Endoscopic Mucosal Resection for the Diagnosis of a-Ganglionosis, a Controlled Prospective Trial (EDGE Trial) (EDGE)

• ClinicalTrials.gov Identifier: NCT01515501
• Recruitment Status: Completed
• First Posted: January 24, 2012
• Last Update Posted: August 16, 2018
• Sponsor: Massachusetts General Hospital
• Information provided by (Responsible Party): Branden Kuo, Massachusetts General Hospital

Study Description

Brief Summary:

Patients undergoing routine rectal suction biopsy will undergo as part of the study an additional Endoscopic mucosal resection. The biopsy results will also be correlated with patient's clinical data including clinical history, Bristol stool scale, anorectal manometry results, and SITZ marker studies. Cost and recovery time will be compared.

Condition or disease	Intervention/treatment	Phase
Constipation; Hirschsprung Disease	Procedure: Endoscopic mucosal resection (EMR)	Not Applicable

Detailed Description:

Background: Adult and pediatric patients presenting with chronic constipation and/or motility disorders may be referred for rectal biopsy to rule out aganglionosis or Hirschsprung's disease. The traditional diagnostic test, rectal suction biopsy, is a blind technique and is insensitive in confirming the diagnosis. Frequently, patients require subsequent referral for a surgical full thickness biopsy.

Hypothesis: Endoscopic mucosal resection (EMR) will improve the diagnostic yield for aganglionosis and decrease the need for subsequent surgical full thickness biopsy.

Methods: This is a prospective, single center, controlled investigation of EMR for the diagnosis of colonic aganglionosis / Hirschsprung's disease. Patients who are offered rectal suction biopsy will be offered enrollment. Enrolled patients will have one additional procedure (EMR) at the time of their rectal suction biopsy. Specimens will be analyzed pathologically for size, submucosal tissue adequacy, the presence of ganglionic tissue, and positivity by acetylcholinesterase staining. The adequacy of the tissue specimen, the proportion of diagnostic specimens, and the proportion of patients that would have required subsequent referral for full thickness biopsy, will be compared. Variable such as cost and recovery time will be compared. Biopsy results will be compared with patient's pre-endoscopy clinical data including their history of constipation, results of SITZ marker studies, Bristol stool scale, and anorectal manometry.

Results: The primary outcome variable will be the proportion of patients with a diagnostic specimen in each group. Secondary outcome variables will include the size of the specimen, the presence of ganglionic tissue, the presence of submucosal tissue, and the positivity of the acetylcholinesterase stain. The biopsy results will also be correlated with patient's clinical data including clinical history, Bristol stool scale, anorectal manometry results, and SITZ marker studies. Cost and recovery time will be compared.

Discussion: The proposed investigation may demonstrate that a simple endoscopic test, which uses direct visualization, can improve the diagnostic yield of rectal biopsies for Hirschsprung's disease, and spare patients an additional surgical full thickness rectal biopsy.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 12 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Diagnostic
• Official Title: Endoscopic Mucosal Resection for the Diagnosis of a-Ganglionosis, a Controlled Prospective Trial (EDGE Trial)
• Actual Study Start Date: October 2007
• Actual Primary Completion Date: February 2012
• Actual Study Completion Date: August 2018

Arms and Interventions

Arm	Intervention/treatment
Endoscopic mucosal resection; At time of rectal section biopsy all subjects will under go the additional intervention of an endoscopic muscosal resection.	Procedure: Endoscopic mucosal resection (EMR); EMR uses an endoscope to take a tissue sample from the rectum. It is the same type of instrument used in a routine colonoscopy. It is hoped that this procedure will help diagnose Hirschsprung's Disease more often than by recal suction biopsy alone, which can often be unclear and result in more invasive surgery for diagnosis.

Outcome Measures

Primary Outcome Measures :

1. The number of EMR Biopsies that are diagnostic specimens [ Time Frame: One Year ]
   The adequacy of the tissue specimen, the proportion of diagnostic specimens, and the proportion of patients that would have required subsequent referral for full thickness biopsy, will be compared. Variable such as cost and recovery time will be compared. Biopsy results will be compared with patient's pre-endoscopy clinical data including their history of constipation, results of SITZ marker studies, Bristol stool scale, and anorectal manometry.

Eligibility Criteria

• Ages Eligible for Study: 10 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• 10 years of age or older
• offered Rectal Suction biopsy

Exclusion Criteria:

• any contraindication to general anesthesia or conscious sedation
• contraindication to endoscopy
• untreated or unmanageable coagulopathy
• thrombocytopenia (<50)
• inability to provide informed consent.

Contacts and Locations

Locations

United States, Massachusetts

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Sponsors and Collaborators

Massachusetts General Hospital

Investigators

• Principal Investigator: Braden Kuo, M.D.; Massachusetts General Hospital

More Information

Publications:

Croffie JM, Davis MM, Faught PR, Corkins MR, Gupta SK, Pfefferkorn MD, Molleston JP, Fitzgerald JF. At what age is a suction rectal biopsy less likely to provide adequate tissue for identification of ganglion cells? J Pediatr Gastroenterol Nutr. 2007 Feb;44(2):198-202.

Hurlstone DP, Cross SS, Drew K, Adam I, Shorthouse AJ, Brown S, Sanders DS, Lobo AJ. An evaluation of colorectal endoscopic mucosal resection using high-magnification chromoscopic colonoscopy: a prospective study of 1000 colonoscopies. Endoscopy. 2004 Jun;36(6):491-8.

Hirose R, Hirata Y, Yamada T, Kawana T, Taguchi T, Suita S. The simple technique of rectal mucosal biopsy for the diagnosis of Hirschsprung's disease. J Pediatr Surg. 1993 Jul;28(7):942-4.

Pini-Prato A, Martucciello G, Jasonni V. Rectal suction biopsy in the diagnosis of intestinal dysganglionoses: 5-year experience with Solo-RBT in 389 patients. J Pediatr Surg. 2006 Jun;41(6):1043-8.

• Responsible Party: Branden Kuo, Instructor in Medicine, Harvard Medical School, Massachusetts General Hospital
• ClinicalTrials.gov Identifier: NCT01515501
• Other Study ID Numbers: MGH2007p001577
• First Posted: January 24, 2012
• Last Update Posted: August 16, 2018
• Last Verified: August 2018

Keywords provided by Branden Kuo, Massachusetts General Hospital:

Constipation; Hirschsprung Disease

Additional relevant MeSH terms:

Hirschsprung Disease; Constipation; Signs and Symptoms, Digestive; Digestive System Abnormalities; Digestive System Diseases; Megacolon; Colonic Diseases; Intestinal Diseases; Gastrointestinal Diseases; Congenital Abnormalities