A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fa… (NCT03737214) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
United States, Australia, Austria107 participantsStarted 2018-12-18
Plain-language summary
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.
Who can participate
Age range
18 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:
* Signed ICF prior to any study-mandated procedure;
* Subject completed the 6-month, double-blind treatment period in study ID 069A301
* Woman of childbearing potential only if agreement 1) to follow a specified contraception scheme, 2) to undertake monthly urine pregnancy tests.
* Fertile male only if agreement 1) to use a condom, 2) to not father a child.
Exclusion Criteria:
* Pregnant / planning to be become pregnant or lactating subject;
* Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
* Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.
In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:
* Subject's eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation \< 15 mL/min/1.73 m2;
* Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
* Subject experienced an event of stroke CTCAE grade 3 or above;
* Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.
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
Treatment-emergent adverse events (AEs)
Timeframe: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up