E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | |
E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 24.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10018048 |
E.1.2 | Term | Gaucher's disease |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
- To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ - To evaluate the safety profile of imiglucerase in maximum dose in the label (60U/kg, IV biweekly) in Chinese patients.
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E.2.2 | Secondary objectives of the trial |
- To evaluate the efficacy on viscera manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ - To evaluate the efficacy on bone disease of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ - To evaluate the effect on quality of life of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
01. Capable of giving signed informed consent. I02. Participant is diagnosed with GD type Ⅲ I03. Participant with neurological manifestations I04. Participant whose age is - 2 years old. I05. Participant whose spleen and/or liver volume is - ULN at Screening.
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E.4 | Principal exclusion criteria |
E 01. Major congenital anomaly E 02. 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). E 03. Prior treatment with ERT. E 04. Physical conditions that cannot tolerate regular treatment or follow-up visit. E 05. Pregnant or lactating women. E 06. Participant is participating in or has participated in another clinical study using any investigational therapy in 3 months. E 07. 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. E 08. 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. E 09. 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. E 10. Any specific situation during study implementation/course that may rise ethics considerations. E 11. 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.
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E.5 End points |
E.5.1 | Primary end point(s) |
1/ Changes in haemoglobin 2/ Changes in platelet count 3/ Adverse events |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1/: Baseline to the end of 12 months 2/ and 3/: Baseline to the end of 13 month3 |
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E.5.2 | Secondary end point(s) |
1/ Changes in spleen volume 2/ Changes in liver volume 3/ Skeletal involvement 4/ Quality of life (QoL) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1/, 2/ and 3/: Baseline to the end of 12 months 4/: Baseline to the end of 3 months, 6 months, 9 months and 12 months |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |