Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
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.
Timeline
- Start
- 2022-07-11
- Primary completion
- 2024-11-30
- Completion
- 2024-12-30
Outcome
Met primary endpoint
release “The pivotal clinical trial of CAN103 achieved positive top-line data in August 2024.” canbridgepharma.com ↗
release “The results demonstrate that the study successfully met its primary efficacy endpoint” canbridgepharma.com ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | CAN103 | Unknown | — | Intravenous |