A National Study in Patients With Unexplained Splenomegaly

Gaucher Disease Clinical Trial

— SMS  

Official title:

The SMS Study, a National Study on Prevalence of Unexplained Splenomegaly Etiologies

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04430881
• Other study ID #: DIREGL07298
• Status: Completed
• 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
• 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.

Related Conditions & MeSH terms

• Gaucher Disease
• Splenomegaly

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