects at risk to undergo endoscopy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Botulinum toxin type AAlso: BotoxBotox injection in the LES | Drug | Botulinum toxin, Botulinum toxin and dilation |
| Endoscope balloon dilatorDistal esophageal dilation | Device | Botulinum toxin and dilation |
| Patient reported outcomesSubjects will complete two patient reported outcome measures (Eckardt and MADS). | Other | Botulinum toxin, Botulinum toxin and dilation |
| Barium esophagramAssess for radiologic severity of achalasia with barium column height measured 1 minute and 5 minutes after upright ingestion of barium | Diagnostic test | Botulinum toxin, Botulinum toxin and dilation |
Primary outcome
dysphagia score
Time frame: 12 months
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).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
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Achalasia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dhyanesh Patel, MD principal investigator
Vanderbilt University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Botulinum toxin type A, Endoscope balloon dilator and Patient reported outcomes in people with Achalasia, run by Vanderbilt University Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
dysphagia score (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2026.