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

Fabry Disease Clinical Trial

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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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03425539
• Other study ID #: ID-069A301
• Status: Completed
• Phase: Phase 3
• Start date: June 21, 2018
• Est. completion date: September 2, 2021

Study information

• Verified date: August 2022
• Source: Idorsia Pharmaceuticals Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 182
• Est. completion date: September 2, 2021
• Est. primary completion date: August 17, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

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.
• 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.

Locations

Country	Name	City	State
Australia	Royal Melbourne Hospital - Department of Nephrology	Parkville
Australia	Royal Perth Hospital, Department of Nephrology	Perth
Austria	Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse	Vienna
Belgium	University Hospital Ghent	Ghent
Belgium	University Hospital Leuven	Leuven
Canada	M.A.G.I.C Clinic Ltd	Calgary
Canada	Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Nephrology	Halifax
Canada	London Health Sciences Centre - Victoria Hospital	London	Ontario
Canada	Research Center, Hôpital Du Sacré-Coeur de Montréal	Montréal
Canada	Vancouver Hospital & Health Sciences - Vancouver General Hospital	Vancouver
Canada	Health Sciences Center Winnipeg	Winnipeg
Germany	Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin	Berlin
Germany	SphinCS GmbH	Hochheim
Germany	Fachinternistische Gemeinschaftspraxis Markgräferland	Mühlheim
Germany	Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie	Würzburg
Ireland	Hosp Alma Mater Studiorum	Dublin
Italy	ASST Monza, Hospital San Gerardo, Nephrology	Monza
Italy	University of Naples Federico II (Nephrology)	Naples
Netherlands	Hospital Academisch Medisch Centrum - Department of Internal Medicine Div. Endrocrinology and Metabolism	Amsterdam
Norway	Haukeland University Hospital Helse Bergen HF	Bergen
Poland	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
Spain	Hospital Universitari de Bellvitge; Hospitalet de Llobregat	Barcelona
Spain	Hospital Universitari Vall d'Hebrón	Barcelona
Spain	Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna	Madrid
Spain	Hospital Quironsalud Zaragoza	Zaragoza
Switzerland	Universität Zürich Psychiatrische Universitätsklinik	Zurich
United Kingdom	University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases	Birmingham
United Kingdom	National Hospital for Neurology and Neurosurgery	London
United Kingdom	The Royal Free Hospital, Department of Haematology Royal Free London NHS Foundation Trust	London
United Kingdom	Salford Royal (Hope) Hospital	Salford
United States	Emory University School of Medicine; Department of Human Genetics	Atlanta	Georgia
United States	University of Alabama at Birmingham - Nephrology Research Clinic	Birmingham	Alabama
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Rush University Medical Center	Chicago	Illinois
United States	Research Baylor Institute of Metabolic Disease	Dallas	Texas
United States	Duke University Medical Center	Durham	North Carolina
United States	Lysosomal and Rare Disorders Research and Treatment Center	Fairfax	Virginia
United States	University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation	Gainesville	Florida
United States	Infusion Associates	Grand Rapids	Michigan
United States	Greenwood Genetic Center	Greenville	South Carolina
United States	University of Iowa Stead Family Children's Hospital - Division of Medical Genetics	Iowa City	Iowa
United States	University of California Irvine	Irvine	California
United States	UCSF Benioff Children's Hospital Oakland	Oakland	California
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Children's Hospital of Pittsburgh (UPMC)	Pittsburgh	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	University of Utah - Division of Medical Genetics	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Idorsia Pharmaceuticals Ltd.

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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"		From baseline to Month 6 (duration: 6 months)
Secondary	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.		From baseline to Month 6 (duration: 6 months)
Secondary	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.		From baseline to Month 6 (duration: 6 months)
Secondary	Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3).		From baseline to Month 6 (duration: 6 months)