Phase 1/2 Study of CAN103 in Newly Treated Subjects With Gaucher Disease
Status:
Not yet recruiting
Trial end date:
2024-12-30
Target enrollment:
Participant gender:
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.