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)

NCT03333447 | Completed

• 1 other identifier: C1294
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

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 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®.

Conditions

Gaucher Disease, Type 1; MRI

Outcome Measures

Primary Outcomes (4)

• The change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T1 measured at the femur.

  To describe changes in bone disease in femur in Gaucher's disease patients treated with Velaglucerase alfa measured by MRI. The primary endpoint is the change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T1 measured at the femur.

  2 YEARS

• The change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T2 measured at the femur.

  To describe changes in bone disease in femur in Gaucher's disease patients treated with Velaglucerase alfa measured by MRI. The primary endpoint is the change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T2 measured at the femur.

  2 YEARS

• The change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T1 measured at the lumbar spine.

  To describe changes in bone disease in lumbar spine in Gaucher's disease patients treated with Velaglucerase alfa measured by MRI. The primary endpoint is the change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T1 measured at the lumbar spine.

  2 YEARS

• The change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T2 measured at the lumbar spine.

  To describe changes in bone disease in lumbar spine in Gaucher's disease patients treated with Velaglucerase alfa measured by MRI. The primary endpoint is the change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T2 measured at the lumbar spine.

  2 YEARS

Secondary Outcomes (42)

• Describe socio-demographic characteristics of patients : age

  2 YEARS

• Describe socio-demographic characteristics of patients : sex

  2 YEARS

• Describe socio-demographic characteristics of patients : occupation

  2 YEARS

• Clinical evolution of patients :weight (kg)

  2 YEARS

• Clinical evolution of patients : height (cm)

  2 YEARS

Study Arms (1)

Study Population

Patients of any age or gender with confirmed diagnosis of type 1 Gaucher disease, treated with VPRIV® at the beginning of the study. Patients should have one MRI data in the 5 previous years before starting VPRIV® treatment (up to 3 months after initiation of VPRIV®.

Other: MRI

Interventions

MRI OTHER

Study Population

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

To reflect the daily practices, this study includes all patients with a confirmed diagnosis of Type 1 Gaucher disease treated with VPRIV® at the date of beginning of the study and who meet the selection criteria of the study.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• CEN Biotech: lead
• BioClinica, Inc.: collaborator
• Takeda: collaborator

Study Sites (1)

Beaujon Hospital

Clichy, 92110, France

Location

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 26, 2017
• First Posted: November 7, 2017
• Study Start: January 17, 2017
• Primary Completion: September 13, 2017
• Study Completion: December 27, 2018
• Last Updated: June 22, 2021

Record last verified: 2021-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)