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

  • STATUS
    Recruiting
  • End date
    Sep 13, 2023
  • participants needed
    12
  • sponsor
    Sanofi
Updated on 13 May 2022
imiglucerase
neuronopathic gaucher disease
cerezyme

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

Details
Condition Gaucher's Disease
Treatment Cerezyme® / Imiglucerase
Clinical Study IdentifierNCT04656600
SponsorSanofi
Last Modified on13 May 2022

Eligibility

Yes No Not Sure

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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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