Gaucher Disease, Type 1Phase 1/2NCT05447494
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
A Phase 1/2 study testing Low-dose CAN103 and High-dose CAN103 in people with Gaucher Disease, Type 1 and Gaucher Disease, Type 3, run by CANbridge (Suzhou) Bio-pharma Co., Ltd. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Gaucher Disease, Type 1, Gaucher Disease, Type 3
- What is being tested?
- Low-dose CAN103 and High-dose CAN103
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 11 Jul 2022to 30 Dec 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
Xiaogang Hui contact
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Qionghui Qiu, qionghui.qiu@canbridgepharma.com
About this study
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
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.
Read the 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)
- Conditions
- Gaucher Disease, Type 1, Gaucher Disease, Type 3
- Being tested
- Low-dose CAN103, High-dose CAN103
- Keywords
- CAN103, Gaucher disease
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low-dose CAN103Drug
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 CAN103Drug
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 group | Role | What they receive |
|---|---|---|
| Low-dose CAN103 | Experimental | Low dose intravenous infusion of CAN103 every other week for 37 weeks Receives: Drug: Low-dose CAN103 |
| High-dose CAN103 | Experimental | High dose intravenous infusion of CAN103 every other week for 37 weeks Receives: Drug: High-dose CAN103 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Platelet count is measured in a central laboratory. An increase in platelet count indicates a therapeutic response.
Measured: 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.
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.
Measured: 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.
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.
Measured: Baseline to Week 39
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.
Measured: Baseline and Week 39
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2022
- Study start11 Jul 2022
- Primary completion30 Nov 2024 (estimated)
- Study completion30 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease, Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CANbridge (Suzhou) Bio-pharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bing Han, MD, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05447494
- Sponsor's protocol ID
- CAN103-GD-201
- Other IDs
- CTR20220507
- Status last verified
- Jul 2022
- First submitted
- 1 Jul 2022
- Last updated
- 27 Jul 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05447494
- Acronym / sponsor ID
- CAN103-GD-201
- Conditions
- Gaucher Disease, Type 1, Gaucher Disease, Type 3
- Keywords
- CAN103, Gaucher disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- CANbridge (Suzhou) Bio-pharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 11 Jul 2022 (actual)
- Primary completion
- 30 Nov 2024 (estimated)
- Study completion
- 30 Dec 2024 (estimated)
- First posted
- 7 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Low-dose CAN103 | Experimental | Drug: Low-dose CAN103Low dose intravenous infusion of CAN103 every other week for 37 weeks |
| High-dose CAN103 | Experimental | Drug: High-dose CAN103High dose intravenous infusion of CAN103 every other week for 37 weeks |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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:
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Low-dose CAN103Phase 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 | Low-dose CAN103 |
| High-dose CAN103Phase 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 |
Primary outcome
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.
4 secondary outcomes
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
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
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
Mean change in hemoglobin level from Baseline to Week 39 in the low-dose group.
Time frame: Baseline and Week 39
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Xiaogang Hui contact
Bing Han, MD principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Low-dose CAN103 and High-dose CAN103 in people with Gaucher Disease, Type 1 and Gaucher Disease, Type 3, run by CANbridge (Suzhou) Bio-pharma Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 27 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Qionghui Qiu (+86 21 52996609 ext. 807, qionghui.qiu@canbridgepharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean change in hemoglobin level from Baseline to Week 39 in the high-dose group (time frame: Baseline to Week 39). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2022.