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Clinical Trial Summary

Gaucher disease is a rare lysosomal storage disorder caused by deficient activity of the enzyme acid β-glucosidase, causing glucosylceramide to accumulate within macrophages and leading to hepatosplenomegaly, anemia, thrombocytopenia, and bone disease. In the non-neuronpathic form (type 1), disease manifestations are mostly systemic, whereas in the neuronopathic forms, glucosylceramide also accumulates in the central nervous sysem and leads to acute (type 2) or chronic (type 3) neurodegeneration. The purpose of this Phase 1/2 first-in-human study is to initially evaluate the safety and tolerability of two doses of CAN103, and then barring any safety concerns, to evaluate the efficacy and safety of the two doses administered intravenously every other week in treatment-naive subjects with Gaucher disease type 1 or type 3.


Clinical Trial Description

Phase 1: 4 newly treated subjects with Type I Gaucher disease (GD1). Phase 2: 36 newly treated subjects with GD1 or Type III Gaucher disease (GD3) ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05447494
Study type Interventional
Source CANbridge Life Sciences Ltd.
Contact Qionghui Qiu
Phone +86 21 52996609
Email qionghui.qiu@canbridgepharma.com
Status Recruiting
Phase Phase 1/Phase 2
Start date July 11, 2022
Completion date December 30, 2024

See also
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