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

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

Tracking Information

• First Submitted Date: July 1, 2022
• First Posted Date: July 7, 2022
• Last Update Posted Date: July 27, 2022
• Actual Study Start Date: July 11, 2022
• Estimated Primary Completion Date: November 30, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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: Same as current

Current Secondary Outcome Measures (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: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• 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: Interventional
• Study Phase: Phase 1; Phase 2

Study Design

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

• Gaucher Disease, Type 1
• Gaucher Disease, Type 3

Intervention

• 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

• 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

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: July 1, 2022): 40
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 30, 2024
• Estimated Primary Completion Date: November 30, 2024 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Qionghui Qiu; +86 21 52996609 ext 807; qionghui.qiu@canbridgepharma.com

Contact: Xiaogang Hui; xiaogang.hui@canbridgepharma.com

• Listed Location Countries: China

Administrative Information

• NCT Number: NCT05447494
• Other Study ID Numbers: 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: Not Provided
• Current Responsible Party: CANbridge Life Sciences Ltd. ( CANbridge (Suzhou) Bio-pharma Co., Ltd. )
• Original Responsible Party: Same as current
• Current Study Sponsor: CANbridge (Suzhou) Bio-pharma Co., Ltd.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Bing Han, MD; Peking Union Medical College Hospital

• PRS Account: CANbridge Life Sciences Ltd.
• Verification Date: July 2022