Gaucher's DiseasePhase 4SEEDNCT04656600
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
A Phase 4 study testing Cerezyme® / Imiglucerase in people with Gaucher's Disease, run by Sanofi at 5 sites in China.
Trial summary
Primary Objective To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ To evaluate the safety profile of imiglucerase in maximum dose in the label (60U/kg, IV biweekly) in Chinese patients. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Gaucher's Disease
- What is being tested?
- Cerezyme® / Imiglucerase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 5 sitesChina
- Study dates
- 2 Mar 2021to 12 Oct 2023
- Phase
- Phase 4
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Capable of giving signed informed consent.
- Participant is diagnosed with GD type Ⅲ
- Participant with neurological manifestations
- Participant whose age is > 2 years old.
- Participant whose spleen and/or liver volume is > ULN at Screening.
Who cannot join
- Major congenital anomaly
- Clinically significant intercurrent organic disease unrelated to Gaucher disease, which means the disease or condition that may have impact on the parameters chosen for primary endpoints (e.g. level of hemoglobin platelets, liver/spleen enlargement and bone pains)
- Prior treatment with ERT.
- Physical conditions that cannot tolerate regular treatment or follow-up visit.
- Pregnant or lactating women
- Participant is participating in or has participated in another clinical study using any investigational therapy in 3 months
Show all 12Show fewer
- Participant has been diagnosed with central nervous system disease unrelated to Gaucher disease, or MRI result of the participant indicates space-occupying lesion in central nervous system
- The patient has a documented hemoglobinopathies, deficiency of iron, vitamin B-12, or folate that requires treatment not yet initiated or, if initiated, the patient has not been stable under treatment for at least 6 months prior to administration of the first dose of Cerezyme in this study
- Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
- Any specific situation during study implementation/course that may rise ethics considerations
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
China5 sites
Investigational Site Number : 107
Beijing, 100020
Investigational Site Number : 101
Beijing, 100032
Investigational Site Number : 104
Chengdu, 610041
Investigational Site Number : 102
Guangzhou, 510080
Investigational Site Number : 105
Guangzhou, 510623
About this study
Official title: A Single Arm, Prospective, Open Label, Multicenter Study to Evaluate Efficacy and Safety of One-year Maximum Dosage in Chinese Label of Imiglucerase Treatment in Chinese Patients Who Are Diagnosed as Gaucher Disease Type Ⅲ
Primary Objective
- To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the safety profile of imiglucerase in maximum dose in the label (60U/kg, IV biweekly) in Chinese patients.
Read more
Secondary Objective
- To evaluate the efficacy on viscera manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the efficacy on bone disease of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the effect on quality of life of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
Read the detailed description
Approximatively 14 months including a 12 months treatment period
- Conditions
- Gaucher's Disease
- Being tested
- Cerezyme® / Imiglucerase
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cerezyme® / ImigluceraseDrug
Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous
| Study group | Role | What they receive |
|---|---|---|
| Cerezyme® / Imiglucerase | Experimental | Cerezyme® (imiglucerase for injection) is administered by intravenous infusion, 60 U/kg once every 2 weeks. Receives: Drug: Cerezyme® / Imiglucerase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in haemoglobin
The mean changes in haemoglobin
Measured: Baseline to the end of 12 months
Changes in platelet count
The mean changes in platelet count
Measured: Baseline to the end of 12 months
Adverse events
Number of participants with AEs
Measured: Baseline to the end of 13 months
Secondary outcomes (4)
Changes in spleen volume
The mean change of spleen volumne
Measured: Baseline to the end of 12 months
Changes in liver volume
The mean change of liver volumne
Measured: Baseline to the end of 12 months
Skeletal involvement
The changes in frequency, duration and severity of bone pain and number of bone crisis The changes in frequency, duration and severity of bone pain and number of bone crisisThe changes in frequency, duration and severity of bone pain and number of bone crisis
Measured: Baseline to the end of 12 months
Quality of life (QoL)
The mean change of QoL
Measured: Baseline to the end of 3 months, 6 months, 9 months and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2020
- Study start2 Mar 2021
- Primary completion12 Oct 2023
- Study completion12 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher's DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04656600
- Sponsor's protocol ID
- LPS16031
- Other IDs
- U1111-1244-1166
- Acronym
- SEED
- Status last verified
- Sep 2025
- First submitted
- 4 Dec 2020
- Last updated
- 15 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Arm, Prospective, Open Label, Multicenter Study to Evaluate Efficacy and Safety of One-year Maximum Dosage in Chinese Label of Imiglucerase Treatment in Chinese Patients Who Are Diagnosed as Gaucher Disease Type Ⅲ
- ClinicalTrials.gov ID
- NCT04656600
- Acronym / sponsor ID
- SEED / LPS16031
- Conditions
- Gaucher's Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 2 Mar 2021 (actual)
- Primary completion
- 12 Oct 2023 (actual)
- Study completion
- 12 Oct 2023 (actual)
- First posted
- 7 Dec 2020 (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.
Primary Objective
- To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the safety profile of imiglucerase in maximum dose in the label (60U/kg, IV biweekly) in Chinese patients.
Secondary Objective
- To evaluate the efficacy on viscera manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the efficacy on bone disease of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the effect on quality of life of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
Detailed description
Approximatively 14 months including a 12 months treatment period
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cerezyme® / Imiglucerase | Experimental | Drug: Cerezyme® / ImigluceraseCerezyme® (imiglucerase for injection) is administered by intravenous infusion, 60 U/kg once every 2 weeks. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Capable of giving signed informed consent.
- Participant is diagnosed with GD type Ⅲ
- Participant with neurological manifestations
- Participant whose age is > 2 years old.
- Participant whose spleen and/or liver volume is > ULN at Screening.
Key exclusion criteria (12 listed)
- Major congenital anomaly
- Clinically significant intercurrent organic disease unrelated to Gaucher disease, which means the disease or condition that may have impact on the parameters chosen for primary endpoints (e.g. level of hemoglobin platelets, liver/spleen enlargement and bone pains)
- Prior treatment with ERT.
- Physical conditions that cannot tolerate regular treatment or follow-up visit.
- Pregnant or lactating women
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 |
|---|---|---|
| Cerezyme® / ImiglucerasePharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous | Drug | Cerezyme® / Imiglucerase |
Primary outcome
Changes in haemoglobin
Time frame: Baseline to the end of 12 months
The mean changes in haemoglobin
Changes in platelet count
Time frame: Baseline to the end of 12 months
The mean changes in platelet count
Adverse events
Time frame: Baseline to the end of 13 months
Number of participants with AEs
4 secondary outcomes
Changes in spleen volume
Time frame: Baseline to the end of 12 months
Changes in liver volume
Time frame: Baseline to the end of 12 months
Skeletal involvement
Time frame: Baseline to the end of 12 months
Quality of life (QoL)
Time frame: Baseline to the end of 3 months, 6 months, 9 months and 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2025.
- LPS16031 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
Clinical Sciences & Operations study director
Sanofi
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Cerezyme® / Imiglucerase in people with Gaucher's Disease, run by Sanofi at 5 sites 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 5 inclusion and 12 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: finished, as of the sponsor's last update on 15 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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 phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in haemoglobin (time frame: Baseline to the end of 12 months). The study lists 3 primary outcomes in all. 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 15 Sep 2025.