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Trial record 1 of 1 for:    NCT05447494
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Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease

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ClinicalTrials.gov Identifier: NCT05447494
Recruitment Status : Recruiting
First Posted : July 7, 2022
Last Update Posted : July 27, 2022
Sponsor:
Information provided by (Responsible Party):
CANbridge Life Sciences Ltd. ( CANbridge (Suzhou) Bio-pharma Co., Ltd. )

Tracking Information
First Submitted Date  ICMJE July 1, 2022
First Posted Date  ICMJE July 7, 2022
Last Update Posted Date July 27, 2022
Actual Study Start Date  ICMJE July 11, 2022
Estimated Primary Completion Date November 30, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 1, 2022)
Mean change in hemoglobin level from Baseline to Week 39 in the high-dose group [ Time Frame: Baseline to Week 39 ]
Hemoglobin is measured in a central laboratory. An increase from Baseline indicates a therapeutic response.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 1, 2022)
  • Mean percent change in platelet count from Baseline to Week 39 in the low-dose and high-dose groups. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: 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.
  • Mean change in hemoglobin level from Baseline to Week 39 in the low-dose group. [ Time Frame: Baseline and Week 39 ]
    Hemoglobin is measured by a central laboratory. An increase from Baseline indicates a therapeutic response.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
Official Title  ICMJE 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
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes 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.
Primary Purpose: Treatment
Condition  ICMJE
  • Gaucher Disease, Type 1
  • Gaucher Disease, Type 3
Intervention  ICMJE
  • Drug: Low-dose CAN103

    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.

  • Drug: High-dose CAN103

    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.

Study Arms  ICMJE
  • Experimental: Low-dose CAN103
    Low dose intravenous infusion of CAN103 every other week for 37 weeks
    Intervention: Drug: Low-dose CAN103
  • Experimental: High-dose CAN103
    High dose intravenous infusion of CAN103 every other week for 37 weeks
    Intervention: Drug: High-dose CAN103
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 1, 2022)
40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 30, 2024
Estimated Primary Completion Date November 30, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Qionghui Qiu +86 21 52996609 ext 807 qionghui.qiu@canbridgepharma.com
Contact: Xiaogang Hui xiaogang.hui@canbridgepharma.com
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05447494
Other Study ID Numbers  ICMJE CAN103-GD-201
CTR20220507 ( Registry Identifier: Center For Drug Evaluation, NMPA, China )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party CANbridge Life Sciences Ltd. ( CANbridge (Suzhou) Bio-pharma Co., Ltd. )
Original Responsible Party Same as current
Current Study Sponsor  ICMJE CANbridge (Suzhou) Bio-pharma Co., Ltd.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Bing Han, MD Peking Union Medical College Hospital
PRS Account CANbridge Life Sciences Ltd.
Verification Date July 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP