Clinical trial NCT03425539: Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Official title: A Multicenter, dOuble-blind, ranDomized, Placebo-controlled, Parallel-group Study to Determine the effIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With FabrY Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Fabry Disease
Lead sponsor: Idorsia Pharmaceuticals Ltd.
Sponsor class: INDUSTRY
First posted: 2018-02-07
Enrollment: 118
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

1. Signed and dated ICF prior to any study-mandated procedure;
2. Male or female adult subjects;
3. FD diagnosis confirmed with local genetic test results;
4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
5. Enzyme replacement therapy (ERT) status:

   1. Subject never treated with ERT; or
   2. Subject has not received ERT for at least 6 months prior to screening; or
   3. 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.
6. A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
7. Subjects with moderate or severe neuropathic pain during the screening period.

Exclusion Criteria:

1. Pregnant, planning to be become pregnant, or lactating subject.
2. Severe renal insufficiency (eGFR \< 30 mL/min/1.73 m2) at screening.
3. Subject on regular dialysis for the treatment of chronic kidney disease.
4. Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
5. Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
6. Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.

Primary outcomes:
measure: Neuropathic Pain Monthly Score: Change From Baseline to Month 6
timeFrame: From baseline to Month 6 (duration: 6 months)
Secondary outcomes:
measure: Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6
timeFrame: From baseline to Month 6 (duration: 6 months)
measure: Abdominal Pain Monthly Score: Change From Baseline to Month 6
timeFrame: From baseline to Month 6 (duration: 6 months)
measure: Number of Days With Diarrhea: Change From Baseline to Month 6
timeFrame: From baseline to Month 6 (duration: 6 months)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham - Nephrology Research Clinic
state: Alabama
city: Irvine
country: United States
facility: University of California Irvine
state: California
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Gainesville
country: United States
facility: University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine; Department of Human Genetics
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
state: Iowa
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Grand Rapids
country: United States
facility: Infusion Associates
state: Michigan
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh (UPMC)
state: Pennsylvania
city: Greenville
country: United States
facility: Greenwood Genetic Center
state: South Carolina
city: Dallas
country: United States
facility: Research Baylor Institute of Metabolic Disease
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah - Division of Medical Genetics
state: Utah
city: Fairfax
country: United States
facility: Lysosomal and Rare Disorders Research and Treatment Center
state: Virginia
city: Parkville
country: Australia
facility: Royal Melbourne Hospital - Department of Nephrology
city: Perth
country: Australia
facility: Royal Perth Hospital, Department of Nephrology
city: Vienna
country: Austria
facility: Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse
city: Ghent
country: Belgium
facility: University Hospital Ghent
city: Leuven
country: Belgium
facility: University Hospital Leuven
city: London
country: Canada
facility: London Health Sciences Centre - Victoria Hospital
state: Ontario
city: Calgary
country: Canada
facility: M.A.G.I.C Clinic Ltd
city: Halifax
country: Canada
facility: Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Nephrology
city: Montréal
country: Canada
facility: Research Center, Hôpital Du Sacré-Coeur de Montréal
city: Vancouver
country: Canada
facility: Vancouver Hospital & Health Sciences - Vancouver General Hospital
city: Winnipeg
country: Canada
facility: Health Sciences Center Winnipeg
city: Berlin
country: Germany
facility: Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin
city: Hochheim
country: Germany
facility: SphinCS GmbH
city: Mühlheim
country: Germany
facility: Fachinternistische Gemeinschaftspraxis Markgräferland
city: Würzburg
country: Germany
facility: Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie
city: Dublin
country: Ireland
facility: Hosp Alma Mater Studiorum
city: Monza
country: Italy
facility: ASST Monza, Hospital San Gerardo, Nephrology
city: Naples
country: Italy
facility: University of Naples Federico II (Nephrology)
city: Amsterdam
country: Netherlands
facility: Hospital Academisch Medisch Centrum - Department of Internal Medicine Div. Endrocrinology and Metabolism
city: Bergen
country: Norway
facility: Haukeland University Hospital Helse Bergen HF
city: Krakow
country: Poland
facility: University Hospital in Cracow - Dep. of of Allergies and Immunology
city: Warsaw
country: Poland
facility: Cardinal Wyszynski Institute of Cardiology
city: Warsaw
country: Poland
facility: Department of Pediatric Nutrition and Metabolic Diseases; The Children's Memorial Health Institute
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebrón
city: Barcelona
country: Spain
facility: Hospital Universitari de Bellvitge; Hospitalet de Llobregat
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
city: Zaragoza
country: Spain
facility: Hospital Quironsalud Zaragoza
city: Zurich
country: Switzerland
facility: Universität Zürich Psychiatrische Universitätsklinik
city: Birmingham
country: United Kingdom
facility: University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases
city: London
country: United Kingdom
facility: The Royal Free Hospital, Department of Haematology Royal Free London NHS Foundation Trust
city: London
country: United Kingdom
facility: National Hospital for Neurology and Neurosurgery
city: Salford
country: United Kingdom
facility: Salford Royal (Hope) Hospital
Study officials:
affiliation: Idorsia Pharmaceuticals Ltd.
name: Clinical Trials
role: STUDY_DIRECTOR
References: