SEED: Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
Study Details
Study Description
Brief Summary
Primary Objective
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To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
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To evaluate the safety profile of imiglucerase in maximum dose in the label (60U/kg, IV biweekly) in Chinese patients.
Secondary Objective
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To evaluate the efficacy on viscera manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
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To evaluate the efficacy on bone disease of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
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To evaluate the effect on quality of life of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 4 |
Detailed Description
Approximatively 14 months including a 12 months treatment period
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Cerezyme® / Imiglucerase Cerezyme® (imiglucerase for injection) is administered by intravenous infusion, 60 U/kg once every 2 weeks. |
Drug: Cerezyme® / Imiglucerase
Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous
|
Outcome Measures
Primary Outcome Measures
- Changes in haemoglobin [Baseline to the end of 12 months]
The mean changes in haemoglobin
- Changes in platelet count [Baseline to the end of 12 months]
The mean changes in platelet count
- Adverse events [Baseline to the end of 13 months]
Number of participants with AEs
Secondary Outcome Measures
- Changes in spleen volume [Baseline to the end of 12 months]
The mean change of spleen volumne
- Changes in liver volume [Baseline to the end of 12 months]
The mean change of liver volumne
- Skeletal involvement [Baseline to the end of 12 months]
The changes in frequency, duration and severity of bone pain and number of bone crisis The changes in frequency, duration and severity of bone pain and number of bone crisisThe changes in frequency, duration and severity of bone pain and number of bone crisis
- Quality of life (QoL) [Baseline to the end of 3 months, 6 months, 9 months and 12 months]
The mean change of QoL
Eligibility Criteria
Criteria
Inclusion criteria:
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Capable of giving signed informed consent.
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Participant is diagnosed with GD type Ⅲ
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Participant with neurological manifestations
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Participant whose age is > 2 years old.
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Participant whose spleen and/or liver volume is > ULN at Screening.
Exclusion criteria:
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Major congenital anomaly
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Clinically significant intercurrent organic disease unrelated to Gaucher disease, which means the disease or condition that may have impact on the parameters chosen for primary endpoints (e.g. level of hemoglobin platelets, liver/spleen enlargement and bone pains)
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Prior treatment with ERT.
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Physical conditions that cannot tolerate regular treatment or follow-up visit.
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Pregnant or lactating women
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Participant is participating in or has participated in another clinical study using any investigational therapy in 3 months
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Participant has been diagnosed with central nervous system disease unrelated to Gaucher disease, or MRI result of the participant indicates space-occupying lesion in central nervous system
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The patient has a documented hemoglobinopathies, deficiency of iron, vitamin B-12, or folate that requires treatment not yet initiated or, if initiated, the patient has not been stable under treatment for at least 6 months prior to administration of the first dose of Cerezyme in this study
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Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
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Any specific situation during study implementation/course that may rise ethics considerations
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Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Investigational Site Number :107 | Beijing | China | 100020 | |
| 2 | Investigational Site Number :101 | Beijing | China | 100730 | |
| 3 | Investigational Site Number :104 | Chengdu | China | 610041 | |
| 4 | Investigational Site Number :102 | Guangzhou | China | 510080 | |
| 5 | Investigational Site Number :105 | Guangzhou | China | 510623 |
Sponsors and Collaborators
- Sanofi
Investigators
- Study Director: Clinical Sciences & Operations, Sanofi
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- LPS16031
- U1111-1244-1166