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

Primary Purpose

Fabry Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Lucerastat
Placebo
Sponsored by
Idorsia Pharmaceuticals Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Alabama at Birmingham - Nephrology Research Clinic
  • University of California Irvine
  • UCSF Benioff Children's Hospital Oakland
  • University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation
  • Emory University School of Medicine; Department of Human Genetics
  • Rush University Medical Center
  • University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
  • Massachusetts General Hospital
  • Infusion Associates
  • Duke University Medical Center
  • Oregon Health and Science University
  • University of Pennsylvania
  • Children's Hospital of Pittsburgh (UPMC)
  • Greenwood Genetic Center
  • Research Baylor Institute of Metabolic Disease
  • University of Utah - Division of Medical Genetics
  • Lysosomal and Rare Disorders Research and Treatment Center
  • Royal Melbourne Hospital - Department of Nephrology
  • Royal Perth Hospital, Department of Nephrology
  • Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse
  • University Hospital Ghent
  • University Hospital Leuven
  • London Health Sciences Centre - Victoria Hospital
  • M.A.G.I.C Clinic Ltd
  • Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Nephrology
  • Research Center, Hôpital Du Sacré-Coeur de Montréal
  • Vancouver Hospital & Health Sciences - Vancouver General Hospital
  • Health Sciences Center Winnipeg
  • Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin
  • SphinCS GmbH
  • Fachinternistische Gemeinschaftspraxis Markgräferland
  • Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie
  • Hosp Alma Mater Studiorum
  • ASST Monza, Hospital San Gerardo, Nephrology
  • University of Naples Federico II (Nephrology)
  • Hospital Academisch Medisch Centrum - Department of Internal Medicine Div. Endrocrinology and Metabolism
  • Haukeland University Hospital Helse Bergen HF
  • University Hospital in Cracow - Dep. of of Allergies and Immunology
  • Cardinal Wyszynski Institute of Cardiology
  • Department of Pediatric Nutrition and Metabolic Diseases; The Children's Memorial Health Institute
  • Hospital Universitari Vall d'Hebrón
  • Hospital Universitari de Bellvitge; Hospitalet de Llobregat
  • Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
  • Hospital Quironsalud Zaragoza
  • Universität Zürich Psychiatrische Universitätsklinik
  • University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases
  • The Royal Free Hospital, Department of Haematology Royal Free London NHS Foundation Trust
  • National Hospital for Neurology and Neurosurgery
  • Salford Royal (Hope) Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Lucerastat

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Change from baseline to Month 6 in the "modified" Brief Pain Inventory-Short Form 3 (BPI-SF3) score of "neuropathic pain at its worst in the last 24 hours"

Secondary Outcome Measures

Change from baseline to Month 6 in the 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.
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.
Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3).

Full Information

First Posted
January 16, 2018
Last Updated
August 5, 2022
Sponsor
Idorsia Pharmaceuticals Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT03425539
Brief Title
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Acronym
MODIFY
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

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Completed
Study Start Date
June 21, 2018 (Actual)
Primary Completion Date
August 17, 2021 (Actual)
Study Completion Date
September 2, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Idorsia Pharmaceuticals Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
182 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Lucerastat
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Lucerastat
Intervention Description
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
Intervention Name(s)
Placebo
Intervention Description
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 Measure Information:
Title
Change from baseline to Month 6 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 Measure Information:
Title
Change from baseline to Month 6 in the 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)
Title
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)
Title
Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3).
Time Frame
From baseline to Month 6 (duration: 6 months)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Idorsia Pharmaceuticals Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama at Birmingham - Nephrology Research Clinic
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
University of California Irvine
City
Irvine
State/Province
California
ZIP/Postal Code
92696
Country
United States
Facility Name
UCSF Benioff Children's Hospital Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Emory University School of Medicine; Department of Human Genetics
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Infusion Associates
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49525
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Children's Hospital of Pittsburgh (UPMC)
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Greenwood Genetic Center
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29605
Country
United States
Facility Name
Research Baylor Institute of Metabolic Disease
City
Dallas
State/Province
Texas
ZIP/Postal Code
75226
Country
United States
Facility Name
University of Utah - Division of Medical Genetics
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Facility Name
Lysosomal and Rare Disorders Research and Treatment Center
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Royal Melbourne Hospital - Department of Nephrology
City
Parkville
ZIP/Postal Code
3050
Country
Australia
Facility Name
Royal Perth Hospital, Department of Nephrology
City
Perth
ZIP/Postal Code
6000
Country
Australia
Facility Name
Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse
City
Vienna
ZIP/Postal Code
1090
Country
Austria
Facility Name
University Hospital Ghent
City
Ghent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
University Hospital Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
London Health Sciences Centre - Victoria Hospital
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Facility Name
M.A.G.I.C Clinic Ltd
City
Calgary
ZIP/Postal Code
T2M 0L6
Country
Canada
Facility Name
Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Nephrology
City
Halifax
ZIP/Postal Code
B3H 3A7
Country
Canada
Facility Name
Research Center, Hôpital Du Sacré-Coeur de Montréal
City
Montréal
ZIP/Postal Code
H4J 1C5
Country
Canada
Facility Name
Vancouver Hospital & Health Sciences - Vancouver General Hospital
City
Vancouver
ZIP/Postal Code
V5Z 1M9
Country
Canada
Facility Name
Health Sciences Center Winnipeg
City
Winnipeg
ZIP/Postal Code
R3A 1S1
Country
Canada
Facility Name
Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
SphinCS GmbH
City
Hochheim
ZIP/Postal Code
65239
Country
Germany
Facility Name
Fachinternistische Gemeinschaftspraxis Markgräferland
City
Mühlheim
ZIP/Postal Code
79379
Country
Germany
Facility Name
Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie
City
Würzburg
ZIP/Postal Code
97080
Country
Germany
Facility Name
Hosp Alma Mater Studiorum
City
Dublin
ZIP/Postal Code
DD7 R2WY
Country
Ireland
Facility Name
ASST Monza, Hospital San Gerardo, Nephrology
City
Monza
ZIP/Postal Code
20900
Country
Italy
Facility Name
University of Naples Federico II (Nephrology)
City
Naples
ZIP/Postal Code
80131
Country
Italy
Facility Name
Hospital Academisch Medisch Centrum - Department of Internal Medicine Div. Endrocrinology and Metabolism
City
Amsterdam
ZIP/Postal Code
22660
Country
Netherlands
Facility Name
Haukeland University Hospital Helse Bergen HF
City
Bergen
ZIP/Postal Code
5021
Country
Norway
Facility Name
University Hospital in Cracow - Dep. of of Allergies and Immunology
City
Krakow
ZIP/Postal Code
31-066
Country
Poland
Facility Name
Cardinal Wyszynski Institute of Cardiology
City
Warsaw
ZIP/Postal Code
04-628
Country
Poland
Facility Name
Department of Pediatric Nutrition and Metabolic Diseases; The Children's Memorial Health Institute
City
Warsaw
ZIP/Postal Code
04-730
Country
Poland
Facility Name
Hospital Universitari Vall d'Hebrón
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital Universitari de Bellvitge; Hospitalet de Llobregat
City
Barcelona
ZIP/Postal Code
08907
Country
Spain
Facility Name
Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Facility Name
Hospital Quironsalud Zaragoza
City
Zaragoza
ZIP/Postal Code
50012
Country
Spain
Facility Name
Universität Zürich Psychiatrische Universitätsklinik
City
Zurich
ZIP/Postal Code
8032
Country
Switzerland
Facility Name
University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases
City
Birmingham
ZIP/Postal Code
B15 2WB
Country
United Kingdom
Facility Name
The Royal Free Hospital, Department of Haematology Royal Free London NHS Foundation Trust
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
National Hospital for Neurology and Neurosurgery
City
London
ZIP/Postal Code
WC1N3BG
Country
United Kingdom
Facility Name
Salford Royal (Hope) Hospital
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

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

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