NCT03411252

Mirabegron in Achalasia: A Clinical and Manometric Proof of Concept Pilot Study

• Status: Terminated
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Thomas Jefferson University · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

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.

Conditions

• Achalasia

Interventions

Type	Name	Description
DRUG	Mirabegron 50 MG	Myrbetriq (Mirabegron) tablet
DRUG	Placebo	Sugar pill manufactured to mimic Mirabegron

Timeline

• Start date: 2018-02-15
• Primary completion: 2019-12-15
• Completion: 2019-12-15
• First posted: 2018-01-26
• Last updated: 2021-03-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study