Clinical trial NCT04656600: Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Gaucher's Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2020-12-07
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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

Detailed description: Approximatively 14 months including a 12 months treatment period

Eligibility criteria: 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.

Primary outcomes:
measure: Changes in haemoglobin
timeFrame: Baseline to the end of 12 months
measure: Changes in platelet count
timeFrame: Baseline to the end of 12 months
measure: Adverse events
timeFrame: Baseline to the end of 13 months
Secondary outcomes:
measure: Changes in spleen volume
timeFrame: Baseline to the end of 12 months
measure: Changes in liver volume
timeFrame: Baseline to the end of 12 months
measure: Skeletal involvement
timeFrame: Baseline to the end of 12 months
measure: Quality of life (QoL)
timeFrame: Baseline to the end of 3 months, 6 months, 9 months and 12 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Investigational Site Number : 107
city: Beijing
country: China
facility: Investigational Site Number : 101
city: Chengdu
country: China
facility: Investigational Site Number : 104
city: Guangzhou
country: China
facility: Investigational Site Number : 102
city: Guangzhou
country: China
facility: Investigational Site Number : 105
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: