Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ

Gaucher's Disease Clinical Trial

— SEED  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04656600
• Other study ID #: LPS16031
• Secondary ID: U1111-1244-1166
• Status: Completed
• Phase: Phase 4
• Start date: March 2, 2021
• Est. completion date: October 12, 2023

Study information

• Verified date: January 2024
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

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

Description:

Approximatively 14 months including a 12 months treatment period

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: October 12, 2023
• Est. primary completion date: October 12, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

Inclusion criteria:

• Capable of giving signed informed consent.

• Participant is diagnosed with GD type ?

• Participant with neurological manifestations

• Participant whose age is > 2 years old.

• Participant whose spleen and/or liver volume is > ULN at Screening.

Exclusion criteria:

• 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

• 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.

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher's Disease

Intervention

Drug:
• Cerezyme® / Imiglucerase
Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous

Locations

Country	Name	City	State
China	Investigational Site Number : 101	Beijing
China	Investigational Site Number : 107	Beijing
China	Investigational Site Number : 104	Chengdu
China	Investigational Site Number : 102	Guangzhou
China	Investigational Site Number : 105	Guangzhou

Sponsors (1)

Lead Sponsor	Collaborator
Sanofi

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Changes in haemoglobin	The mean changes in haemoglobin	Baseline to the end of 12 months
Primary	Changes in platelet count	The mean changes in platelet count	Baseline to the end of 12 months
Primary	Adverse events	Number of participants with AEs	Baseline to the end of 13 months
Secondary	Changes in spleen volume	The mean change of spleen volumne	Baseline to the end of 12 months
Secondary	Changes in liver volume	The mean change of liver volumne	Baseline to the end of 12 months
Secondary	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	Baseline to the end of 12 months
Secondary	Quality of life (QoL)	The mean change of QoL	Baseline to the end of 3 months, 6 months, 9 months and 12 months