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

Studying an Investigational Medication for Gaucher Disease Type III

Not Recruiting
2 years or above
All
Phase 4
12 participants needed

Study Overview

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

Study Details

Approximatively 14 months including a 12 months treatment period

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Gaucher's Disease
  • Age: 2 years or above
  • Gender: All

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.

Updated on 23 Jan 2024. Study ID: NCT04656600

This study investigates the effects of an investigational medication on Gaucher Disease Type III in Chinese patients. Gaucher Disease Type III is a rare genetic disorder where fatty substances build up in certain organs, especially the spleen and liver, and can affect the brain. The purpose of this study is to evaluate how well the investigational medication works on blood-related symptoms and its safety when given at the highest dose recommended.

Participants will receive the investigational medication through an intravenous (IV) infusion every two weeks. The study will also assess its effects on organ size, bone health, and overall quality of life. Gaucher Disease Type III can lead to enlarged organs and bone issues, so the study aims to see if the medication can help manage these symptoms.

  • Who can participate: Participants must be over 2 years old and have Gaucher Disease Type III with neurological symptoms. They should have enlarged spleen or liver at screening and be able to give informed consent.
  • Study details: Participants will receive the investigational medication through an IV infusion every two weeks. A placebo is not used in this study. The study will monitor the effects on organ size, blood health, and quality of life.
  • Study timelines: The study will last 14 months.