Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease

Gaucher Disease, Type 1 Clinical Trial

Official title: A Phase 1/2 Open-label, Dose Escalation Study Followed by a Multi-center, Randomized, Double-blind, Dose Comparison Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics of CAN103 in Newly Treated Gaucher Disease

Status Recruiting

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) ;

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease, Type 1
• Gaucher Disease, Type 3

• 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