Gaucher Disease Clinical Trial
— SMSOfficial title:
The SMS Study, a National Study on Prevalence of Unexplained Splenomegaly Etiologies
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 |
Verified date | April 2022 |
Source | Sanofi |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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
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. |
Country | Name | City | State |
---|---|---|---|
France | Investigational site France | France |
Lead Sponsor | Collaborator |
---|---|
Sanofi |
France,
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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