ClinicalTrials.gov
ClinicalTrials.gov Menu

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) (EIROS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03333447
Recruitment Status : Recruiting
First Posted : November 7, 2017
Last Update Posted : July 18, 2018
Sponsor:
Collaborators:
Shire
BioClinica, Inc.
Information provided by (Responsible Party):
CEN Biotech

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®.


Condition or disease Intervention/treatment
Gaucher Disease, Type 1 MRI Other: MRI

  Show Detailed Description

Study Type : Observational
Estimated Enrollment : 40 participants
Observational Model: Other
Time Perspective: Cross-Sectional
Official Title: 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)
Actual Study Start Date : January 17, 2017
Actual Primary Completion Date : September 13, 2017
Estimated Study Completion Date : December 2019


Group/Cohort Intervention/treatment
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



Primary Outcome Measures :
  1. The change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T1 measured at the femur. [ Time Frame: 2 YEARS ]
    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. The change over time between the baseline MRI and the follow-up MRIs, of the BMB score weighted at T2 measured at the femur. [ Time Frame: 2 YEARS ]
    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.

  3. 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. [ Time Frame: 2 YEARS ]
    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.

  4. 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. [ Time Frame: 2 YEARS ]
    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.


Secondary Outcome Measures :
  1. Describe socio-demographic characteristics of patients : age [ Time Frame: 2 YEARS ]
    To describe the socio-demographic of patients : age

  2. Describe socio-demographic characteristics of patients : sex [ Time Frame: 2 YEARS ]
    To describe the socio-demographic of patients : sex

  3. Describe socio-demographic characteristics of patients : occupation [ Time Frame: 2 YEARS ]
    To describe the socio-demographic of patients : occupation

  4. Clinical evolution of patients :weight (kg) [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : weight (kg)

  5. Clinical evolution of patients : height (cm) [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : height (cm)

  6. Clinical evolution of patients : absence or presence (mild, moderate or severe) asthenia [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : absence or presence (mild, moderate or severe) asthenia

  7. Clinical evolution of patients : abdominal pain [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : abdominal pain

  8. Clinical evolution of patients : chronic bone pain [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : chronic bone pain

  9. Clinical evolution of patients : bone crises [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : bone crises

  10. Clinical evolution of patients : bleeding and hemorrhagic syndrome [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : bleeding and hemorrhagic syndrome

  11. Clinical evolution of patients : pulmonary impairment [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : pulmonary impairment

  12. Clinical evolution of patients : neurological impairment [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : neurological impairment

  13. Clinical evolution of patient : absence or presence of hepatomegaly [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : absence or presence of hepatomegaly, with the extent of the arrow (medio-clavicular line) (cm)

  14. Clinical evolution of patients : absence or presence of splenomegaly [ Time Frame: 2 YEARS ]
    To describe the clinical evolution of patients : absence or presence of splenomegaly, with the extent of the costal overhang (cm)

  15. Evolution of the parameters measured by MRI : Bone alterations [ Time Frame: 2 YEARS ]

    To describe the evolution of the parameters measured by MRI : bone alterations (spine, pelvis, femurs, tibias and other symptomatic localization). Absence or presence of bone lesions and their locations: bone infiltration, cortical thinning, osteonecrosis, bone infarction, stroke sequelae, vertebral compression, fracture or other bone disease.

    If possible, compare the evolution of MRI parameters between treatment-naive patients and patients previously treated with another specific treatment for Gaucher disease at the baseline MRI.


  16. Evolution of the parameters measured by MRI : Liver evaluation : absence or presence of hepatomegaly with the extent of the arrow (medio-clavicular line) (cm). [ Time Frame: 2 YEARS ]

    To describe the evolution of the parameters measured by MRI : Visceral involvement : Liver evaluation criteria MRI : absence or presence of hepatomegaly, with the extent of the arrow (medio-clavicular line) (cm).

    If possible, compare the evolution of this MRI parameters between treatment-naive patients and patients previously treated with another specific treatment for Gaucher disease at the baseline MRI.


  17. Evolution of the parameters measured by MRI : Liver evaluation : evolution of the liver volume (m3). [ Time Frame: 2 YEARS ]

    To describe the evolution of the parameters measured by MRI : Liver evaluation criteria MRI : evolution of the liver volume (m3).

    If possible, compare the evolution of this MRI parameters between treatment-naive patients and patients previously treated with another specific treatment for Gaucher disease at the baseline MRI.


  18. Evolution of the parameters measured by MRI : Spleen evaluation : absence or presence of splenomegaly [ Time Frame: 2 YEARS ]

    To describe the evolution of the parameters measured by MRI : Spleen evaluation MRI : absence or presence of splenomegaly.

    If possible, compare the evolution of this MRI parameters between treatment-naive patients and patients previously treated with another specific treatment for Gaucher disease at the baseline MRI.


  19. Evolution of the parameters measured by MRI : Spleen evaluation : evolution of the extent of the costal overhang (cm) [ Time Frame: 2 YEARS ]

    To describe the evolution of the parameters measured by MRI : Spleen evaluation MRI : evolution of the extent of the costal overhang (cm).

    If possible, compare the evolution of this MRI parameters between treatment-naive patients and patients previously treated with another specific treatment for Gaucher disease at the baseline MRI.


  20. Evolution of the parameters measured by MRI : Spleen evaluation : absence or presence of splenomegaly. [ Time Frame: 2 YEARS ]

    To describe the evolution of the parameters measured by MRI : Spleen evaluation MRI : absence or presence of splenomegaly.

    If possible, compare the evolution of this MRI parameters between treatment-naive patients and patients previously treated with another specific treatment for Gaucher disease at the baseline MRI.


  21. Evolution of the parameters measured by MRI : Spleen evaluation : evolution of the spleen volume (m3) [ Time Frame: 2 YEARS ]

    To describe the evolution of the parameters measured by MRI : Spleen evaluation MRI : evolution of the spleen volume (m3).

    If possible, compare the evolution of this MRI parameters between treatment-naive patients and patients previously treated with another specific treatment for Gaucher disease at the baseline MRI.


  22. Evolution of the parameters measured by MRI : Presence or absence of myocardia alterations [ Time Frame: 2 YEARS ]

    To describe the evolution of the parameters measured by MRI : Presence or absence of myocardial, nodules, fibrosis, vesicular stones or other alterations.

    If possible, compare the evolution of MRI parameters between treatment-naive patients and patients previously treated with another specific treatment for Gaucher disease at the baseline MRI.


  23. Evolution of Biological parameters : hemoglobin (g/dl) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : hemoglobin (g/dl)

  24. Evolution of Biological parameters : leukocytes (cells/mm3) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : leukocytes (cells/mm3)

  25. Evolution of Biological parameters : platelets (mm3) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : platelets (mm3)

  26. Evolution of Biological parameters : Angiotensin Converting Enzyme (ACE) (U/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : ACE (angiotensin converting enzyme (U/l)

  27. Evolution of Biological parameters: ferritin (mg/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : ferritin (mg/l)

  28. Evolution of Biological parameters : chitotriosidase (nmol/h/ml) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : chitotriosidase (nmol/h/ml)

  29. Evolution of Biological parameters : CCL18 (ng/ml) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : CCL18 (ng/ml)

  30. Evolution of Biological parameters : vitamin D (nmol/L) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : vitamin D (nmol/L)

  31. Evolution of Biological parameters : vitamin B12 (pmol/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : vitamin B12 (pmol/l)

  32. Evolution of Biological parameters : serum glutamate oxaloacetate transaminase (SGOT) (U/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : SGOT (U/l)

  33. Evolution of Biological parameters : Aspartate aminotransferase (AST) (U/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : AST (U/l)

  34. Evolution of Biological parameters : serum glutamate pyruvate transaminase (SGPT) (U/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : SGPT (U/l)

  35. Evolution of Biological parameters : Alanine Aminotransferase (ALT) (U/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : ALT (U/l)

  36. Evolution of Biological parameters : Gamma-glutamyltransferase (GGT) (U/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : GGT (U/l)

  37. Evolution of Biological parameters : alkaline phosphatase (U/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : alkaline phosphatase (U/l)

  38. Evolution of Biological parameters : triglycerides (mmol/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : triglycerides (mmol/l)

  39. Evolution of Biological parameters : total cholesterol (mmol/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : total cholesterol (mmol/l)

  40. Evolution of Biological parameters : gamma globulin (g/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : gamma globulin (g/l).

  41. Evolution of Biological parameters : serum calcium (mmol/l) [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : serum calcium (mmol/l)

  42. Evolution of Biological parameters : presence or absence of a monoclonal peak and the type if any. [ Time Frame: 2 YEARS ]
    To describe the evolution of biological parameters : presence or absence of a monoclonal peak and the type if any.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Criteria

Inclusion Criteria:

  1. Patients of any age or gender with confirmed diagnosis of type 1 Gaucher disease,
  2. 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®
  3. Patients should have one MRI data in the 5 previous years before starting VPRIV® Treatment (up to 3 months after initiation of VPRIV®
  4. 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 Criteria:

  1. Patients for whom MRI is contra-indicated
  2. 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®.
  3. Patients included in an ongoing clinical trial where the product is blinded

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03333447


Contacts
Contact: ALLAERT FRANCOIS ANDRE +33 3 80 68 05 05 francois.allaert@groupecen.com

Locations
France
Beaujon Hospital Recruiting
Clichy, France, 92110
Contact: BELMATOUG NADIA, DOCTOR    +33 1 40 87 52 86    nadia.belmatoug@aphp.fr   
Contact: BENGHERBIA MONIA, CRA    +33 1 71 11 46 08    monia.bengherbia@aphp.fr   
Sponsors and Collaborators
CEN Biotech
Shire
BioClinica, Inc.
  Study Documents (Full-Text)

Documents provided by CEN Biotech:
Study Protocol  [PDF] April 5, 2016


Responsible Party: CEN Biotech
ClinicalTrials.gov Identifier: NCT03333447     History of Changes
Other Study ID Numbers: C1294
First Posted: November 7, 2017    Key Record Dates
Last Update Posted: July 18, 2018
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Gaucher Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors