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 range
6 Years
Sex
ALL
See this in plain English?
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 .
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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.