Clinical trial NCT02314741: Peroral Endoscopic Myotomy (POEM) for Esophageal Motility
Official title: Peroral Endoscopic Myotomy (POEM) for Esophageal Motility in Patients With a Clinical Diagnosis of Achalasia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Achalasia
Keywords: poem
per oral endoscopic myotomy
achalasia
Lead sponsor: University of Pennsylvania
Sponsor class: OTHER
First posted: 2014-12-11
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The investigators wish to monitor the adoption of a new, incisionless approach to performing a Heller myotomy for the surgical treatment of achalasia. The method, the Peroral Endoscopic Myotomy (POEM), will provide less-invasive treatment for esophageal achalasia, ideally providing similar if not better outcomes (safety and efficacy) as the Heller myotomy. The investigators hope to enroll 10 patients with a clinical diagnosis of achalasia who meet inclusion criteria.

The POEM procedure has been done in many hospitals without any research associated with it. Dr. Ginsberg, Dr. Chandrasekhara and Dr. Kochman will perform the procedures after being trained. Dr. Ginsberg has personally witnessed the performance of 10 POEM procedures and has performed in a swine model. The PI is credentialed to initiate POEM at HUP with the first case to be proctored by an experienced operator. The PI will then proctor the other adopters. The investigators would like to evaluate the safety of it and the effectiveness of it. The investigators will use their symptom scores and radiology tests pre- and post-POEM to evaluate effectiveness.

Detailed description: An esophageal motility disorder is when muscular contractions become discoordinated or weak and interfere with movement of food down the esophagus. Some esophageal motility disorders persist long enough to cause severe problems requiring surgical intervention. Achalasia, one subtype of esophageal motility disorder that is a rare disease, can be defined by the esophageal sphincter and muscle unable to relax or dilate. Left untreated, symptoms such as difficulty swallowing, regurgitation, heartburn, and chest pain may easily turn into complications such as severe weight loss, malnutrition, coughing, pulmonary infection, pneumonia, and perforation of the esophagus. Diagnosis of esophageal achalasia can be determined by esophageal manometry and/or barium swallow esophagram. Treatment for achalasia includes balloon dilation, botulinum toxin injection and surgical intervention. Balloon dilation is performed by inserting a balloon through the esophageal sphincter, inflating the balloon, disrupting the esophageal muscle. Botox has also been known to successfully relax spastic muscle contractions of achalasia through direct injection into the esophageal muscle. Unfortunately, each alternative to surgical treatment often requires repeated administration to improve the symptoms of achalasia. Traditional treatment of achalasia has included open abdominal or thoracic surgical procedures to cut valve muscles between the esophagus and stomach (Heller Myotomy). Laparoscopic multi-port Heller myotomies have become the preferred approach, requiring 4 smaller abdominal incisions for placement of laparoscope equipment. Results of this laparoscopic technique have proven that although 2/3 of the patient population was successfully treated, a subset of this group still need repeat surgical procedures or balloon dilation. Recently, single-incision laparoscopic Heller myotomies have produced favorable results, with a single umbilical incision preferential to multiple-incision laparoscopy. We propose adoption of a new, incisionless approach to performing a Heller myotomy for the surgical treatment of achalasia. The method, the Peroral Endoscopic Myotomy (POEM) is expected to provide a less-invasive treatment for esophageal achalasia, ideally providing similar if not better outcomes as the Heller myotomy and can be adopted safely and effectively at Penn Medicine.

Eligibility criteria: Inclusion Criteria:

* Age \> or = to 18 years of age
* Clinical diagnosis of achalasia
* A candidate for Heller myotomy
* Esophageal manometry and barium esophogram with findings supportive of achalasia diagnosis
* Women of childbearing potential: negative urine pregnancy test
* Able to undergo general anesthesia
* Willing and able to give informed consent

Exclusion Criteria:

* \< 18 years of age
* Pregnancy
* Previous mediastinal or esophageal surgery
* Contraindications for esophagogastroduodenoscopy
* Presence of malignancy
* Coagulopathy (INR 1.5)
* Thrombocytopenia (platelet count \< 100K/microliter)
* ASA Score \> Class II
* History of mental illness
* Any medical conditions as determined by the PI to be a contraindication to the procedure
* Unable to give informed consent

Primary outcomes:
measure: Gather Standardized Data on Patients with Achalasia Who Have Been Treated with the POEM Procedure
timeFrame: About 12 months
Secondary outcomes:
measure: Pre-Procedure versus Post-Procedure Barium Swallow Results in POEM Patients
timeFrame: About 6 Months
measure: Pre-Procedure Versus Post-Procedure Eckart Score Results in POEM Patients
timeFrame: About 6 Months
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
Study officials:
affiliation: University of Pennsylvania
name: Vinay Chandrasekhara, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Pennsylvania
name: Gregory G Ginsberg, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Pennsylvania
name: Gary W Falk, MD, MS
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Pennsylvania
name: Michael Kochman, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Pennsylvania
name: David C Metz, MD
role: PRINCIPAL_INVESTIGATOR
References: