Gaucher Disease Clinical Trial
Official title:
The SMS Study, a National Study on Prevalence of Unexplained Splenomegaly Etiologies
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 ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Withdrawn |
NCT04189601 -
Complement Activation in the Lysosomal Storage Disorders
|
||
Completed |
NCT02536937 -
A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
|
Phase 1 | |
Completed |
NCT02536911 -
A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
|
Phase 1 | |
Completed |
NCT01411228 -
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
|
Phase 3 | |
Terminated |
NCT04094181 -
A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
|
||
Completed |
NCT00391625 -
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
|
Phase 1/Phase 2 | |
Completed |
NCT03625882 -
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
|
||
Active, not recruiting |
NCT05526664 -
Omics Gaucher Study: Multiomic Approach
|
||
Completed |
NCT02536755 -
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
|
Phase 3 | |
Recruiting |
NCT01344096 -
Thrombocytopathy in Gaucher Disease Patients
|
N/A | |
Completed |
NCT01881633 -
A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers
|
Phase 1 | |
Recruiting |
NCT06116071 -
Biomarkers Related to Bone in Pediatric Gaucher Disease
|
||
Recruiting |
NCT01951989 -
Intra-monocyte Imiglucerase Kinetics in Gaucher Disease
|
Phase 2 | |
Completed |
NCT00258778 -
Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
|
Phase 1 | |
Recruiting |
NCT04388969 -
World Data on Ambroxol for Patients With GD and GBA Related PD
|
||
Recruiting |
NCT05992532 -
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
|
||
Terminated |
NCT04145037 -
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
|
Phase 1/Phase 2 | |
Completed |
NCT00302146 -
Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
|
||
Active, not recruiting |
NCT02605603 -
SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
|
||
Completed |
NCT02053896 -
A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease
|
Phase 2 |