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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04430881
Other study ID # DIREGL07298
Secondary ID
Status Completed
Phase
First received
Last updated
Start date September 2015
Est. completion date April 27, 2021

Study information

Verified date April 2022
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

Primary Objective: To estimate the prevalence of Gaucher disease and of other etiologies, in patients of 15 years old or more presenting with unexplained splenomegaly after exclusion of first intention-diagnoses (e.g. portal hypertension, diagnosis or suspicion of haematological malignancy, haemolytic anemia) based on basic physical and biological exams (e.g. full blood count, liver enzymes, reticulocytes) Secondary Objective: To describe the exams and tests conducted for diagnosis purpose and the more frequent associations


Description:

Study duration per participant is between 1 and 12 months


Recruitment information / eligibility

Status Completed
Enrollment 506
Est. completion date April 27, 2021
Est. primary completion date April 27, 2021
Accepts healthy volunteers No
Gender All
Age group 15 Years and older
Eligibility Inclusion criteria: - Participants referred for the first time for splenomegaly exploration defined as : - Either a palpable mass on left upper abdominal quadrant, further confirmed by a = 13 cm craniocaudal length on abdominal Imaging - Or a non palpable splenomegaly discovered on abdominal imaging and with a craniocaudal length = 13 cm - Participants with splenomegaly (as defined above) of unknown origin Exclusion criteria: - Participants with obvious diagnostics based on clinical exam, patient's interview and the previous initial routine biological tests : - Diagnosis of portal hypertension - Diagnosis of hemolytic anemia - Diagnosis of hematological malignancy - Known diagnosis of Gaucher Disease The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
France Investigational site France France

Sponsors (1)

Lead Sponsor Collaborator
Sanofi

Country where clinical trial is conducted

France, 

Outcome

Type Measure Description Time frame Safety issue
Primary Percentage of patients diagnosed with Gaucher disease in the included population of patients with unexplained splenomegaly The diagnosis of Gaucher Disease is based on a value of beta-glucosidase enzyme activity between 1 and 12 months
Secondary Percentage of patients with other than Gaucher disease-etiologies in the included population These will be any etiology among all the diseases that can be considered in the differential diagnosis of unexplained splenomegaly, e.g.: infection, haematological, congestive, inflammatory, neoplastic, infiltrative, benign tumors, immune, iron deficiencies and other miscellaneous rare causes between 1 and 12 months
Secondary Number of participants by type of exams and tests conducted for diagnosis purpose Relevant exams and tests performed, in each site, for the participant diagnosis other than Gaucher disease will be reported , this may include dried blood spot, medullary biopsy, imaging exploration between 1 and 12 months
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