Fabry disease MedDRA version: 20.0 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Idorsia Pharmaceuticals Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Signed and dated ICF prior to any study-mandated procedure. 2. Male or female subjects; 18-years old and above. 3. FD diagnosis confirmed with local genetic test results (i.e., presence of at least 1 mutation in GLA, the gene coding for a-galactosidase A) 4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 89 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: 1. Pregnant, planning to be become pregnant up to 30 days after study treatment discontinuation or lactating subject; 2. Severe renal insufficiency defined as an estimated glomerular filtration rate (eGFR) per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 30 mL/min/1.73 m2 at screening; 3. Subject on regular dialysis for the treatment of chronic kidney disease; 4. Subject has undergone, or is on a waiting list for, or is scheduled to undergo kidney or other organ transplantation; 5. Known and documented transient ischemic attack, stroke, unstable angina or myocardial infarction within 6 months prior to screening; 6. Clinically significant unstable cardiac disease in the opinion of the investigator (e.g., uncontrolled symptomatic arrhythmia, New York Heart Association class III or IV congestive heart failure); 7. Any other subject at high risk for developing clinical signs of organ involvement within the time period of the study, as per investigator judgment; 8. Subject planned for imminent initiation of treatment with ERT.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To determine the effect of lucerastat on neuropathic pain in subjects with Fabry disease (FD).;Primary end point(s): The primary efficacy endpoint is change from baseline to Month 6 in the "modified" Brief Pain Inventory-Short Form item 3 (BPI-SF3) score of "neuropathic pain at its worst in the last 24 hours".;Timepoint(s) of evaluation of this end point: From baseline to Month 6;Secondary Objective: • To determine the effects of lucerastat on gastro-intestinal (GI) symptoms (abdominal pain and diarrhea) in subjects with FD and GI symptom(s) at baseline. • To confirm the effect of lucerastat on biomarkers of FD. • To determine the safety and tolerability of lucerastat in subjects with FD. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change from baseline to Month 6 in the 11-point Numerical Rating Scale (NRS-11) score of “abdominal pain at its worst in the last 24 hours” in subjects with GI symptoms at baseline. • Change from baseline to Month 6 in the number of days with at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 in subjects with GI symptoms at baseline. • Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3). ;Timepoint(s) of evaluation of this end point: From baseline to Month 6 | — |
Countries
Australia, Austria, Belgium, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Spain, Switzerland, United Kingdom, United States
Contacts
Public ContactClinical Trial Disclosure Desk
Idorsia Pharmaceuticals Ltd
Outcome results
None listed