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

Primary Purpose

Gaucher's Disease

Status

Active

Phase

Phase 4

Locations

China

Study Type

Interventional

Intervention

Cerezyme® / Imiglucerase

About this trial

This is an interventional treatment trial for Gaucher's Disease

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sites / Locations

Investigational Site Number :107
Investigational Site Number :101
Investigational Site Number :104
Investigational Site Number :102
Investigational Site Number :105

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Cerezyme® / Imiglucerase

Arm Description

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

Outcomes

Primary Outcome Measures

Changes in haemoglobin

The mean changes in haemoglobin

Changes in platelet count

The mean changes in platelet count

Adverse events

Number of participants with AEs

Secondary Outcome Measures

Changes in spleen volume

The mean change of spleen volumne

Changes in liver volume

The mean change of liver volumne

Skeletal involvement

The changes in frequency, duration and severity of bone pain and number of bone crisis The changes in frequency, duration and severity of bone pain and number of bone crisisThe changes in frequency, duration and severity of bone pain and number of bone crisis

Quality of life (QoL)

The mean change of QoL

Full Information

NCT ID

NCT04656600

First Posted

December 4, 2020

Last Updated

April 4, 2023

Sponsor

Sanofi

1. Study Identification

Unique Protocol Identification Number

NCT04656600

Brief Title

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

Acronym

SEED

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

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

March 2, 2021 (Actual)

Primary Completion Date

October 18, 2023 (Anticipated)

Study Completion Date

October 18, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Sanofi

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Yes

Data Monitoring Committee

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Gaucher's Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cerezyme® / Imiglucerase

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Cerezyme® / Imiglucerase

Intervention Description

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

Primary Outcome Measure Information:

Title

Changes in haemoglobin

Description

The mean changes in haemoglobin

Time Frame

Baseline to the end of 12 months

Title

Changes in platelet count

Description

The mean changes in platelet count

Time Frame

Baseline to the end of 12 months

Title

Adverse events

Description

Number of participants with AEs

Time Frame

Baseline to the end of 13 months

Secondary Outcome Measure Information:

Title

Changes in spleen volume

Description

The mean change of spleen volumne

Time Frame

Baseline to the end of 12 months

Title

Changes in liver volume

Description

The mean change of liver volumne

Time Frame

Baseline to the end of 12 months

Title

Skeletal involvement

Description

Time Frame

Baseline to the end of 12 months

Title

Quality of life (QoL)

Description

The mean change of QoL

Time Frame

Baseline to the end of 3 months, 6 months, 9 months and 12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Sciences & Operations

Organizational Affiliation

Sanofi

Official's Role

Study Director

Facility Information:

Facility Name

Investigational Site Number :107

City

Beijing

ZIP/Postal Code

100020

Country

China

Facility Name

Investigational Site Number :101

City

Beijing

ZIP/Postal Code

100730

Country

China

Facility Name

Investigational Site Number :104

City

Chengdu

ZIP/Postal Code

610041

Country

China

Facility Name

Investigational Site Number :102

City

Guangzhou

ZIP/Postal Code

510080

Country

China

Facility Name

Investigational Site Number :105

City

Guangzhou

ZIP/Postal Code

510623

Country

China

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Learn more about this trial

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

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