E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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 | 24.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10016016 |
E.1.2 | Term | Fabry'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 safety and tolerability of Fabrazyme in Chinese Fabry disease participants |
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E.2.2 | Secondary objectives of the trial |
To evaluate the changes of plasma globotriaosylsphingosine (lysoGL3) level with Fabrazyme treatment in Chinese Fabry disease participants To evaluate the changes of plasma globotriaosylceramide (GL3) level with Fabrazyme treatment in Chinese Fabry disease participants To evaluate the number and percentage of participants with abnormal plasma GL3 value with Fabrazyme treatment in Chinese Fabry disease participants To evaluate the change of symptoms of Fabry disease with Fabrazyme treatment in Chinese Fabry disease participants To evaluate the change in renal function with Fabrazyme treatment in Chinese Fabry disease participants
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Participant must be 8 years of age or older, at the time of signing the informed consent Participants naive to agalsidase beta and agalsidase alpha Chinese participants diagnosed with Fabry disease and with documented plasma or leukocyte αGAL activity deficient below laboratory’s reference range, and/or documented diagnosis by genotyping Participants must have one or more symptoms and signs consistent with manifestations of Fabry disease (not limited to neuropathic pain, chronic kidney disease, hypertrophic cardiomyopathy, cardiac rhythm disturbances, cerebrovascular involvement, cornea verticillata, angiokeratoma, gastrointestinal symptoms, hypo- or anhydrosis) A female participant is eligible to participate if she is not pregnant or breastfeeding and use an acceptable contraceptive method Participants and/or participant’s legal representative capable of giving signed informed consent.
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E.4 | Principal exclusion criteria |
Participants are excluded from the study if any of the following criteria apply: The participant has undergone kidney transplantation. The participant has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) in the opinion of the Investigator, would preclude participation in the trial. Received an investigational drug, or device, other than Fabrazyme, within 30 days of anticipated IMPs administration or 5 half-lives of the previous investigational drug, whichever is longer. The patient has current evidence of kidney failure or renal insufficiency, as defined by eGFR <30 mL/min/1.73 m2. Individuals who have life threatening hypersensitivity (anaphylactic reaction) to the active substance or any of the excipients included.
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E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of treatment-emergent adverse events (AEs) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1/ The absolute changes of plasma lyso-GL3
2/ The percent changes of plasma lyso-GL3
3/ The absolute changes of plasma GL3
4/ The percent changes of plasma GL3
5/ The number of participants with abnormal plasma GL3 values per central lab reference range
6/ The percentage of participants with abnormal plasma GL3 values per central lab reference range
7/ The change of Fabry disease symptoms
8/ The absolute change of estimated glomerular filtration rate (eGFR) by chronic kidney disease epidemiology collaboration (CKD-EPI) for adult (≥18 years)
9/ The absolute change of estimated glomerular filtration rate (eGFR) by Schwartz for children (8 ≤age <18 years)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1/ to 6/ from baseline to Week 6, Week 12, Week 24 and Week 48
7/from baseline to Week 24 and Week 48
8/ and 9/ from baseline to Week 12, Week 24, Week 36 and Week 48 |
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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 | No |
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 | 5 |