A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
- Registration Number
- NCT03737214
- Lead Sponsor
- Idorsia Pharmaceuticals Ltd.
- Brief 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.
- Detailed Description
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest.
Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 107
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Lucerastat Lucerastat Dose will be based on subject's eGFR.
- Primary Outcome Measures
Name Time Method Treatment-emergent adverse events (AEs) From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (42)
University of Alabama at Birmingham - Nephrology Research Clinic
🇺🇸Birmingham, Alabama, United States
University of California Irvine
🇺🇸Irvine, California, United States
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
University of Florida Clinical and Translational Science Institute, UF Clinical Research Center
🇺🇸Gainesville, Florida, United States
Rush University Medical Center - Dept of Pediatrics
🇺🇸Chicago, Illinois, United States
University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
🇺🇸Iowa City, Iowa, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Infusion Associates
🇺🇸Grand Rapids, Michigan, United States
University of Pennsylvania - Dept of Medicine
🇺🇸Philadelphia, Pennsylvania, United States
Greenwood Genetics Center
🇺🇸Greenville, South Carolina, United States
Scroll for more (32 remaining)University of Alabama at Birmingham - Nephrology Research Clinic🇺🇸Birmingham, Alabama, United States