An observational study in Gaucher Disease and Splenomegaly, sponsored by Sanofi. Completed at 1 site in France. Open to participants aged 15 Years and older. Per ClinicalTrials.gov, last updated 2022-04-25.
Sponsored by Sanofi · 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
Study duration per participant is between 1 and 12 months
171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.
This study's enrollment of 506 is above the median of 60 across 63 observational studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →Sanofi is the lead sponsor of 1,508 studies on the registry; 90 are open to participants now.
Of its 198 completed or terminated interventional studies of FDA-regulated products, 118 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Participants 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
Participants referred for the first time for splenomegaly exploration defined as :
Exclusion criteria:
Participants with obvious diagnostics based on clinical exam, patient's interview and the previous initial routine biological tests :
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
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
Time frame: between 1 and 12 months
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
Time frame: between 1 and 12 months
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
Time frame: between 1 and 12 months
Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
No publications or documents are linked to this record.
This study is completed, as verified in Apr 2022. You cannot join it, but the record below documents what was studied.
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