Peroral Endoscopic Myotomy Versus Botulinum Toxin Injection in Spastic Esophageal Disorders

Esophageal Achalasia Clinical Trial

Official title:

Peroral Endoscopic Myotomy Versus Botulinum Toxin Injection in the Treatment of Medical Refractory Spastic Esophageal Disorders

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02663206
• Other study ID #: IRB00049663
• Status: Withdrawn
• Phase: N/A
• First received: January 5, 2016
• Last updated: January 10, 2018
• Start date: September 2016
• Est. completion date: July 2020

Study information

• Verified date: January 2018
• Source: Johns Hopkins University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

To compare the efficacy of peroral endoscopic myotomy and Botulinum toxin injection in spastic esophageal disorders.

Description:

Spastic disorders of the esophagus encompass hyperactive conditions of the esophagus due to either abnormal premature contractions or extreme vigor. In the current iteration of the Chicago classification, spastic esophageal disorders include spastic (type III) achalasia, diffuse esophageal spasm (DES), and hypercontractile (jackhammer) esophagus. Management of these spastic esophageal disorders is challenging and not clearly defined. Several medical therapies have been suggested and include acid suppression, nitrates, muscle relaxants, and visceral analgesics. For those who fail to response to medical therapy, the treatment options are limited.

Botulinum toxin (BTX) injection is an effective therapeutic option for spastic esophageal disorders, however many patients experience symptoms relapse with this treatment requiring repeated injections.

Heller myotomy is a surgical option for patients with esophageal spastic disorders. As compared to other types of achalasia, the response rate to surgical myotomy was lower in patient with spastic achalasia. The theoretical reason for this is that the disease involves not only the lower esophageal sphincter (LES) but also the esophageal body. Given data to suggest that surgical myotomy may be effective in treating patients with spastic esophageal disorders, peroral endoscopic myotomy (POEM), which is a less invasive treatment modality, has recently been studied for these difficult-to-treat patients. An initial study reported high success rate of POEM for severe spastic esophageal disorders. The response rate as defined by Eckardt score to ≤ 3 was 96% in spastic achalasia, 100% in DES and 70% in those with Jackhammer esophagus after a median follow-up of 234 days in a largest case series of medically refractory spastic esophageal disorders.

To date, the optimal treatment for patients with severe symptomatic esophageal spastic disorders who fail medical therapy is unclear. Here, investigators aim to compare POEM and BTX injection in a randomized design.

To compare the efficacy of peroral endoscopic myotomy and Botulinum toxin injection in spastic esophageal disorders.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: July 2020
• Est. primary completion date: July 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

Inclusion Criteria:

1. Adult patients age 18 - 80 years old.

2. Spastic disorders of the esophagus include spastic (type III) achalasia, distal esophageal spasm (DES), and hypercontractile (jackhammer) esophagus via high resolution esophageal manometry (HRM) 2.

• DES is characterized by normal esophagogastric junction relaxation (integrated relaxation pressure [IRP] <15 mm Hg) and = 20% premature contractions.

• Spastic achalasia is defined as impaired EGJ relaxation (IRP =15 mm Hg) associated with = 20% premature contractions.

• The diagnosis of jackhammer esophagus is defined as at least 1 swallow with a distal contractile integral (DCI) greater than 8000 mm Hg- s- cm.

3. At least 6 months of symptoms (chest pain, dysphagia, regurgitation and/or weight loss) with no adequate response or intolerance to medical therapy including nitrates and/or calcium channel blockers.

4. Overall symptoms score (Eckardt score) > 3

5. Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria:

1. Diagnosis of spastic esophageal disorder was not confirmed by HRM testing.

2. Previous surgery of the esophagus or stomach

3. Previous BTX injection at the esophagogastric junction (EGJ) or LES.

4. Active severe esophagitis

5. Large lower esophageal diverticula

6. Large > 3cm hiatal hernia

7. Megaesophagus (> 6 cm)

8. Sigmoid esophagus

9. Known gastroesophageal malignancy

10. Inability to tolerate sedated upper endoscopy due to cardiopulmonary instability, severe pulmonary disease or other contraindication to endoscopy

11. Cirrhosis with portal hypertension, varices, and/or ascites

12. Uncorrectable coagulopathy defined by prothrombin time < 50% of control; partial thromboplastin time (PTT) > 50 sec, or international normalized ratio (INR) > 1.5), on chronic anticoagulation, or platelet count <75,000.

13. Pregnant or breastfeeding women (all women of child-bearing age will undergo urine pregnancy testing)

Related Conditions & MeSH terms

• Disease
• Esophageal Achalasia
• Esophageal Diseases
• Esophageal Motility Disorders
• Esophageal Spasm, Diffuse
• Muscle Spasticity

Intervention

Procedure:
• peroral endoscopic myotomy
peroral endoscopic myotomy
• Botulinum toxin injection
endoscopic Botulinum toxin (BTX) injection at lower esophagus

Locations

Country	Name	City	State
United States	Johns Hopkins Hospital	Baltimore	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Eckardt score	symptoms scores	3-month
Secondary	Eckardt score		1-year
Secondary	changes in individual symptom scores	Each symptoms scores that are used to calculated Eckardt score	3 months and 12 months
Secondary	changes in esophageal manometry		3 months
Secondary	rate of complications		3 months and 12 months
Secondary	quality of life scores		3 months and 12 months