Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
- Registration Number
- NCT03425539
- Lead Sponsor
- Idorsia Pharmaceuticals Ltd.
- 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 neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 118
-
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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Lucerastat Lucerastat - Placebo Placebo -
- Primary Outcome Measures
Name Time Method Neuropathic Pain Monthly Score: Change From Baseline to Month 6 From baseline to Month 6 (duration: 6 months) Neuropathic pain on the modified BPI-SF3: subjects rated their neuropathic pain intensity ("neuropathic pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no neuropathic pain) to 10 (worst imaginable neuropathic pain).
- Secondary Outcome Measures
Name Time Method Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6 From baseline to Month 6 (duration: 6 months) Abdominal Pain Monthly Score: Change From Baseline to Month 6 From baseline to Month 6 (duration: 6 months) Abdominal pain on the 11-point Numerical Rating Scale (NRS-11): subjects rated their abdominal pain intensity ("abdominal pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no pain) to 10 (worst imaginable pain).
Number of Days With Diarrhea: Change From Baseline to Month 6 From baseline to Month 6 (duration: 6 months) A subject was considered to have diarrhea on a specific day if at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 was reported. The number of days with diarrhea at baseline and Month 6 was the number of days with diarrhea over the 4 weeks prior to the randomization visit or the Month 6 visit, respectively, adjusted for the number of days with data available.
Trial Locations
- Locations (49)
Emory University School of Medicine; Department of Human Genetics
🇺🇸Atlanta, Georgia, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Research Baylor Institute of Metabolic Disease
🇺🇸Dallas, Texas, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
University of Alabama at Birmingham - Nephrology Research Clinic
🇺🇸Birmingham, Alabama, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Infusion Associates
🇺🇸Grand Rapids, Michigan, United States
Royal Melbourne Hospital - Department of Nephrology
🇦🇺Parkville, Australia
Royal Perth Hospital, Department of Nephrology
🇦🇺Perth, Australia
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
🇺🇸Iowa City, Iowa, United States
University Hospital Leuven
🇧🇪Leuven, Belgium
M.A.G.I.C Clinic Ltd
🇨🇦Calgary, Canada
SphinCS GmbH
🇩🇪Hochheim, Germany
Fachinternistische Gemeinschaftspraxis Markgräferland
🇩🇪Mühlheim, Germany
Research Center, Hôpital Du Sacré-Coeur de Montréal
🇨🇦Montréal, Canada
Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin
🇩🇪Berlin, Germany
Vancouver Hospital & Health Sciences - Vancouver General Hospital
🇨🇦Vancouver, Canada
Hospital Universitari Vall d'Hebrón
🇪🇸Barcelona, Spain
ASST Monza, Hospital San Gerardo, Nephrology
🇮🇹Monza, Italy
Health Sciences Center Winnipeg
🇨🇦Winnipeg, Canada
Hospital Universitari de Bellvitge; Hospitalet de Llobregat
🇪🇸Barcelona, Spain
Hospital Quironsalud Zaragoza
🇪🇸Zaragoza, Spain
University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases
🇬🇧Birmingham, United Kingdom
University of Utah - Division of Medical Genetics
🇺🇸Salt Lake City, Utah, United States
Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie
🇩🇪Würzburg, Germany
Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
🇪🇸Madrid, Spain
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
Lysosomal and Rare Disorders Research and Treatment Center
🇺🇸Fairfax, Virginia, United States
University of California Irvine
🇺🇸Irvine, California, United States
Children's Hospital of Pittsburgh (UPMC)
🇺🇸Pittsburgh, Pennsylvania, United States
London Health Sciences Centre - Victoria Hospital
🇨🇦London, Ontario, Canada
Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Nephrology
🇨🇦Halifax, Canada
University of Naples Federico II (Nephrology)
🇮🇹Naples, Italy
National Hospital for Neurology and Neurosurgery
🇬🇧London, United Kingdom
The Royal Free Hospital, Department of Haematology Royal Free London NHS Foundation Trust
🇬🇧London, United Kingdom
Salford Royal (Hope) Hospital
🇬🇧Salford, United Kingdom
Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse
🇦🇹Vienna, Austria
University Hospital Ghent
🇧🇪Ghent, Belgium
Hosp Alma Mater Studiorum
🇮🇪Dublin, Ireland
Greenwood Genetic Center
🇺🇸Greenville, South Carolina, United States
Cardinal Wyszynski Institute of Cardiology
🇵🇱Warsaw, Poland
Haukeland University Hospital Helse Bergen HF
🇳🇴Bergen, Norway
University Hospital in Cracow - Dep. of of Allergies and Immunology
🇵🇱Krakow, Poland
Hospital Academisch Medisch Centrum - Department of Internal Medicine Div. Endrocrinology and Metabolism
🇳🇱Amsterdam, Netherlands
Universität Zürich Psychiatrische Universitätsklinik
🇨🇭Zurich, Switzerland
Department of Pediatric Nutrition and Metabolic Diseases; The Children's Memorial Health Institute
🇵🇱Warsaw, Poland
University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation
🇺🇸Gainesville, Florida, United States