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

NCT ID: NCT03425539

Last Updated: 2024-08-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 118 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-06-21
• Study Completion Date: 2021-09-02

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.

Conditions

Fabry Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Lucerastat

Group Type: EXPERIMENTAL

Lucerastat

Intervention Type: 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.

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: 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.

Interventions

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.

Intervention Type: 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.

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Idorsia Pharmaceuticals Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trials

Role: STUDY_DIRECTOR

Idorsia Pharmaceuticals Ltd.

Locations

University of Alabama at Birmingham - Nephrology Research Clinic

Birmingham, Alabama, United States

University of California Irvine

Irvine, California, United States

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

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

Gainesville, Florida, United States

Emory University School of Medicine; Department of Human Genetics

Atlanta, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

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

Iowa City, Iowa, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Infusion Associates

Grand Rapids, Michigan, United States

Duke University Medical Center

Durham, North Carolina, United States

Oregon Health and Science University

Portland, Oregon, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh (UPMC)

Pittsburgh, Pennsylvania, United States

Greenwood Genetic Center

Greenville, South Carolina, United States

Research Baylor Institute of Metabolic Disease

Dallas, Texas, United States

University of Utah - Division of Medical Genetics

Salt Lake City, Utah, United States

Lysosomal and Rare Disorders Research and Treatment Center

Fairfax, Virginia, United States

Royal Melbourne Hospital - Department of Nephrology

Parkville, , Australia

Royal Perth Hospital, Department of Nephrology

Perth, , Australia

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

Vienna, , Austria

University Hospital Ghent

Ghent, , Belgium

University Hospital Leuven

Leuven, , Belgium

London Health Sciences Centre - Victoria Hospital

London, Ontario, Canada

M.A.G.I.C Clinic Ltd

Calgary, , Canada

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

Halifax, , Canada

Research Center, Hôpital Du Sacré-Coeur de Montréal

Montreal, , Canada

Vancouver Hospital & Health Sciences - Vancouver General Hospital

Vancouver, , Canada

Health Sciences Center Winnipeg

Winnipeg, , Canada

Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin

Berlin, , Germany

SphinCS GmbH

Höchheim, , Germany

Fachinternistische Gemeinschaftspraxis Markgräferland

Mühlheim, , Germany

Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie

Würzburg, , Germany

Hosp Alma Mater Studiorum

Dublin, , Ireland

ASST Monza, Hospital San Gerardo, Nephrology

Monza, , Italy

University of Naples Federico II (Nephrology)

Naples, , Italy

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

Amsterdam, , Netherlands

Haukeland University Hospital Helse Bergen HF

Bergen, , Norway

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

Krakow, , Poland

Cardinal Wyszynski Institute of Cardiology

Warsaw, , Poland

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

Warsaw, , Poland

Hospital Universitari Vall d'Hebrón

Barcelona, , Spain

Hospital Universitari de Bellvitge; Hospitalet de Llobregat

Barcelona, , Spain

Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna

Madrid, , Spain

Hospital Quironsalud Zaragoza

Zaragoza, , Spain

Universität Zürich Psychiatrische Universitätsklinik

Zurich, , Switzerland

University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases

Birmingham, , United Kingdom

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

London, , United Kingdom

National Hospital for Neurology and Neurosurgery

London, , United Kingdom

Salford Royal (Hope) Hospital

Salford, , United Kingdom

Countries

United States; Australia; Austria; Belgium; Canada; Germany; Ireland; Italy; Netherlands; Norway; Poland; Spain; Switzerland; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

ID-069A301

Identifier Type: -

Identifier Source: org_study_id