Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

PHASE3CompletedINTERVENTIONAL
Enrollment

118

Participants

Timeline

Start Date

June 21, 2018

Primary Completion Date

August 17, 2021

Study Completion Date

September 2, 2021

Conditions
Fabry Disease
Interventions
DRUG

Lucerastat

Hard gelatin capsules containing 250 mg of lucerastat and inactive excipients; 1000 mg (4 capsules) twice daily (b.i.d.); dose adjusted for renal function.

DRUG

Placebo

Placebo capsules are identical in appearance to the lucerastat capsules, and contain inactive excipients; 4 capsules b.i.d.; dose adjusted for renal function.

Trial Locations (49)

1090

Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse, Vienna

3000

University Hospital Leuven, Leuven

3050

Royal Melbourne Hospital - Department of Nephrology, Parkville

5021

Haukeland University Hospital Helse Bergen HF, Bergen

6000

Royal Perth Hospital, Department of Nephrology, Perth

8032

Universität Zürich Psychiatrische Universitätsklinik, Zurich

9000

University Hospital Ghent, Ghent

13353

Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin, Berlin

15224

Children's Hospital of Pittsburgh (UPMC), Pittsburgh

19104

University of Pennsylvania, Philadelphia

20900

ASST Monza, Hospital San Gerardo, Nephrology, Monza

22030

Lysosomal and Rare Disorders Research and Treatment Center, Fairfax

22660

Hospital Academisch Medisch Centrum - Department of Internal Medicine Div. Endrocrinology and Metabolism, Amsterdam

27710

Duke University Medical Center, Durham

28034

Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna, Madrid

29605

Greenwood Genetic Center, Greenville

30322

Emory University School of Medicine; Department of Human Genetics, Atlanta

32610

University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation, Gainesville

35233

University of Alabama at Birmingham - Nephrology Research Clinic, Birmingham

49525

Infusion Associates, Grand Rapids

50012

Hospital Quironsalud Zaragoza, Zaragoza

52242

University of Iowa Stead Family Children's Hospital - Division of Medical Genetics, Iowa City

60612

Rush University Medical Center, Chicago

65239

SphinCS GmbH, Höchheim

75226

Research Baylor Institute of Metabolic Disease, Dallas

79379

Fachinternistische Gemeinschaftspraxis Markgräferland, Mühlheim

80131

University of Naples Federico II (Nephrology), Naples

84113

University of Utah - Division of Medical Genetics, Salt Lake City

92696

University of California Irvine, Irvine

94609

UCSF Benioff Children's Hospital Oakland, Oakland

97080

Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie, Würzburg

97239

Oregon Health and Science University, Portland

02114

Massachusetts General Hospital, Boston

N6A 5W9

London Health Sciences Centre - Victoria Hospital, London

T2M 0L6

M.A.G.I.C Clinic Ltd, Calgary

B3H 3A7

Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Nephrology, Halifax

H4J 1C5

Research Center, Hôpital Du Sacré-Coeur de Montréal, Montreal

V5Z 1M9

Vancouver Hospital & Health Sciences - Vancouver General Hospital, Vancouver

R3A 1S1

Health Sciences Center Winnipeg, Winnipeg

DD7 R2WY

Hosp Alma Mater Studiorum, Dublin

31-066

University Hospital in Cracow - Dep. of of Allergies and Immunology, Krakow

04-628

Cardinal Wyszynski Institute of Cardiology, Warsaw

04-730

Department of Pediatric Nutrition and Metabolic Diseases; The Children's Memorial Health Institute, Warsaw

08035

Hospital Universitari Vall d'Hebrón, Barcelona

08907

Hospital Universitari de Bellvitge; Hospitalet de Llobregat, Barcelona

B15 2WB

University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases, Birmingham

NW3 2QG

The Royal Free Hospital, Department of Haematology Royal Free London NHS Foundation Trust, London

WC1N3BG

National Hospital for Neurology and Neurosurgery, London

M6 8HD

Salford Royal (Hope) Hospital, Salford

Sponsors
All Listed Sponsors
lead

Idorsia Pharmaceuticals Ltd.

INDUSTRY

NCT03425539 - Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease | Biotech Hunter | Biotech Hunter