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

Sanofi

Status and phase

Completed

Phase 4

Conditions

Gaucher's Disease

Treatments

Drug: Cerezyme® / Imiglucerase

Study type

Interventional

Funder types

Industry

Identifiers

NCT04656600

U1111-1244-1166 (Other Identifier)

LPS16031

Details and patient eligibility

About

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

Full description

Approximatively 14 months including a 12 months treatment period

Enrollment

12 patients

Sex

All

Ages

2+ years old

Volunteers

No Healthy Volunteers

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