Clinical trial NCT05447494: Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Gaucher Disease, Type 1
Gaucher Disease, Type 3
Keywords: CAN103
Gaucher disease
Lead sponsor: CANbridge Life Sciences Ltd.
Sponsor class: INDUSTRY
First posted: 2022-07-07
Enrollment: 40
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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)

Eligibility criteria: 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.

Primary outcomes:
measure: Mean change in hemoglobin level from Baseline to Week 39 in the high-dose group
timeFrame: Baseline to Week 39
Secondary outcomes:
measure: Mean percent change in platelet count from Baseline to Week 39 in the low-dose and high-dose groups.
timeFrame: Baseline to Week 39
measure: 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.
timeFrame: Baseline to Week 39
measure: Mean percent change in spleen volume (MN) measured by MRI from Baseline to Week 39 in the low-dose and high-dose groups.
timeFrame: Baseline to Week 39
measure: Mean change in hemoglobin level from Baseline to Week 39 in the low-dose group.
timeFrame: Baseline and Week 39
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
affiliation: Peking Union Medical College Hospital
name: Bing Han, MD
role: PRINCIPAL_INVESTIGATOR
References: