A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease
- Conditions
- Fabry diseaseMedDRA version: 20.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2017-003369-85-GB
- Lead Sponsor
- Idorsia Pharmaceuticals Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 99
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
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: • To determine the effect of lucerastat on neuropathic pain in subjects with Fabry disease (FD).;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.<br>• To confirm the effect of lucerastat on biomarkers of FD.<br>• To determine the safety and tolerability of lucerastat in subjects with FD.<br>;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 Outcome Measures
Name Time Method 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.<br>• 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.<br>• Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3).<br>;Timepoint(s) of evaluation of this end point: From baseline to Month 6