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

NCT03425539 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: ID-069A301
• Study Type: interventional
• Target: 118
• Locations: 13 countries
• Sites: 47

Brief Summary

This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• Neuropathic Pain Monthly Score: Change From Baseline to Month 6

  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).

  From baseline to Month 6 (duration: 6 months)

Secondary Outcomes (3)

• 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)

• Number of Days With Diarrhea: Change From Baseline to Month 6

  From baseline to Month 6 (duration: 6 months)

Study Arms (2)

Lucerastat

EXPERIMENTAL

Drug: Lucerastat

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Lucerastat DRUG

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.

Lucerastat

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Idorsia Pharmaceuticals Ltd.: lead

Study Sites (49)

University of Alabama at Birmingham - Nephrology Research Clinic

Birmingham, Alabama, 35233, United States

Location

University of California Irvine

Irvine, California, 92696, United States

Location

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

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

Gainesville, Florida, 32610, United States

Location

Emory University School of Medicine; Department of Human Genetics

Atlanta, Georgia, 30322, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

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

Iowa City, Iowa, 52242, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Infusion Associates

Grand Rapids, Michigan, 49525, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh (UPMC)

Pittsburgh, Pennsylvania, 15224, United States

Location

Greenwood Genetic Center

Greenville, South Carolina, 29605, United States

Location

Research Baylor Institute of Metabolic Disease

Dallas, Texas, 75226, United States

Location

University of Utah - Division of Medical Genetics

Salt Lake City, Utah, 84113, United States

Location

Lysosomal and Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030, United States

Location

Royal Melbourne Hospital - Department of Nephrology

Parkville, 3050, Australia

Location

Royal Perth Hospital, Department of Nephrology

Perth, 6000, Australia

Location

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

Vienna, 1090, Austria

Location

University Hospital Ghent

Ghent, 9000, Belgium

Location

University Hospital Leuven

Leuven, 3000, Belgium

Location

London Health Sciences Centre - Victoria Hospital

London, Ontario, N6A 5W9, Canada

Location

M.A.G.I.C Clinic Ltd

Calgary, T2M 0L6, Canada

Location

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

Halifax, B3H 3A7, Canada

Location

Research Center, Hôpital Du Sacré-Coeur de Montréal

Montreal, H4J 1C5, Canada

Location

Vancouver Hospital & Health Sciences - Vancouver General Hospital

Vancouver, V5Z 1M9, Canada

Location

Health Sciences Center Winnipeg

Winnipeg, R3A 1S1, Canada

Location

Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin

Berlin, 13353, Germany

Location

SphinCS GmbH

Höchheim, 65239, Germany

Location

Fachinternistische Gemeinschaftspraxis Markgräferland

Mühlheim, 79379, Germany

Location

Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie

Würzburg, 97080, Germany

Location

Hosp Alma Mater Studiorum

Dublin, DD7 R2WY, Ireland

Location

ASST Monza, Hospital San Gerardo, Nephrology

Monza, 20900, Italy

Location

University of Naples Federico II (Nephrology)

Naples, 80131, Italy

Location

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

Amsterdam, 22660, Netherlands

Location

Haukeland University Hospital Helse Bergen HF

Bergen, 5021, Norway

Location

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

Krakow, 31-066, Poland

Location

Cardinal Wyszynski Institute of Cardiology

Warsaw, 04-628, Poland

Location

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

Warsaw, 04-730, Poland

Location

Hospital Universitari Vall d'Hebrón

Barcelona, 08035, Spain

Location

Hospital Universitari de Bellvitge; Hospitalet de Llobregat

Barcelona, 08907, Spain

Location

Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna

Madrid, 28034, Spain

Location

Hospital Quironsalud Zaragoza

Zaragoza, 50012, Spain

Location

Universität Zürich Psychiatrische Universitätsklinik

Zurich, 8032, Switzerland

Location

University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases

Birmingham, B15 2WB, United Kingdom

Location

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

London, NW3 2QG, United Kingdom

Location

National Hospital for Neurology and Neurosurgery

London, WC1N3BG, United Kingdom

Location

Salford Royal (Hope) Hospital

Salford, M6 8HD, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease

Interventions

migalastat

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Idorsia Clinical Trial Information
• Organization: Idorsia Pharmaceuticals Ltd

idorsiaclinicaltrials@idorsia.com

+1 856 661 3721

Study Officials

• Clinical Trials

  Idorsia Pharmaceuticals Ltd.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 16, 2018
• First Posted: February 7, 2018
• Study Start: June 21, 2018
• Primary Completion: August 17, 2021
• Study Completion: September 2, 2021
• Last Updated: August 9, 2024
• Results First Posted: August 9, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

ES (4); CH (1); GB (4); DE (4); IE (1); NO (1); AU (1); CA (6); US (17); IT (2); NL (1); PL (3); BE (2)