Overview

Eliglustat on Gaucher Disease Type IIIB

Status:
Unknown status
Trial end date:
2021-11-01
Target enrollment:
0
Participant gender:
All
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.
Phase:
N/A
Accepts Healthy Volunteers?
Accepts Healthy Volunteers
Details
Lead Sponsor:
National Taiwan University Hospital
Collaborator:
Sanofi
Treatments:
Eliglustat
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 .