Status:
COMPLETED
Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)
Lead Sponsor:
CEN Biotech
Collaborating Sponsors:
BioClinica, Inc.
Takeda
Conditions:
Gaucher Disease, Type 1
MRI
Eligibility:
All Genders
Brief Summary
This study with standardized reading MRIs, will provide for the first time objective and quantified data on organomegaly (liver and spleen volumes) as well as bone alteration (Bone Marrow Burden11) of...
Detailed Description
All data entered will be used for analysis purposes and results will be submitted to inform regulatory agencies. Patient care and management is freely determined by the participating physician, in the...
Eligibility Criteria
Inclusion
- Patients of any age or gender with confirmed diagnosis of type 1 Gaucher disease,
- Patients treated with VPRIV® at the beginning of the study. Prior starting VPRIV® patients could be either treatment naïve or previously treated with any other Gaucher treatment than VPRIV®
- Patients should have one MRI data in the 5 previous years before starting VPRIV® Treatment (up to 3 months after initiation of VPRIV®
- Informed written consent obtained from the patient, and/or patient's parent(s), and/or legal representative. Assent, if old enough to grant, will be obtained from all patients under the age of 18 years
Exclusion
- Patients for whom MRI is contra-indicated
- Patients who did not had an MRI during the five years prior to the initiation of treatment with VPRIV® or within three months after initiation of VPRIV®.
- Patients included in an ongoing clinical trial where the product is blinded
Key Trial Info
Start Date :
January 17 2017
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 27 2018
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT03333447
Start Date
January 17 2017
End Date
December 27 2018
Last Update
June 22 2021
Active Locations (1)
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1
Beaujon Hospital
Clichy, France, 92110