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

Phase 1

Recruiting

• Conditions: Gaucher Disease, Type 1; Gaucher Disease, Type 3
• Interventions: Drug: Low-dose CAN103; Drug: High-dose CAN103

• Registration Number: NCT05447494
• Lead Sponsor: CANbridge (Suzhou) Bio-pharma Co., Ltd.

Brief 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.

Detailed 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 Timeline

• Status Verified: 2022-07
• Study First Submitted: 2022-07-01
• Study First Submitted QC: 2022-07-01
• Study First Posted: 2022-07-07
• Study Start: 2022-07-11
• Last Update Submitted: 2022-07-25
• Last Update Posted: 2022-07-27
• Primary Completion: 2024-11-30
• Study Completion: 2024-12-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Subjects have a confirmed clinical, enzymatic, and genetic diagnosis of Gaucher disease (Type 1 or Type 3);

• Phase 1: Subjects with GD1 aged ≥18 years; Phase 2: Subjects with GD1 or GD3 aged ≥12 years;

• Subjects have not received enzyme replacement therapy (ERT) or substrate replacement therapy (SRT) within 3 months before screening;

• Subjects have GD-related anemia and one or more of the following disease manifestations:

  1. Spleen volume ≥2 MN as measured by MRI, or
  2. Liver volume ≥1.5 MN as measured by MRI, or
  3. Platelet count ≥20 × 10^9/L and <100×10^9/L.

Exclusion Criteria

• Subjects have received or stopped treatment with other investigational drugs or devices within 30 days before screening or less than 5 half-lives, whichever is longer (drugs only);
• Subjects have anemia due to other causes during screening, including nutritional anemia. Subjects whose nutritional anemia recovers with the treatment of iron, folic acid, or Vitamin B12 may be rescreened;
• Subjects have received hepatectomy or splenectomy;
• Subjects have had an allergic reaction to imiglucerase or other ERTs and their components;
• Subjects have received treatment with erythropoietin, whole blood or packed red blood cell transfusions, or chronic systemic corticosteroids within 3 months before screening, or have received a platelet transfusion within 1 month before screening.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Low-dose CAN103	Low-dose CAN103	Low dose intravenous infusion of CAN103 every other week for 37 weeks
High-dose CAN103	High-dose CAN103	High dose intravenous infusion of CAN103 every other week for 37 weeks

Primary Outcome Measures

Name	Time	Method
Mean change in hemoglobin level from Baseline to Week 39 in the high-dose group	Baseline to Week 39	Hemoglobin is measured in a central laboratory. An increase from Baseline indicates a therapeutic response.

Secondary Outcome Measures

Name	Time	Method
Mean change in hemoglobin level from Baseline to Week 39 in the low-dose group.	Baseline and Week 39	Hemoglobin is measured by a central laboratory. An increase from Baseline indicates a therapeutic response.
Mean percent change in platelet count from Baseline to Week 39 in the low-dose and high-dose groups.	Baseline to Week 39	Platelet count is measured in a central laboratory. An increase in platelet count indicates a therapeutic response.
Mean percent change in liver volume (multiples of normal, MN) measured by magnetic resonance imaging (MRI) from Baseline to Week 39 in the low-dose and high-dose groups.	Baseline to Week 39	Quantitative liver volume is calculated centrally by blinded radiologists. Normal liver volume is defined as 2.5% of body weight. A decrease from Baseline indicates a therapeutic response.
Mean percent change in spleen volume (MN) measured by MRI from Baseline to Week 39 in the low-dose and high-dose groups.	Baseline to Week 39	Quantitative spleen volume is calculated centrally by blinded radiologists. Normal spleen volume is defined at 0.2% of body weight. A decrease in spleen volume indicates a therapeutic response.

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China