Eliglustat on Gaucher Disease Type IIIB

Gaucher Disease, Type III Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03519646
• Other study ID #: 201612250MIPB
• Status: Completed
• Phase: N/A
• Start date: April 23, 2018
• Est. completion date: September 11, 2020

Study information

• Verified date: January 2021
• Source: National Taiwan University Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: September 11, 2020
• Est. primary completion date: September 11, 2020
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 6 Years and older

Eligibility

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 .

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease, Type III

Intervention

Drug:
• Eliglustat
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.

Locations

Country	Name	City	State
Taiwan	National Taiwan University Hospital	Taipei

Sponsors (2)

Lead Sponsor	Collaborator
National Taiwan University Hospital	Sanofi

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Number of adverse events in patients.	From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Secondary	Assessment of Gaucher related biomarkers test :CCL18 (30% decrease)	Measure of Gaucher disease type I biomarkers:CCL18(ng/ml) in plasma.	Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Secondary	Assessment of Gaucher related biomarkers test :Lyso GL1(30% decrease)	Measure of Gaucher disease type I biomarkers:Lyso GL1 (ng/mL) in plasma	Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Secondary	Assessment of Gaucher related biomarkers test:Chitotriosidase(30% decrease)	Measure of Gaucher disease type I biomarkers:Chitotriosidase(nmol/ml/h) in plasma.	Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Secondary	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.	From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Secondary	Pharmacokinetics	Eliglustat plasma concentration over time	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.