An Early Phase 1 interventional study of Mirabegron 50 MG and Placebo in Achalasia, sponsored by Thomas Jefferson University. Terminated at 1 site in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2021-03-24.
Sponsored by Thomas Jefferson University · Early Phase 1, Interventional, and Treatment
This study evaluates whether a medication called mirabegron is better than placebo (sugar pill) in helping patients with achalasia swallow better. Each patient will receive either mirabegron or the placebo for 4 weeks followed by the opposite medication. Each patient will complete several surveys and undergo several tests to determine if the mirabegron is helping reduce the pressures in the esophagus (swallowing tube).
Achalasia is characterized by incomplete or absent relaxation of the lower esophageal sphincter (LES) and loss of esophageal peristalsis which leads to dysphagia. Standard of care for achalasia includes endoscopic management (dilation and injection of injection of botulinum toxin) and surgery, however both of these options carry procedural risks, may lose efficacy over time and many patients are not appropriate candidates for these treatment options. Unfortunately, there are limited oral medications for patients with achalasia. Mirabegron is an oral beta-3 agonist currently FDA approved for overactive bladder that works by relaxing the bladder muscles. Beta-3 receptors have also been identified in the LES with stimulation leading to LES relaxation in preclinical studies. Through a proof of concept pilot study, the investigators aim to evaluate the effect of mirabegron in patients with achalasia via high resolution manometry and a validated dysphagia scale.
Exclusion Criteria:
Patients with prolonged QTc interval or risk factors to develop it:
Patients will receive 50 mg of oral Mirabegron daily for 4 weeks and then switch to placebo by mouth daily for an additional 4 weeks.
Drug: Mirabegron 50 MG · Drug: Placebo
Patients will receive placebo by mouth daily for 4 weeks and then switch to oral Mirabegron 50 mg daily for an additional 4 weeks.
Drug: Mirabegron 50 MG · Drug: Placebo
Myrbetriq (Mirabegron) tablet
Also known as: Myrbetriq
Sugar pill manufactured to mimic Mirabegron
Also known as: Placebo (for Mirabegron)
Changes in lower esophageal sphincter pressures
Evaluation of changes in lower esophageal sphincter pressures using high resolution manometry
Time frame: Change in lower esophageal sphincter pressure from baseline after 4 weeks of placebo or Mirabegron
Eckardt Achalasia Score (EAS)
Patients will complete the Eckardt Achalasia score which is a simple written scale evaluating dysphagia, regurgitation, retrosternal pain and weight loss. Patients report their symptoms from a 0 to 3. Weight loss (0-none, 1: \< 5 kg, 2: 5-10 kg, 3: \> 10 kg), Dysphagia (0-none, 1-occasional, 2-daily, 3-each meal), Retrosternal pain (0-none, 1-occasional, 2-daily, 3-each meal), Regurgitation (0-none, 1-occasional, 2-daily, 3-each meal). The value for each of the 4 categories is added together to give the EAS. This EAS will be reported for each time point below. A higher score is consistent with worse achalasia and worse outcomes. A lower score is consistent with less severe achalasia and better outcomes. The total range is 0 (no symptoms) to 12 (severe symptoms). There are no subscales.
Time frame: Patients will complete the EAS on study day -14, 0, 7, 28, 42, 49, 70, 84. We will evaluate changes in patient's EAS between day 0 and all of these time points.
Plan to share: No
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This study is terminated, as verified in Mar 2021. You cannot join it, but the record below documents what was studied.
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Thomas Jefferson University