Status:
COMPLETED
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Lead Sponsor:
Idorsia Pharmaceuticals Ltd.
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
Detailed Description
The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropa...
Eligibility Criteria
Inclusion
- Signed and dated ICF prior to any study-mandated procedure;
- Male or female adult subjects;
- FD diagnosis confirmed with local genetic test results;
- Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
- Enzyme replacement therapy (ERT) status:
- Subject never treated with ERT; or
- Subject has not received ERT for at least 6 months prior to screening; or
- Subject treated with ERT since at least 12 months at the time of the screening visit, and agreeing to stop ERT for approximately 8 months.
- A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
- Subjects with moderate or severe neuropathic pain during the screening period.
Exclusion
- Pregnant, planning to be become pregnant, or lactating subject.
- Severe renal insufficiency (eGFR \< 30 mL/min/1.73 m2) at screening.
- Subject on regular dialysis for the treatment of chronic kidney disease.
- Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
- Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
- Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.
Key Trial Info
Start Date :
June 21 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 2 2021
Estimated Enrollment :
118 Patients enrolled
Trial Details
Trial ID
NCT03425539
Start Date
June 21 2018
End Date
September 2 2021
Last Update
August 9 2024
Active Locations (49)
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1
University of Alabama at Birmingham - Nephrology Research Clinic
Birmingham, Alabama, United States, 35233
2
University of California Irvine
Irvine, California, United States, 92696
3
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
4
University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation
Gainesville, Florida, United States, 32610