Full Thickness vs. Rectal Suction Biopsy in the Diagnosis of Hirschsprungs Disease (BIOPSY)

May 16, 2022 updated by: University of Southern Denmark

Prospective Randomised Blinded Study Comparing Full Thickness Biopsy and Rectal Suction Biopsies in the Diagnosis of Mb: Hirschsprung

The objective of this prospective study is to compare the diagnostic accuracy of rectal suction biopsy (RSB) compared to full thickness rectal biopsy (FTB) in the diagnosis of Hirschsprungs Disease (HD) in children. The secondary objective to compare cost-effectiveness of the two procedures

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Diagnostic procedures involving the need of surgically obtaining tissue material, carry the risk of complications and the risk of being inconclusive due to various reasons. Lowering the risk of inconclusive diagnostic tests with highest sensitivity and specificity is of high importance in all diagnostic procedures.

A full thickness rectal biopsy (FTB) includes both the submucosal and the myenteric nerve plexus for histological evaluation for presence of ganglion cells and hypertrophic nerve fibers. FTB is performed in general anesthesia (GA), the biopsy is cut with scissors under direct visualization of the intestine and with suturing of the biopsy defect. The other method is a suction biopsy (RSB) in which the biopsy is more superficial acquiring only the submucosal layers. RSB's are performed with a specially designed rectoscope, and the biopsy is obtained under negative pressure within the scope, without visual guidance. This procedure can be performed without anesthesia in children blow 1 year and does not necessitate suturing of the bowel.

The problem with especially RSB could be that the biopsy is too superficial or too small in size for proper pathological evaluation. As a consequence the child has to undergo a new procedure. There is also the risk of false positive or false negative results, which is a serious clinical issue carrying the risk of performing extensive surgery in an otherwise healthy child.

Inconclusive biopsies differ extensively from series to series. But the two largest studies show a rate of inconclusive biopsies at 10% for RSB and 5% for FTB.(Freidmacher and Bjorn). The biopsy profile is similar with less than 1% severe complications (Above Clavien DindoIII) in both procedures, with bleeding as dominating in RSB and Fever in FTB.

Thus there are different advantages and disadvantages between the two methods of biopsy in children suspicious of HD, and different centers advocate either. A prospective comparison of both methods with the child as its own control is a more precise way to compare the two methods and has never been performed. Thus, this study is expected to give an answer to an important research question that will have potential implication worldwide.

Hypothesis:

Our hypothesis is that FTB has lower rate of inconclusive biopsies compared to RSB in the diagnosis of HD.

Design:

Prospective, biopsy sample randomised study.

Method:

Patients referred for rectal biopsy at Odense University Hospital on the suspicion of HD will undergo both an FTB and RSB performed under GA in the same procedure.

Patients will be randomised to which biopsy will be examined first at the pathology department. If a diagnosis can bed securely made on the first tissue samples the other biopsy materials will be stores for examination by another pathologist 30 days later. If any nerve cell is present the sample is negative for Mb. Hirschsprung. If no nervecells is present the biopsy the patient is positive for mb. Hirschsprung. If the tissue is evaluated as not suitable for diagnosis the sample is inconclusive. If the sample is inconclusive the tissue from the other biopsy method is evaluated immediately to secure a fast diagnosis.

All children between 0-15 years of age undergoing biopsies for diagnosis of the diagnosis of HD in Western Denmark from 1/9 2020 will be offered to participate and parental accept will be acquired.

Cost-effectiveness will be measured upon with data from pathology cost and surgery utensils cost.

These will be compared between the two groups.

Power calculation:

With the assumption of a 5 % inconclusive rate for FTB and 10% for RSB (2,5) a McNemar's test for matched comparison of dichotomous outcome (inconclusive / conclusive) indicates the need for 282 patients to obtain 70% strength and a significance level of 5%. (P-value 0.05).

These calculations are based on the largest numbers of biopsy evaluations in the literature.

In these publications a rate of 5% inconclusive biopsies at FTB and 10% at RSB are found.

Interim analyse will be performed after 25%, 50% and 75%. Above 8% complications on 30-day follow-up will result in the study will be paused and examinations of the complications will be performed.

Study Type

Interventional

Enrollment (Anticipated)

300

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Denmark
    • Fyn
      • Odense, Fyn, Denmark, 5000
        • Recruiting
        • Odense University Hospital
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 15 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patients that are referred for rectal biopsy for suspected HD, age 0-15 years.
  • Written informed consent of the parents or guardian.

Exclusion Criteria:

  • Lack of consent from parents or guardians.
  • Previous surgery on the ano-rectum e.g. crohns disease and/or fistulas

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Diagnostic
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Rectal suction biopsy first
Children randomised for pahtological evaluation of rectal suction biopsy tissue first.
Procedure: Rectal suction biopsy.
Suction biopsy performed
Procedure: Full-thickness rectal biopsy.
Full-thickness biopsy performed
Active Comparator: Full thickness biopsy first
Children randomised for pathological evaluation of full thickness biopsy tissue first.
Procedure: Rectal suction biopsy.
Suction biopsy performed
Procedure: Full-thickness rectal biopsy.
Full-thickness biopsy performed

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of inconclusive biopsies between FTB and RSB.
Time Frame: 30 days
Prospective comparison of RSB and FTB when performed on the same patient. Evaluation of diagnostic accuracy.
30 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Surgical and pathology cost.
Time Frame: 30 days
Cost-effectiveness will be measured from surgical and pathology procedural and utensils costs in euros, between RSB and FTB.
30 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Southern Denmark

Collaborators

Odense University Hospital

Investigators

  • Study Director: Mark mr Ellebaek, Dr, University of Southern Denmark
  • Study Chair: Rasmus mr Nielsen, Dr, Odense University Hospital
  • Study Chair: Gunvor ms Madsen, Dr, Odense University Hospital
  • Principal Investigator: Niels mr Bjorn, Dr, Odense University Hospital
  • Study Chair: Lene ms Christensen, Dr, Odense University Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2022

Primary Completion (Anticipated)

January 1, 2024

Study Completion (Anticipated)

February 1, 2024

Study Registration Dates

First Submitted

February 2, 2022

First Submitted That Met QC Criteria

March 31, 2022

First Posted (Actual)

April 1, 2022

Study Record Updates

Last Update Posted (Actual)

May 17, 2022

Last Update Submitted That Met QC Criteria

May 16, 2022

Last Verified

May 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • S-20190116

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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