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

NCT05447494 | Unknown Phase 1

• 2 other identifiers: CAN103-GD-201CTR20220507
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Gaucher Disease, Type 1; Gaucher Disease, Type 3

Keywords

CAN103; Gaucher disease

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline to Week 39

Secondary Outcomes (4)

• Mean percent change in platelet count from Baseline to Week 39 in the low-dose and high-dose groups.

  Baseline to Week 39

• 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.

  Baseline to Week 39

• Mean percent change in spleen volume (MN) measured by MRI from Baseline to Week 39 in the low-dose and high-dose groups.

  Baseline to Week 39

• Mean change in hemoglobin level from Baseline to Week 39 in the low-dose group.

  Baseline and Week 39

Study Arms (2)

Low-dose CAN103

EXPERIMENTAL

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

Drug: Low-dose CAN103

High-dose CAN103

EXPERIMENTAL

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

Drug: High-dose CAN103

Interventions

Low-dose CAN103 DRUG

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.

Low-dose CAN103

High-dose CAN103 DRUG

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.

High-dose CAN103

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CANbridge (Suzhou) Bio-pharma Co., Ltd.: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Bing Han, MD

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Qionghui Qiu

CONTACT

+86 21 52996609; qionghui.qiu@canbridgepharma.com

Xiaogang Hui

CONTACT

xiaogang.hui@canbridgepharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 1, 2022
• First Posted: July 7, 2022
• Study Start: July 11, 2022
• Primary Completion: November 30, 2024
• Study Completion: December 30, 2024
• Last Updated: July 27, 2022

Record last verified: 2022-07

Locations

CN (1)