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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-03-02

Study Completion Date

2023-10-12

Brief Summary

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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

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Detailed Description

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Approximatively 14 months including a 12 months treatment period

Conditions

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Gaucher's Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cerezyme® / Imiglucerase

Cerezyme® (imiglucerase for injection) is administered by intravenous infusion, 60 U/kg once every 2 weeks.

Group Type EXPERIMENTAL

Cerezyme® / Imiglucerase

Intervention Type DRUG

Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous

Interventions

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Cerezyme® / Imiglucerase

Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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.

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.

Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No