Eliglustat on Gaucher Disease Type IIIB (NCT03519646) | Clinical Trial Compass
CompletedNot Applicable
Eliglustat on Gaucher Disease Type IIIB
Taiwan4 participantsStarted 2018-04-23
Plain-language 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.
Who can participate
Age range6 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 .
What they're measuring
1
Adverse Events
Timeframe: From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.