Trial Radar
Sign in

NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

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

NCT04845958

CompletedN/A

Sponsor: Sanofi

Conditions: Gaucher Disease, Splenomegaly, Acid SphingoMyelinase Deficiency

Countries: France

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

Eligibility overview

Sex: ALL

Age: 0 Years to 18 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria
Inclusion criteria:

* Patient under the age of 18 years
* Patient with unexplained SMG (SMG defined as a palpable spleen, already known or discovered for the first time) and who has undergone tests to eliminate obvious causes of SMG

Exclusion criteria:

Patient with any obvious cause of SMG as described by clinical examination and/or lab or imaging test available in medical records and/or having been diagnosed with any of the following conditions:

1. hemolytic anemia
2. hematological malignancy
3. portal hypertension
4. infectious disease associated with SMG (Cytomegalovirus, Epstein Barr virus, leishmaniasis or other obvious infectious cause revealed by the medical history)

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Locations (40)
  • Amiens, France
  • Angers, France
  • Argenteuil, France
  • Bayonne, France
  • Besançon, France
  • Bordeaux, France
  • Brest, France
  • Caen, France
  • Chambéry, France
  • Clermont-Ferrand, France
  • Colombes, France
  • Créteil, France
  • Dijon, France
  • Gleizé, France
  • Jossigny, France
  • La Rochelle, France
  • La Tronche, France
  • Limoges, France
  • Lyon, France
  • Mantes-la-Jolie, France
  • Marseille, France
  • Meaux, France
  • Montpellier, France
  • Nantes, France
  • Nice, France
  • Nîmes, France
  • Paris, France
  • Perpignan, France
  • Poissy, France
  • Poitiers, France
  • + 10 more on CT.gov