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

CANbridge Life Sciences

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Gaucher Disease, Type 3; Gaucher Disease, Type 1

Treatments: Drug: Low-dose CAN103; Drug: High-dose CAN103

Study type: Interventional

Funder types: Industry

Identifiers

NCT05447494

CAN103-GD-201

CTR20220507 (Registry Identifier)

Details and patient eligibility

About

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.

Full 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)

Enrollment: 40 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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40 participants in 2 patient groups

Low-dose CAN103

Experimental group

Description:

Low dose intravenous infusion of CAN103 every other week for 37 weeks

Treatment:

Drug: Low-dose CAN103

High-dose CAN103

Experimental group

Description:

High dose intravenous infusion of CAN103 every other week for 37 weeks

Treatment:

Drug: High-dose CAN103