What is the mechanism of action of this treatment?

Botox injections and esophageal dilation are common treatments for Achalasia that target the lower esophageal sphincter (LES). Botox injections work by paralyzing the LES muscles, reducing pressure and allowing easier passage of food into the stomach. Esophageal dilation mechanically widens the esophagus by stretching the LES, alleviating the obstruction. These treatments are crucial for Achalasia patients as they address the core issue of the disorder—impaired LES relaxation—thereby improving swallowing and reducing discomfort.

What side effects are associated with this type of intervention?

The most common treatments for Achalasia, such as botox injections to the lower esophageal sphincter (LES) and pneumatic dilation, have known side effects. Botox injections can cause transient chest pain, esophageal perforation, and may require repeat treatments due to their temporary effect. Pneumatic dilation can lead to complications like esophageal perforation, chest pain, and gastroesophageal reflux. Both treatments aim to relieve symptoms but carry potential risks that should be considered.

What prior FDA approvals exist?

The FDA has approved several treatments for achalasia that involve mechanical widening and muscle paralysis. Pneumatic dilation, a procedure that mechanically widens the esophagus, is a well-established treatment. Botulinum toxin (Botox) injections, which paralyze the lower esophageal sphincter to alleviate symptoms, are also FDA-approved and commonly used, especially in patients with significant comorbidities. These treatments aim to improve esophageal motility and reduce symptoms by addressing the functional obstruction at the lower esophageal sphincter.

What other types of research exist?

Promising novel alternatives to esophageal dilation and Botox injection for achalasia patients include per-oral endoscopic myotomy (POEM) and surgical myotomy. These procedures aim to provide long-term relief by addressing the underlying motility disorder more effectively than mechanical widening or muscle paralysis.

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Neurotoxin

Botox and/or Esophageal Dilation for Achalasia

Phase 4

Waitlist Available

Led By Dhyanesh Patel, MD

Research Sponsored by Vanderbilt University Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Adults with known diagnosis of achalasia who are NOT candidates for definitive therapy with pneumatic dilation, surgical myotomy, or POEM

Be older than 18 years old

Must not have

Previous surgery for reflux or peptic ulcer disease

Less than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

Drug Has Already Been Approved

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a combination of esophageal stretching and botox injections for patients with achalasia who have other health issues. The treatment aims to make swallowing easier by both stretching the esophagus and relaxing its muscles. Botox injections have been used for treating achalasia and other esophageal motility disorders, providing temporary relief.

Who is the study for?

This trial is for adults with achalasia, a rare disorder affecting esophagus movement, who can't have standard treatments like dilation or surgery. They must be undergoing routine upper endoscopy for their condition but cannot participate if they're under 18 or have had previous surgeries for reflux or peptic ulcers.

What is being tested?

The study compares two approaches to ease symptoms of achalasia: one group receives Botox injections in the lower esophageal sphincter (LES), while the other gets both Botox and esophageal dilation using a balloon dilator during an endoscopy.

What are the potential side effects?

Possible side effects include sore throat, infection risk at the injection site, difficulty swallowing, heartburn, chest pain, and in rare cases breathing difficulties due to botulinum toxin spreading from the area of injection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have achalasia and cannot undergo surgery or other definitive treatments.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have had surgery for acid reflux or ulcers.

Select...

I am under 18 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

dysphagia score

Awards & Highlights

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Active Control

Group I: Botulinum toxinActive Control3 Interventions

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.

Group II: Botulinum toxin and dilationActive Control4 Interventions

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.

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Botox injections and esophageal dilation are common treatments for Achalasia that target the lower esophageal sphincter (LES). Botox injections work by paralyzing the LES muscles, reducing pressure and allowing easier passage of food into the stomach. Esophageal dilation mechanically widens the esophagus by stretching the LES, alleviating the obstruction. These treatments are crucial for Achalasia patients as they address the core issue of the disorder—impaired LES relaxation—thereby improving swallowing and reducing discomfort.