Trials Sponsors Conditions Drugs Pricing

Conditions

Acid SphingoMyelinase Deficiency Gaucher Disease, Splenomegaly

Outcome Measures

Primary Outcomes (1)

Percentage of patients diagnosed with GD among enrolled patients
Diagnosis of GD based on deficient β-glucocerebrosidase activity in peripheral blood leukocytes or other nucleated cells, or genetic analysis.
Up to 3 months after inclusion

Secondary Outcomes (3)

Rate of each identified disease category at the end of the study among enrolled patients
Up to 3 months after inclusion
Rate of patients with no final diagnosis at the end of the study among enrolled patients
Up to 3 months after inclusion
Number of patients based on specific char. (clinical, lab, genetics)
Up to 3 months after inclusion

Study Arms (1)

Pediatric Patients with Unexplained Enlarged Spleen

Eligibility Criteria

Age0 Years - 18 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

Sampling MethodProbability Sample

Study Population

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

You may qualify if:

Patient under the age of 18 years

Sponsors & Collaborators

Sanofi
Industrylead

Study Sites (40)

Investigational Site Number 2500036

Amiens, 80054, France

Investigational Site Number 2500014

Angers, 49933, France

Investigational Site Number 2500034

Argenteuil, 95107, France

Investigational Site Number 2500039

Bayonne, 64100, France

Investigational Site Number 2500015

Besançon, 25030, France

Investigational Site Number 2500003

Bordeaux, 33000, France

Investigational Site Number 2500038

Brest, 29609, France

MeSH Terms

Conditions

Gaucher DiseaseSplenomegalyNiemann-Pick Diseases

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidoses

Study Officials

Clinical Sciences & Operations
Sanofi
STUDY DIRECTOR

Enrollment

60 participants (actual enrollment reported by the registry).

Study Design

Study Type: observational
Observational Model: COHORT
Time Perspective: CROSS SECTIONAL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

April 12, 2021

First Posted

April 15, 2021

Study Start

June 30, 2021

Actual (reported)

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (3)

Study Arms (1)

Pediatric Patients with Unexplained Enlarged Spleen

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (40)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (3)

Study Arms (1)

Pediatric Patients with Unexplained Enlarged Spleen

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (40)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations