Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Why do some kids have an enlarged spleen? new study investigates

NCT ID NCT04845958

Knowledge-focused Sponsor: Sanofi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study examined 60 children under 18 with an unexplained enlarged spleen to see how often rare diseases like Gaucher disease were the cause. Researchers ruled out common reasons first, then tested for specific enzyme deficiencies. The goal was to better understand what hidden conditions might be behind this symptom.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this study could help doctors better understand which rare diseases to test for in children with unexplained enlarged spleen.
What could go wrong
This is a small, completed observational study with only 60 children, so results may not apply to all populations. It does not test any treatment.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

60 people

The number who actually took part.

Started

Jun 2021

Finished

Nov 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patient under 18 year-old 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 including: hemolytic anemia, hematological malignancy, portal hypertension, infectious disease associated with SMG

Ages

0 to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Acid sphingomyelinase deficiency are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

acid sphingomyelinase deficiency Gaucher disease Niemann-Pick disease Splenomegaly

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acid sphingomyelinase deficiency Gaucher disease, splenomegaly

Contacts and locations

Locations

  • Investigational Site Number 2500001

    Lyon, 69008, France

  • Investigational Site Number 2500002

    Montpellier, 34090, France

  • Investigational Site Number 2500003

    Bordeaux, 33000, France

  • Investigational Site Number 2500004

    Strasbourg, 67200, France

  • Investigational Site Number 2500005

    Marseille, 13005, France

  • Investigational Site Number 2500006

    Dijon, 21079, France

  • Investigational Site Number 2500007

    La Tronche, 38700, France

  • Investigational Site Number 2500008

    Colombes, 92700, France

  • Investigational Site Number 2500009

    Clermont-Ferrand, 63000, France

  • Investigational Site Number 2500010

    Gleizé, 69400, France

  • Investigational Site Number 2500011

    Poitiers, 86000, France

  • Investigational Site Number 2500012

    Vandœuvre-lès-Nancy, 54500, France

  • Investigational Site Number 2500013

    Perpignan, 66000, France

  • Investigational Site Number 2500014

    Angers, 49933, France

  • Investigational Site Number 2500015

    Besançon, 25030, France

  • Investigational Site Number 2500016

    Paris, 75012, France

  • Investigational Site Number 2500017

    Saint-Priest-en-Jarez, 42270, France

  • Investigational Site Number 2500018

    Chambéry, 73000, France

  • Investigational Site Number 2500019

    Nice, 06200, France

  • Investigational Site Number 2500020

    Tours, France

  • Investigational Site Number 2500021

    Nantes, 44000, France

  • Investigational Site Number 2500022

    Nîmes, 30029, France

  • Investigational Site Number 2500023

    Rennes, 35203, France

  • Investigational Site Number 2500024

    Rouen, 76031, France

  • Investigational Site Number 2500025

    Valenciennes, 59300, France

  • Investigational Site Number 2500026

    Caen, 14033, France

  • Investigational Site Number 2500027

    Meaux, 77100, France

  • Investigational Site Number 2500028

    Créteil, 94000, France

  • Investigational Site Number 2500029

    Reims, 51100, France

  • Investigational Site Number 2500030

    Limoges, 87042, France

  • Investigational Site Number 2500031

    Jossigny, 77600, France

  • Investigational Site Number 2500032

    Mantes-la-Jolie, 78200, France

  • Investigational Site Number 2500033

    Toulouse, France

  • Investigational Site Number 2500034

    Argenteuil, 95107, France

  • Investigational Site Number 2500035

    Poissy, 78300, France

  • Investigational Site Number 2500036

    Amiens, 80054, France

  • Investigational Site Number 2500037

    Roubaix, 59056, France

  • Investigational Site Number 2500038

    Brest, 29609, France

  • Investigational Site Number 2500039

    Bayonne, 64100, France

  • Investigational Site Number 2500040

    La Rochelle, 17000, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.