Botox and/or Esophageal Dilation for Achalasia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores different treatments for achalasia, a rare condition affecting how the esophagus moves food toward the stomach. Researchers aim to determine if combining Botox injections with esophageal dilation relieves symptoms more effectively than Botox alone. Botox relaxes the muscle at the bottom of the esophagus, while dilation gently stretches this area to improve swallowing. This trial may suit adults with achalasia who are not candidates for other treatments like surgical procedures. Participants will undergo routine endoscopy as part of their care. As a Phase 4 trial, the treatment is already FDA-approved and proven effective, helping researchers understand its benefits for more patients.
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 is the safety track record for these treatments?
Studies have shown that botulinum toxin type A, commonly known as Botox, is generally safe for treating achalasia, a rare swallowing disorder. Botox relaxes the muscles at the lower end of the esophagus. Research indicates that while some patients find relief, others may not experience long-term benefits. The treatment is usually well-tolerated, with most side effects being mild, such as temporary pain at the injection site.
When combined with esophageal dilation (a procedure that stretches the esophagus), Botox is also considered safe. This combination aims to further relax the muscles and improve swallowing. Research on this combined treatment shows it is generally well-received, with few reports of serious side effects. However, as with any medical procedure, individual experiences can vary.12345Why are researchers enthusiastic about this study treatment?
Unlike the standard treatments for achalasia, which often include pneumatic dilation or surgery, the investigational use of Botulinum toxin (Botox) offers a unique approach by blocking acetylcholine release to relax the lower esophageal sphincter (LES). Researchers are excited about Botox because it provides a less invasive alternative with the potential for quicker relief without the need for multiple sessions. Additionally, combining Botox with esophageal dilation could enhance the effectiveness, offering a one-two punch to improve swallowing and reduce symptoms more efficiently than traditional methods alone.
What evidence suggests that this trial's treatments could be effective for achalasia?
Studies have shown that Botox, derived from botulinum toxin type A, can treat achalasia by relaxing the lower esophageal sphincter (LES). About two-thirds of patients experience improvement, with benefits lasting around 1.3 years. However, it proves less effective for type 1 achalasia, with lower success rates. In this trial, one group of participants will receive Botox alone, while another group will receive a combination of Botox and esophageal dilation. Combining Botox with esophageal dilation, which stretches the esophagus, may lead to better results. Research suggests that using a balloon to dilate the esophagus could provide long-term relief, possibly more effectively than Botox alone. This combination aims to make swallowing easier for people with achalasia.26789
Who Is on the Research Team?
Dhyanesh Patel, MD
Principal Investigator
Vanderbilt University Medical Center
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a one-time dose of Botulinum toxin (Botox) injection into the LES, with or without esophageal dilation
Follow-up
Participants are monitored for symptomatic response and dysphagia score over time
What Are the Treatments Tested in This Trial?
Interventions
- Botulinum toxin type A
- Esophageal Dilation
Botulinum toxin type A is already approved in United States, European Union for the following indications:
- Upper limb spasticity (excluding spasticity caused by cerebral palsy)
- Chronic sialorrhea
- Upper limb spasticity
- Chronic sialorrhea
- Blepharospasm
- Cervical dystonia
Find a Clinic Near You
Who Is Running the Clinical Trial?
Vanderbilt University Medical Center
Lead Sponsor