Clinical Trials Logo

Clinical Trial 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 French patients treated with VPRIV®. These data will help to better assess the impact of this treatment on these parameters. The result of this study will also answer in part to the request of the French Transparency Commission (CT: Commission de Transparence) of the French National Health Authority to provide them with data of French patients treated with VPRIV®.


Clinical Trial 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 framework of an observational study reflecting the daily medical practice. The clinical data, the results of routine clinical and laboratory testing that are part of standard medical care for patients with Gaucher disease will be collected from the medical record of the patient : - Socio-demographic characteristics - History of the disease and context of diagnosis - Medical background - MRI: abdominal and bone, as defined below - Clinical data available in the range from 3 months before or after each MRI - Biological data available in the range from 3 months before or after each MRI - Treatment with VPRIV® (velaglucerase alfa), including the doses and regimens. The MRI images collected during the study: The MRIs performed in the patient's usual medical follow-up for its Gaucher disease (abdominal MRI (liver and spleen) and bone MRI (back injury pelvis, lower limbs) and / or whole-body MRI) will be collected. Among the MRIs available in the patient's medical records, MRIs will be collected as following: - Reference MRI: MRI closest to the date of initiation of VPRIV® in the five years preceding the start of treatment or within three months after initiation of VPRIV®. - MRIs of the retrospective phase of the study: All MRIs available between the reference MRI and the inclusion date of the patient. - MRIs of the prospective phase of the study: all the MRIs that will be realized during the prospective phase of the study, that is to say during the year following the date patient inclusion in the study Second reading of the MRI The collected MRIs will be subject to a second reading, which will be conducted centrally by a medical image processing center (BioClinica). This second reading of MRIs will particularly provide quantitative data not available initially in the patient records, such as Bone Marrow Burden (BMB) score at the lumbar spine and femur, and liver and spleen volumes. In case the second reading provides additional data to the first reading, as any new comments or discordant diagnosis, the investigating doctor will integrate these data as soon as he has knowledge of them in the medical care of the patient. MRI collection and transmission Except the data from the second reading of MRIs by BioClinica, all data defined above, will be collected in the case report of the study, from the patient's medical record. For the second centralized reading of the MRIs, the MRIs will be transmitted as follows: the investigation center will make a copy on Compact Disc - Read Only Memory (CD-ROM) of each MRI collected for the study needs, and will replace the identity the patient by an identification number whose correspondence with the patient's identity is only known by the investigation center. This patient identification number will be predefined and attributed to the patient's MRI when the patient is included in the study. Copies of MRIs and coded will be sent securely at BioClinica center for analysis. In case of connection failure from the participating centers, MRI scans will be sent by post. Data from this second reading by BioClinica will be retrieved for analysis. The results of the second reading by BioClinica will be forwarded to investigation center, which will include them in the patient's record. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03333447
Study type Observational
Source CEN Biotech
Contact
Status Completed
Phase
Start date January 17, 2017
Completion date December 27, 2018

See also
  Status Clinical Trial Phase
Recruiting NCT05036629 - Development of MRI Protocols and Associated Neuro-physiological Explorations in Healthy and Pathological Subjects N/A
Completed NCT02548819 - University Hospital Cardiac Device MR Registry: The Safety of MR Imaging in Patients With Implanted Cardiac Devices N/A
Completed NCT01168479 - FLAME: Investigate the Benefit of a Focal Lesion Ablative Microboost in Prostate Cancer Phase 3
Completed NCT00512226 - Iron Overload Assesment in Sickle Cell Anemia and Sickle Cell Thalassemia N/A
Recruiting NCT05439330 - Application of a Dental-dedicated MRI in the Diagnosis of Temporomandibular Joint Disorders, Tissue Alterations Related to Third Molars, Periapical and Periodontal Inflammatory Diseases, and Implant Treatment Planning N/A
Not yet recruiting NCT05464576 - Tumor Staging T of Bladder Tumours: Correlation of MRI and Anatomopathologic Analysis N/A
Recruiting NCT04028375 - Study of CT and MR in the Gastric Cancer
Recruiting NCT04034667 - Study of CT and MR in the Lung Cancer
Recruiting NCT04231175 - Dedicated MR Imaging vs Surgical Staging of Peritoneal Carcinomatosis in Colorectal Cancer N/A
Completed NCT00949507 - Comparison of Two Regimens of Anesthesia for Children Undergoing Magnetic Resonance Imaging (MRI) in General Anesthesia Phase 4
Completed NCT01420211 - Influence of the OATP1B1 and OATP1B3 Genotype on the Hepatic Uptake of Primovist® Phase 1/Phase 2
Recruiting NCT05167669 - Pain Relief in Symptomatic Bone Metastases With Adjuvant Hyperthermia MR Guided HIFU Early Phase 1
Recruiting NCT05618990 - OPTIMIZATION of ADVANCED MR SEQUENCES
Recruiting NCT05107232 - OSV-IRM - Volunteer MRI Sequence Optimization N/A
Completed NCT00556101 - Patient Acceptance of Whole Body Magnetic Resonance Angiography N/A
Recruiting NCT03142698 - Evaluation of 4 MRI Methods (PDFF 3, 6 and 11 Gradient Echoes and Spectroscopy) Compared to the Reference Method (Liver Biopsy) in Quantification of Hepatic Steatosis N/A
Suspended NCT00554073 - Whole Body Magnetic Resonance Angiography in Ischemic Patients at 1.5 and 3T N/A
Completed NCT06366906 - 10-year Retrospective Study of Oral and Maxillofacial Squamous Cell Carcinoma
Not yet recruiting NCT05359497 - Value of MRCP+ And Liver Multiscan in the Management of Dominant Strictures in Primary Sclerosing Cholangitis N/A
Recruiting NCT05192629 - Intranasal Dexmedetomidine Versus Oral Midazolam as Premedication for Propofol Sedation in Pediatric Patients Undergoing Magnetic Resonance Imaging Phase 3