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 .