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

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ClinicalTrials.gov Identifier: NCT03425539
Recruitment Status : Recruiting
First Posted : February 7, 2018
Last Update Posted : May 15, 2019
Sponsor:
Information provided by (Responsible Party):
Idorsia Pharmaceuticals Ltd.

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

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: Lucerastat Drug: Placebo Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 108 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : June 21, 2018
Estimated Primary Completion Date : December 7, 2019
Estimated Study Completion Date : January 7, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Lucerastat Drug: Lucerastat
Hard gelatin capsules containing 250 mg of lucerastat and inactive excipients; 1000 mg (4 capsules) b.i.d.; dose adjusted for renal function.

Placebo Comparator: Placebo 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.




Primary Outcome Measures :
  1. Response to study treatment on neuropathic pain defined as a reduction from baseline to Month 6 of at least 30% in the "modified" Brief Pain Inventory-Short Form 3 (BPI-SF3) score of "neuropathic pain at its worst in the last 24 hours" [ Time Frame: From baseline to Month 6 (duration: 6 months) ]

Secondary Outcome Measures :
  1. Change from baseline to Month 6 in the average daily 11-point Numerical Rating Scale (NRS-11) score of "abdominal pain at its worst in the last 24 hours" in subjects with GI symptoms at baseline. [ Time Frame: From baseline to Month 6 (duration: 6 months) ]
  2. Change from baseline to Month 6 in the number of days with at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 in subjects with GI symptoms at baseline. [ Time Frame: From baseline to Month 6 (duration: 6 months) ]
  3. Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3). [ Time Frame: From baseline to Month 6 (duration: 6 months) ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03425539


Contacts
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Contact: Clinical Trial Disclosure Desk +18566613721 clinical-trials-disclosure@idorsia.com

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Locations
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United States, Alabama
University of Alabama at Birmingham - Nephrology Research Clinic Recruiting
Birmingham, Alabama, United States, 35233
United States, California
University of California Irvine Recruiting
Irvine, California, United States, 92696
United States, Florida
University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation Recruiting
Gainesville, Florida, United States, 32610
United States, Georgia
Emory University School of Medicine; Department of Human Genetics Recruiting
Atlanta, Georgia, United States, 30322
United States, Iowa
University of Iowa Stead Family Children's Hospital - Division of Medical Genetics Recruiting
Iowa City, Iowa, United States, 52242
United States, Minnesota
University of Minnesota - Pediatric Gene Therapy Recruiting
Minneapolis, Minnesota, United States, 55455
United States, Ohio
Cincinnati Children's Hospital Medical Center - Human Genetics Dept. Not yet recruiting
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Children's Hospital of Pittsburgh (UPMC) Recruiting
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Research Baylor Institute of Metabolic Disease Recruiting
Dallas, Texas, United States, 75226
United States, Utah
University of Utah - Division of Medical Genetics Recruiting
Salt Lake City, Utah, United States, 84113
United States, Virginia
Lysosomal and Rare Disorders Research and Treatment Center Recruiting
Fairfax, Virginia, United States, 22030
Australia
Royal Melbourne Hospital - Department of Nephrology Recruiting
Parkville, Australia, 3050
Royal Perth Hospital, Department of Nephrology Recruiting
Perth, Australia, 6000
Austria
Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse Recruiting
Vienna, Austria, 1090
Canada, Alberta
M.A.G.I.C Clinic Ltd Recruiting
Calgary, Alberta, Canada, T2M 0L6
Canada
Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Neurosurgery Recruiting
Halifax, Canada, B3H 3A7
Vancouver Hospital & Health Sciences - Vancouver General Hospital Recruiting
Vancouver, Canada, V5Z 1M9
Health Sciences Center Winnipeg Recruiting
Winnipeg, Canada, R3A 1S1
Germany
Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin Recruiting
Berlin, Germany, 13353
Fachinternistische Gemeinschaftspraxis Markgräferland Recruiting
Mühlheim, Germany, 79379
Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie Recruiting
Würzburg, Germany, 97080
Netherlands
Hospital Academisch Medisch Centrum - Department of Internal Medicine Div. Endrocrinology and Metabolism Recruiting
Amsterdam, Netherlands, 22660
Poland
University Hospital in Cracow - Dep. of of Allergies and Immunology Recruiting
Krakow, Poland, 31-066
Cardinal Wyszynski Institute of Cardiology Not yet recruiting
Warsaw, Poland, 04-628
Department of Pediatric Nutrition and Metabolic Diseases; The Children's Memorial Health Institute Recruiting
Warsaw, Poland, 04-730
United Kingdom
University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases Recruiting
Birmingham, United Kingdom, B15 2WB
University Hospital of Wales Not yet recruiting
Cardiff, United Kingdom, CF14 4XW
Lysosomal Storage Disorders Unit, Department of Hematology - Royal Free London NHS Foundation Trust Recruiting
London, United Kingdom, NW3 2QG
The Royal Free Hospital, Department of Haematology Royal Free London NHS Foundation Trust Recruiting
London, United Kingdom, NW3 2QG
National Hospital for Neurology and Neurosurgery Not yet recruiting
London, United Kingdom, WC1N3BG
Salford Royal (Hope) Hospital Recruiting
Salford, United Kingdom, M6 8HD
Sponsors and Collaborators
Idorsia Pharmaceuticals Ltd.
Investigators
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Study Director: Clinical Trials Idorsia Pharmaceuticals Ltd.

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Responsible Party: Idorsia Pharmaceuticals Ltd.
ClinicalTrials.gov Identifier: NCT03425539     History of Changes
Other Study ID Numbers: ID-069A301
First Posted: February 7, 2018    Key Record Dates
Last Update Posted: May 15, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Fabry Disease
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
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders