Gaucher Disease, Type 1EIROSNCT03333447
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)
An observational study of Gaucher Disease, Type 1 and MRI, run by CEN Biotech at 1 site in France.
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®. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Gaucher Disease, Type 1, MRI
- What is being tested?
- MRI
- How many people
- 20 participants
- Where
- 1 siteFrance
- Study dates
- 17 Jan 2017to 27 Dec 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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®.
Read the 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 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.
- Conditions
- Gaucher Disease, Type 1, MRI
- Being tested
- MRI
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRIOther
| Study group | Role | What they receive |
|---|---|---|
| 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®. Receives: Other: MRI |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 2 YEARS
Secondary outcomes (42)
Describe socio-demographic characteristics of patients : age
To describe the socio-demographic of patients : age
Measured: 2 YEARS
Describe socio-demographic characteristics of patients : sex
To describe the socio-demographic of patients : sex
Measured: 2 YEARS
Describe socio-demographic characteristics of patients : occupation
To describe the socio-demographic of patients : occupation
Measured: 2 YEARS
Clinical evolution of patients :weight (kg)
To describe the clinical evolution of patients : weight (kg)
Measured: 2 YEARS
Clinical evolution of patients : height (cm)
To describe the clinical evolution of patients : height (cm)
Measured: 2 YEARS
Clinical evolution of patients : absence or presence (mild, moderate or severe) asthenia
To describe the clinical evolution of patients : absence or presence (mild, moderate or severe) asthenia
Measured: 2 YEARS
Clinical evolution of patients : abdominal pain
To describe the clinical evolution of patients : abdominal pain
Measured: 2 YEARS
Clinical evolution of patients : chronic bone pain
To describe the clinical evolution of patients : chronic bone pain
Measured: 2 YEARS
Clinical evolution of patients : bone crises
To describe the clinical evolution of patients : bone crises
Measured: 2 YEARS
Clinical evolution of patients : bleeding and hemorrhagic syndrome
To describe the clinical evolution of patients : bleeding and hemorrhagic syndrome
Measured: 2 YEARS
Clinical evolution of patients : pulmonary impairment
To describe the clinical evolution of patients : pulmonary impairment
Measured: 2 YEARS
Clinical evolution of patients : neurological impairment
To describe the clinical evolution of patients : neurological impairment
Measured: 2 YEARS
Clinical evolution of patient : absence or presence of hepatomegaly
To describe the clinical evolution of patients : absence or presence of hepatomegaly, with the extent of the arrow (medio-clavicular line) (cm)
Measured: 2 YEARS
Clinical evolution of patients : absence or presence of splenomegaly
To describe the clinical evolution of patients : absence or presence of splenomegaly, with the extent of the costal overhang (cm)
Measured: 2 YEARS
Evolution of the parameters measured by MRI : Bone alterations
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.
Measured: 2 YEARS
Evolution of the parameters measured by MRI : Liver evaluation : absence or presence of hepatomegaly with the extent of the arrow (medio-clavicular line) (cm).
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.
Measured: 2 YEARS
Evolution of the parameters measured by MRI : Liver evaluation : evolution of the liver volume (m3).
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.
Measured: 2 YEARS
Evolution of the parameters measured by MRI : Spleen evaluation : absence or presence of splenomegaly
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.
Measured: 2 YEARS
Evolution of the parameters measured by MRI : Spleen evaluation : evolution of the extent of the costal overhang (cm)
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.
Measured: 2 YEARS
Evolution of the parameters measured by MRI : Spleen evaluation : absence or presence of splenomegaly.
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.
Measured: 2 YEARS
Evolution of the parameters measured by MRI : Spleen evaluation : evolution of the spleen volume (m3)
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.
Measured: 2 YEARS
Evolution of the parameters measured by MRI : Presence or absence of myocardia alterations
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.
Measured: 2 YEARS
Evolution of Biological parameters : hemoglobin (g/dl)
To describe the evolution of biological parameters : hemoglobin (g/dl)
Measured: 2 YEARS
Evolution of Biological parameters : leukocytes (cells/mm3)
To describe the evolution of biological parameters : leukocytes (cells/mm3)
Measured: 2 YEARS
Evolution of Biological parameters : platelets (mm3)
To describe the evolution of biological parameters : platelets (mm3)
Measured: 2 YEARS
Evolution of Biological parameters : Angiotensin Converting Enzyme (ACE) (U/l)
To describe the evolution of biological parameters : ACE (angiotensin converting enzyme (U/l)
Measured: 2 YEARS
Evolution of Biological parameters: ferritin (mg/l)
To describe the evolution of biological parameters : ferritin (mg/l)
Measured: 2 YEARS
Evolution of Biological parameters : chitotriosidase (nmol/h/ml)
To describe the evolution of biological parameters : chitotriosidase (nmol/h/ml)
Measured: 2 YEARS
Evolution of Biological parameters : CCL18 (ng/ml)
To describe the evolution of biological parameters : CCL18 (ng/ml)
Measured: 2 YEARS
Evolution of Biological parameters : vitamin D (nmol/L)
To describe the evolution of biological parameters : vitamin D (nmol/L)
Measured: 2 YEARS
Evolution of Biological parameters : vitamin B12 (pmol/l)
To describe the evolution of biological parameters : vitamin B12 (pmol/l)
Measured: 2 YEARS
Evolution of Biological parameters : serum glutamate oxaloacetate transaminase (SGOT) (U/l)
To describe the evolution of biological parameters : SGOT (U/l)
Measured: 2 YEARS
Evolution of Biological parameters : Aspartate aminotransferase (AST) (U/l)
To describe the evolution of biological parameters : AST (U/l)
Measured: 2 YEARS
Evolution of Biological parameters : serum glutamate pyruvate transaminase (SGPT) (U/l)
To describe the evolution of biological parameters : SGPT (U/l)
Measured: 2 YEARS
Evolution of Biological parameters : Alanine Aminotransferase (ALT) (U/l)
To describe the evolution of biological parameters : ALT (U/l)
Measured: 2 YEARS
Evolution of Biological parameters : Gamma-glutamyltransferase (GGT) (U/l)
To describe the evolution of biological parameters : GGT (U/l)
Measured: 2 YEARS
Evolution of Biological parameters : alkaline phosphatase (U/l)
To describe the evolution of biological parameters : alkaline phosphatase (U/l)
Measured: 2 YEARS
Evolution of Biological parameters : triglycerides (mmol/l)
To describe the evolution of biological parameters : triglycerides (mmol/l)
Measured: 2 YEARS
Evolution of Biological parameters : total cholesterol (mmol/l)
To describe the evolution of biological parameters : total cholesterol (mmol/l)
Measured: 2 YEARS
Evolution of Biological parameters : gamma globulin (g/l)
To describe the evolution of biological parameters : gamma globulin (g/l).
Measured: 2 YEARS
Evolution of Biological parameters : serum calcium (mmol/l)
To describe the evolution of biological parameters : serum calcium (mmol/l)
Measured: 2 YEARS
Evolution of Biological parameters : presence or absence of a monoclonal peak and the type if any.
To describe the evolution of biological parameters : presence or absence of a monoclonal peak and the type if any.
Measured: 2 YEARS
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2017
- Study start17 Jan 2017
- Primary completion13 Sep 2017
- Study completion27 Dec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease, Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- CEN Biotech
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- BioClinica, Inc., Takeda
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03333447
- Sponsor's protocol ID
- C1294
- Acronym
- EIROS
- Status last verified
- Jun 2021
- First submitted
- 26 Oct 2017
- Last updated
- 22 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03333447
- Acronym / sponsor ID
- EIROS / C1294
- Conditions
- Gaucher Disease, Type 1, MRI
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- CEN BiotechIndustry (pharma, biotech, devices)
- Collaborators
- BioClinica, Inc., Takeda
- Study start
- 17 Jan 2017 (actual)
- Primary completion
- 13 Sep 2017 (actual)
- Study completion
- 27 Dec 2018 (actual)
- First posted
- 7 Nov 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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®.
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 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
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study Population | – | Other: MRIPatients 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®. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MRI | Other | Study Population |
Primary outcome
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.
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.
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.
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.
42 secondary outcomes
Describe socio-demographic characteristics of patients : age
Time frame: 2 YEARS
Describe socio-demographic characteristics of patients : sex
Time frame: 2 YEARS
Describe socio-demographic characteristics of patients : occupation
Time frame: 2 YEARS
Clinical evolution of patients :weight (kg)
Time frame: 2 YEARS
Clinical evolution of patients : height (cm)
Time frame: 2 YEARS
Clinical evolution of patients : absence or presence (mild, moderate or severe) asthenia
Time frame: 2 YEARS
Clinical evolution of patients : abdominal pain
Time frame: 2 YEARS
Clinical evolution of patients : chronic bone pain
Time frame: 2 YEARS
Clinical evolution of patients : bone crises
Time frame: 2 YEARS
Clinical evolution of patients : bleeding and hemorrhagic syndrome
Time frame: 2 YEARS
Clinical evolution of patients : pulmonary impairment
Time frame: 2 YEARS
Clinical evolution of patients : neurological impairment
Time frame: 2 YEARS
Clinical evolution of patient : absence or presence of hepatomegaly
Time frame: 2 YEARS
Clinical evolution of patients : absence or presence of splenomegaly
Time frame: 2 YEARS
Evolution of the parameters measured by MRI : Bone alterations
Time frame: 2 YEARS
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
Evolution of the parameters measured by MRI : Liver evaluation : evolution of the liver volume (m3).
Time frame: 2 YEARS
Evolution of the parameters measured by MRI : Spleen evaluation : absence or presence of splenomegaly
Time frame: 2 YEARS
Evolution of the parameters measured by MRI : Spleen evaluation : evolution of the extent of the costal overhang (cm)
Time frame: 2 YEARS
Evolution of the parameters measured by MRI : Spleen evaluation : absence or presence of splenomegaly.
Time frame: 2 YEARS
Evolution of the parameters measured by MRI : Spleen evaluation : evolution of the spleen volume (m3)
Time frame: 2 YEARS
Evolution of the parameters measured by MRI : Presence or absence of myocardia alterations
Time frame: 2 YEARS
Evolution of Biological parameters : hemoglobin (g/dl)
Time frame: 2 YEARS
Evolution of Biological parameters : leukocytes (cells/mm3)
Time frame: 2 YEARS
Evolution of Biological parameters : platelets (mm3)
Time frame: 2 YEARS
Evolution of Biological parameters : Angiotensin Converting Enzyme (ACE) (U/l)
Time frame: 2 YEARS
Evolution of Biological parameters: ferritin (mg/l)
Time frame: 2 YEARS
Evolution of Biological parameters : chitotriosidase (nmol/h/ml)
Time frame: 2 YEARS
Evolution of Biological parameters : CCL18 (ng/ml)
Time frame: 2 YEARS
Evolution of Biological parameters : vitamin D (nmol/L)
Time frame: 2 YEARS
Evolution of Biological parameters : vitamin B12 (pmol/l)
Time frame: 2 YEARS
Evolution of Biological parameters : serum glutamate oxaloacetate transaminase (SGOT) (U/l)
Time frame: 2 YEARS
Evolution of Biological parameters : Aspartate aminotransferase (AST) (U/l)
Time frame: 2 YEARS
Evolution of Biological parameters : serum glutamate pyruvate transaminase (SGPT) (U/l)
Time frame: 2 YEARS
Evolution of Biological parameters : Alanine Aminotransferase (ALT) (U/l)
Time frame: 2 YEARS
Evolution of Biological parameters : Gamma-glutamyltransferase (GGT) (U/l)
Time frame: 2 YEARS
Evolution of Biological parameters : alkaline phosphatase (U/l)
Time frame: 2 YEARS
Evolution of Biological parameters : triglycerides (mmol/l)
Time frame: 2 YEARS
Evolution of Biological parameters : total cholesterol (mmol/l)
Time frame: 2 YEARS
Evolution of Biological parameters : gamma globulin (g/l)
Time frame: 2 YEARS
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Gaucher Disease, Type 1 and MRI, run by CEN Biotech at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2021.