Botox and/or Esophageal Dilation for Achalasia

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Vanderbilt University Medical Center

Disqualifiers: Under 18, Previous reflux surgery, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

50 Participants Needed

Trial Summary

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug Botulinum toxin type A for treating achalasia?

Research shows that injecting botulinum toxin into the esophagus can effectively treat achalasia by helping the lower esophageal sphincter (a muscle that controls the opening between the esophagus and stomach) relax, which improves swallowing. Many patients experience improvement for 2-6 months, although repeat treatments may be needed. ^{1 2 3 4 5}

Is Botox safe for treating achalasia?

Botox injections for achalasia are generally considered safe, with few reported adverse reactions, although serious events like a sub-diaphragmatic abscess are rare. It is often used as a safer alternative for patients at high surgical risk. ^{1 2 6 7 8}

How does the drug botulinum toxin differ from other treatments for achalasia?

Botulinum toxin is a less invasive treatment for achalasia that works by blocking nerve signals to reduce muscle pressure in the esophagus, offering a safer option with fewer complications compared to pneumatic dilation, which involves stretching the esophagus and carries a risk of perforation. However, the effects of botulinum toxin are temporary, often requiring repeat treatments every few months. ^{1 2 9 10 11}

What is the purpose of this trial?

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.

Research Team

Dhyanesh Patel, MD

Principal Investigator

Vanderbilt University Medical Center

Eligibility Criteria

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.

Inclusion Criteria

I am having an endoscopy for my achalasia.

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

Exclusion Criteria

Significant medical conditions possibly placing subjects at risk to undergo endoscopy

I have had surgery for acid reflux or ulcers.

I am under 18 years old.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a one-time dose of Botulinum toxin (Botox) injection into the LES, with or without esophageal dilation

1 day

1 visit (in-person)

Follow-up

Participants are monitored for symptomatic response and dysphagia score over time

12 months

Treatment Details

Interventions

• Botulinum toxin type A
• Esophageal Dilation

Trial Overview 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.

Participant Groups

Group I: Botulinum toxin Active Control 3 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 dilation Active Control 4 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.

Botulinum toxin type A is already approved in United States, European Union for the following indications:

Approved in United States as Xeomin for:

• Upper limb spasticity (excluding spasticity caused by cerebral palsy)
• Chronic sialorrhea

Approved in European Union as Xeomin for:

• Upper limb spasticity
• Chronic sialorrhea
• Blepharospasm
• Cervical dystonia

Who Is Running the Clinical Trial?

Vanderbilt University Medical Center

Lead Sponsor

Trials

922

Recruited

939,000+

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Use of botulinum toxin in the treatment of achalasia. [2018]

2. United States pubmed.ncbi.nlm.nih.gov

Controlled trial of botulinum toxin injection versus placebo and pneumatic dilation in achalasia. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Randomized controlled trial of intrasphincteric botulinum toxin A injection versus balloon dilatation in treatment of achalasia cardia. [2019]

4. Chile pubmed.ncbi.nlm.nih.gov

[Intrasphincteric injection of botulinum toxin in patients with esophageal achalasia]. [2010]

5. England pubmed.ncbi.nlm.nih.gov

Botulinum toxin versus pneumatic dilatation in the treatment of achalasia: a randomised trial. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Long-term follow-up of achalasic patients treated with botulinum toxin. [2019]

7. United States pubmed.ncbi.nlm.nih.gov

Prospective study of esophageal botulinum toxin injection in high-risk achalasia patients. [2010]

8. United States pubmed.ncbi.nlm.nih.gov

A Case of Sub-Diaphragmatic Abscess after Injection of Botulinum Toxin to Treat Achalasia. [2020]

9. United States pubmed.ncbi.nlm.nih.gov

Intrasphincteric botulinum toxin versus pneumatic balloon dilation for treatment of primary achalasia. [2019]

10. Italy pubmed.ncbi.nlm.nih.gov

Pneumatic dilation and botulinum toxin: when and why? [2013]

11. United States pubmed.ncbi.nlm.nih.gov

Endoscopic intrasphincteric injection of botulinum toxin for the treatment of achalasia. [2019]