Back to resultsPhase 1/2 Study of CAN103 in Subjects With Gaucher Disease
Primary Purpose
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Status
Recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Low-dose CAN103
High-dose CAN103
Sponsored by
About this trial
This is an interventional treatment trial for Gaucher Disease, Type 1 focused on measuring CAN103, Gaucher disease
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:
- Spleen volume ≥2 MN as measured by MRI, or
- Liver volume ≥1.5 MN as measured by MRI, or
- 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.
Sites / Locations
- Peking Union Medical College HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Low-dose CAN103
High-dose CAN103
Arm Description
Low dose intravenous infusion of CAN103 every other week for 37 weeks
High dose intravenous infusion of CAN103 every other week for 37 weeks
Outcomes
Primary Outcome Measures
Mean change in hemoglobin level from Baseline to Week 39 in the high-dose group
Hemoglobin is measured in a central laboratory. An increase from Baseline indicates a therapeutic response.
Secondary Outcome Measures
Mean percent change in platelet count from Baseline to Week 39 in the low-dose and high-dose groups.
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.
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.
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.
Mean change in hemoglobin level from Baseline to Week 39 in the low-dose group.
Hemoglobin is measured by a central laboratory. An increase from Baseline indicates a therapeutic response.
Full Information
NCT ID
NCT05447494
First Posted
July 1, 2022
Last Updated
July 25, 2022
Sponsor
CANbridge (Suzhou) Bio-pharma Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT05447494
Brief Title
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
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Recruiting
Study Start Date
July 11, 2022 (Actual)
Primary Completion Date
November 30, 2024 (Anticipated)
Study Completion Date
December 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CANbridge (Suzhou) Bio-pharma Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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)
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Keywords
CAN103, Gaucher disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Phase 2 is blinded to dose group. Except for the non-blind team, no other participants associated with this study should attempt to learn the treatment group assignment or which study treatment they are receiving. The unblinding of all subjects will take place after database lock.
Allocation
Randomized
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Low-dose CAN103
Arm Type
Experimental
Arm Description
Low dose intravenous infusion of CAN103 every other week for 37 weeks
Arm Title
High-dose CAN103
Arm Type
Experimental
Arm Description
High dose intravenous infusion of CAN103 every other week for 37 weeks
Intervention Type
Drug
Intervention Name(s)
Low-dose CAN103
Intervention Description
Phase 1 is a within-subject dose escalation study to evaluate the safety, tolerability, and pharmacokinetics of two doses of CAN103 in newly treated subjects with GD1.
Phase 2 is a randomized, double-blind, parallel group, dose comparison study to evaluate the efficacy and safety of two doses of CAN103 administered intravenously every other week for 37 weeks in newly treated GD1 or GD3 subjects with significant non-neurological clinical manifestations.
Intervention Type
Drug
Intervention Name(s)
High-dose CAN103
Intervention Description
Phase 1 is a within-subject dose escalation study to evaluate the safety, tolerability, and pharmacokinetics of two doses of CAN103 in newly treated subjects with GD1.
Phase 2 is a randomized, double-blind, parallel group, dose comparison study to evaluate the efficacy and safety of two doses of CAN103 administered intravenously every other week for 37 weeks in newly treated GD1 or GD3 subjects with significant non-neurological clinical manifestations.
Primary Outcome Measure Information:
Title
Mean change in hemoglobin level from Baseline to Week 39 in the high-dose group
Description
Hemoglobin is measured in a central laboratory. An increase from Baseline indicates a therapeutic response.
Time Frame
Baseline to Week 39
Secondary Outcome Measure Information:
Title
Mean percent change in platelet count from Baseline to Week 39 in the low-dose and high-dose groups.
Description
Platelet count is measured in a central laboratory. An increase in platelet count indicates a therapeutic response.
Time Frame
Baseline to Week 39
Title
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.
Description
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.
Time Frame
Baseline to Week 39
Title
Mean percent change in spleen volume (MN) measured by MRI from Baseline to Week 39 in the low-dose and high-dose groups.
Description
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.
Time Frame
Baseline to Week 39
Title
Mean change in hemoglobin level from Baseline to Week 39 in the low-dose group.
Description
Hemoglobin is measured by a central laboratory. An increase from Baseline indicates a therapeutic response.
Time Frame
Baseline and Week 39
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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:
Spleen volume ≥2 MN as measured by MRI, or
Liver volume ≥1.5 MN as measured by MRI, or
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Qionghui Qiu
Phone
+86 21 52996609
Ext
807
Email
qionghui.qiu@canbridgepharma.com
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaogang Hui
Email
xiaogang.hui@canbridgepharma.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bing Han, MD
Organizational Affiliation
Peking Union Medical College Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking Union Medical College Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100730
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Qionghui Qiu
Email
qionghui.qiu@canbridgepharma.com
12. IPD Sharing Statement
Learn more about this trial
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
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