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Eliglustat on Gaucher Disease Type IIIB

Primary Purpose

Gaucher Disease, Type III

Status
Completed
Phase
Not Applicable
Locations
Taiwan
Study Type
Interventional
Intervention
Eliglustat
Sponsored by
National Taiwan University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gaucher Disease, Type III focused on measuring Enzyme Replacement Therapy, Eliglustat (Cerdelga®)

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

  • Gaucher disease patients diagnosed by low B-glucocerebrosidase deficiency and GBA mutation .
  • The participant is at least 6 years old at time of enrollment.
  • Under stable Cerezyme dosage for at least for 3 months.
  • Presence of lymphadenopathy.
  • Patient (and/or their parent/legal guardian) is willing to participate and able to provide signed informed consent.

Exclusion Criteria:

  • The participant is CYP2D6 ultra-rapid metabolizer.
  • The participant had received substrate reduction therapy for Gaucher disease within 3 months of enrollment.
  • The participant had any clinically significant disease other than GD, including cardiovascular (especially arrhythmia), renal, liver, pulmonary, endocrinopathy, hypokalemia, or hypomagnesemia that may confound the study result.
  • The participant is pregnant or lactating.
  • The participant is known to be allergy to Cerdelga.
  • The participant use drugs that will strongly inhibit CYP2D6 or CYP3A activity .

Sites / Locations

  • National Taiwan University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Experimental Case_Eiglustat

Arm Description

Besides regular ERT, patients also need to take Eiglustat for 24 months.

Outcomes

Primary Outcome Measures

Adverse Events
Number of adverse events in patients.

Secondary Outcome Measures

Assessment of Gaucher related biomarkers test :CCL18 (30% decrease)
Measure of Gaucher disease type I biomarkers:CCL18(ng/ml) in plasma.
Assessment of Gaucher related biomarkers test :Lyso GL1(30% decrease)
Measure of Gaucher disease type I biomarkers:Lyso GL1 (ng/mL) in plasma
Assessment of Gaucher related biomarkers test:Chitotriosidase(30% decrease)
Measure of Gaucher disease type I biomarkers:Chitotriosidase(nmol/ml/h) in plasma.
Change in lymphadenopathy manifestations.
Physician will pay close attention to the lymphadenopathy including size, location, and number of enlarged lymph nodes, evaluate by palpation and radiological (MRI examination). The total size of the lympadenopathy will be combined into one report as "Total size" cm^3.
Pharmacokinetics
Eliglustat plasma concentration over time

Full Information

First Posted
March 20, 2018
Last Updated
September 13, 2022
Sponsor
National Taiwan University Hospital
Collaborators
Sanofi
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1. Study Identification

Unique Protocol Identification Number
NCT03519646
Brief Title
Eliglustat on Gaucher Disease Type IIIB
Official Title
Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.
Study Type
Interventional

2. Study Status

Record Verification Date
January 2021
Overall Recruitment Status
Completed
Study Start Date
April 23, 2018 (Actual)
Primary Completion Date
September 11, 2020 (Actual)
Study Completion Date
September 11, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Taiwan University Hospital
Collaborators
Sanofi

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No

5. Study Description

Brief Summary
Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases.
Detailed Description
This is a 3-year study and the enrollment time of this study is 24 months. The participants have to receive the investigational agent:Cerdelga.(Cerdelga have 21 mg、42 mg and 84 mg capsule.) The participants have to go back to the hospital and receive the investigational agent and take the Gaucher related biomarkers test before receiving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga. The participants have to inform if any adverse events happened. The investigators will follow up by phone if adverse events happened in the participants after one months start the treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease, Type III
Keywords
Enzyme Replacement Therapy, Eliglustat (Cerdelga®)

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Experimental Case_Eiglustat
Arm Type
Experimental
Arm Description
Besides regular ERT, patients also need to take Eiglustat for 24 months.
Intervention Type
Drug
Intervention Name(s)
Eliglustat
Other Intervention Name(s)
Cerdelga
Intervention Description
This is a 3-year study and the enrollment time of this study is 24 months. The participants have to receive the investigational agent, Cerdelga The participants have to go back to the hospital and receive the investigational agent and take the test before receving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga. IMP Administration Method: Cerdelga have 21 mg、42 mg and 84 mg capsule. No need for fasting before use, but can't take with grapefruit juice. Five ml Blood and 10 ml urine shoud be taken before receiving Cerdelga, and 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga for Gaucher related biomarkers test.
Primary Outcome Measure Information:
Title
Adverse Events
Description
Number of adverse events in patients.
Time Frame
From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Secondary Outcome Measure Information:
Title
Assessment of Gaucher related biomarkers test :CCL18 (30% decrease)
Description
Measure of Gaucher disease type I biomarkers:CCL18(ng/ml) in plasma.
Time Frame
Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Title
Assessment of Gaucher related biomarkers test :Lyso GL1(30% decrease)
Description
Measure of Gaucher disease type I biomarkers:Lyso GL1 (ng/mL) in plasma
Time Frame
Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Title
Assessment of Gaucher related biomarkers test:Chitotriosidase(30% decrease)
Description
Measure of Gaucher disease type I biomarkers:Chitotriosidase(nmol/ml/h) in plasma.
Time Frame
Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Title
Change in lymphadenopathy manifestations.
Description
Physician will pay close attention to the lymphadenopathy including size, location, and number of enlarged lymph nodes, evaluate by palpation and radiological (MRI examination). The total size of the lympadenopathy will be combined into one report as "Total size" cm^3.
Time Frame
From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Title
Pharmacokinetics
Description
Eliglustat plasma concentration over time
Time Frame
Plasma concentration-time data will be obtained pre-dose (within 30 minutes prior to dosing) and at 1, 2, 6, 12, 26, 36 hours after 1st dosing, and 1,3,6,12,18 and 24 months thereafter.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria: Gaucher disease patients diagnosed by low B-glucocerebrosidase deficiency and GBA mutation . The participant is at least 6 years old at time of enrollment. Under stable Cerezyme dosage for at least for 3 months. Presence of lymphadenopathy. Patient (and/or their parent/legal guardian) is willing to participate and able to provide signed informed consent. Exclusion Criteria: The participant is CYP2D6 ultra-rapid metabolizer. The participant had received substrate reduction therapy for Gaucher disease within 3 months of enrollment. The participant had any clinically significant disease other than GD, including cardiovascular (especially arrhythmia), renal, liver, pulmonary, endocrinopathy, hypokalemia, or hypomagnesemia that may confound the study result. The participant is pregnant or lactating. The participant is known to be allergy to Cerdelga. The participant use drugs that will strongly inhibit CYP2D6 or CYP3A activity .
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ni-Chung Lee, M.D., Ph.D
Organizational Affiliation
National Taiwan University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
10041
Country
Taiwan

12. IPD Sharing Statement

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Eliglustat on Gaucher Disease Type IIIB

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