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Eliglustat on Gaucher Disease Type IIIB

NCT03519646

CompletedNA

Sponsor: National Taiwan University Hospital

Conditions: Gaucher Disease, Type III

Interventions: Eliglustat

Countries: Taiwan

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.

Eligibility overview

Sex: ALL

Age: 6 Years to —

Healthy volunteers: Yes

Study type: INTERVENTIONAL

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 .

Locations (1)

Taipei, Taiwan