Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
NA
4 participants
INTERVENTIONAL
2018-04-23
2020-09-11
Brief Summary
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Detailed Description
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* 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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Experimental Case_Eiglustat
Besides regular ERT, patients also need to take Eiglustat for 24 months.
Eliglustat
1. This is a 3-year study and the enrollment time of this study is 24 months.
2. The participants have to receive the investigational agent, Cerdelga
3. 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.
4. IMP Administration Method: Cerdelga have 21 mg、42 mg and 84 mg capsule.
5. No need for fasting before use, but can't take with grapefruit juice.
6. 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.
Interventions
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Eliglustat
1. This is a 3-year study and the enrollment time of this study is 24 months.
2. The participants have to receive the investigational agent, Cerdelga
3. 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.
4. IMP Administration Method: Cerdelga have 21 mg、42 mg and 84 mg capsule.
5. No need for fasting before use, but can't take with grapefruit juice.
6. 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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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 .
6 Years
ALL
Yes
Sponsors
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Sanofi
INDUSTRY
National Taiwan University Hospital
OTHER
Responsible Party
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Principal Investigators
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Ni-Chung Lee, M.D., Ph.D
Role: PRINCIPAL_INVESTIGATOR
National Taiwan University Hospital
Locations
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National Taiwan University Hospital
Taipei, , Taiwan
Countries
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Other Identifiers
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201612250MIPB
Identifier Type: -
Identifier Source: org_study_id
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