NCT04528303

Brief Summary

This study will seek to determine if whole genome sequencing (WGS) improves diagnostic rates, and outcomes for congenital diarrhea and enteropathy (CODE) patients. The investigator will enroll 180 patients in a randomized controlled study to either WGS or whole exome sequencing (WES). This study is designed to evaluate whether CODE patients would benefit from WGS guided precision medicine.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
180

participants targeted

Target at P75+ for not_applicable

Timeline
7mo left

Started May 2024

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress76%
May 2024Dec 2026

First Submitted

Initial submission to the registry

August 24, 2020

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 27, 2020

Completed
3.7 years until next milestone

Study Start

First participant enrolled

May 1, 2024

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2026

Last Updated

March 24, 2026

Status Verified

March 1, 2026

Enrollment Period

2.7 years

First QC Date

August 24, 2020

Last Update Submit

March 20, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • Diagnostic rates between WGS and WES

    Diagnostic rate of genome and exome based on rate of clinically confirmed diagnoses.

    Within approximately 60 days of enrollment

Secondary Outcomes (4)

  • Number of patients receiving precision medicine guided by sequencing results

    Within approximately 60 days of enrollment

  • Mortality of patients

    Within approximately 1 year of enrollment

  • Rate of parental satisfaction with sequencing

    Within one week of patient enrollment

  • Number of parents who are available for trio sequencing

    Within one week of patient enrollment

Study Arms (2)

Whole genome sequencing

EXPERIMENTAL
Genetic: Whole genome sequencing

Whole exome sequencing

ACTIVE COMPARATOR
Genetic: Whole exome sequencing

Interventions

Genomic sequencing and molecular diagnostic results

Whole genome sequencing

Genomic sequencing and molecular diagnostic results

Whole exome sequencing

Eligibility Criteria

AgeUp to 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Patients with chronic diarrhea lasting greater than 2 months
  • Patients with consent from parents or legal guardians
  • Biological relative of a patient enrolled in this study.

You may not qualify if:

  • Chronic diarrhea caused by specific infections, i.e. CMV, Clostridioides difficile
  • Chronic diarrhea with necrotizing enterocolitis, short bowel syndrome
  • Functional diarrhea
  • Patients with previously confirmed monogenic diarrhea
  • Patients with poor compliance

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ying Huang

Shanghai, Shanghai Municipality, 201102, China

RECRUITING

MeSH Terms

Conditions

Diarrhea, InfantileIntestinal Diseases

Interventions

Whole Genome SequencingExome Sequencing

Condition Hierarchy (Ancestors)

DiarrheaSigns and Symptoms, DigestiveSigns and SymptomsPathological Conditions, Signs and SymptomsGastrointestinal DiseasesDigestive System Diseases

Intervention Hierarchy (Ancestors)

Sequence Analysis, DNASequence AnalysisGenetic TechniquesInvestigative Techniques

Study Officials

  • Ying Huang, MD,PhD

    Children's Hospital of Fudan University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Ying Huang, MD, PhD

CONTACT

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, OUTCOMES ASSESSOR
Purpose
DIAGNOSTIC
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 24, 2020

First Posted

August 27, 2020

Study Start

May 1, 2024

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

December 31, 2026

Last Updated

March 24, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations