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A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

NCT04845958 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 60

Last updated 2025-09-23

No results posted yet for this study

Summary

Primary Objective:

To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses (e.g. portal hypertension, haematological malignancy, hemolytic anemia, infection) based on clinical examination and routine biological tests (full blood count, reticulocytes, liver tests, abdominal ultrasound, Coombs test and Epstein Barr virus serology).

Secondary Objectives:

* To describe the rate of each identified disease category and the rate of patients with no final diagnosis at the end of the study in pediatric patients with unexplained SMG after exclusion of first intention diagnoses
* To describe the characteristics (clinical, lab, genetics) of all pediatric patients included in the study and to describe the characteristics subdivided by identified disease category and absence of final diagnosis at the end of the study

Conditions

  • Gaucher Disease, Splenomegaly
  • Acid SphingoMyelinase Deficiency

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Eligibility

Min Age
0 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-06-30
Primary Completion
2024-11-25
Completion
2024-11-25

Countries

  • France

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04845958 on ClinicalTrials.gov