A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

Fabry Disease Clinical Trial

Official title:

A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03737214
• Other study ID #: ID-069A302
• Secondary ID: 2018-002210-12
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: December 18, 2018
• Est. completion date: November 29, 2025

Study information

• Verified date: January 2024
• Source: Idorsia Pharmaceuticals Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease

Description:

Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country or until all subjects have reached Month 24 (for subjects participating only in Stage 1) or Month 48 (for subjects participating in Stages 1 and 2) or Month 72 (for subjects participating in Stages 1, 2 and 3), whichever is earliest.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 107
• Est. completion date: November 29, 2025
• Est. primary completion date: October 29, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Signed ICF prior to any study-mandated procedure;
• Subject completed the 6-month, double-blind treatment period in study ID 069A301
• Woman of childbearing potential only if agreement 1) to follow a specified contraception scheme, 2) to undertake monthly urine pregnancy tests , 3) not to donate ova.
• Fertile male only if agreement 1) to use a condom, 2) to not father a child, 3) not to donate sperm.

Exclusion Criteria:

• Pregnant / planning to be become pregnant or lactating subject;
• Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
• Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment. In addition, the subject must not be enrolled in study ID-069A302 if at any time during

study ID-069A301, one of the following criteria was met:

• Subject's eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
• Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
• Subject experienced an event of stroke CTCAE grade 3 or above;
• Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• Lucerastat
Administered in hard gelatin capsules containing 250 mg of lucerastat.

Locations

Country	Name	City	State
Australia	Royal Melbourne Hospital - Department of Nephrology	Parkville
Australia	Royal Perth Hospital, Department of Nephrology	Perth
Austria	Medizinische Universität Wien, Universitätsklinik für Innere Medizin III, Klinische Abteilung für Nephrologie und Dialyse	Vienna
Belgium	University Hospital Ghent (UZ Ghent)	Ghent
Belgium	University Hospital Gasthuisberg, Leuven (UZ Leuven)	Leuven
Canada	University of Calgary - Heritage Medical Research Clinic	Calgary
Canada	London Health Sciences CTR, Victoria Hospital	London
Canada	Research Center, Hôpital du Sacré-Coeur de Montréal	Montréal
Canada	Vancouver General Hospital - Adult Metabolic Diseases Clinic	Vancouver
Canada	Children's Hospital Research Institute of Manitoba	Winnipeg
France	Raymond Poincaré Hosp - Med Genetics Dept	Garches
Germany	Universitätsmedizin Berlin - Charité Campus Mitte	Berlin
Germany	SphinCS GmbH	Hochheim
Germany	Nephrologicum Markgräflerland MVZ GmbH	Müllheim
Germany	Universitätsklinikum Würzburg	Würzburg
Netherlands	Hospital Academisch Medisch Centrum - Department of Internal Medicine, Div. Endrocrinology and Metabolism	Amsterdam
Norway	Haukeland University Hospital	Bergen
Poland	Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital	Kraków
Poland	Narodowy Instytut Kardiologii Stefana kardynala Wyszynskiego - Panstwowy Instytut Badawczy	Warsaw
Poland	The Children's Memorial Health Institute, Department of Pediatric, Nutrition and Metabolic Diseases	Warsaw
Spain	Hospital Universitari de Bellvitge / Nephrology Dpt	Barcelona
Spain	Vall d'Hebron University Hospital - Unit of Inherited Metabolic Disorders and Rare Diseases	Barcelona
Spain	Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna	Madrid
Spain	Hospital Quironsalud Zaragoza	Zaragoza
Switzerland	Psychiatrische Universitätsklinik Zürich	Zürich
United Kingdom	National Hospital for Neurology and Neurosurgery	London
United Kingdom	Royal Free London NHS Foundation Trust Lysosomal Storage Disorder Unit; Department of Hematology	London
United Kingdom	Salford Hospital	Manchester
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 - Dept of Pediatrics	Chicago	Illinois
United States	Baylore University Medical Center	Dallas	Texas
United States	Renal Disease Research Institute LLC	Dallas	Texas
United States	Lysosomal and Rare Disorders Research and Treatment Center, Inc.	Fairfax	Virginia
United States	University of Florida Clinical and Translational Science Institute, UF Clinical Research Center	Gainesville	Florida
United States	Infusion Associates	Grand Rapids	Michigan
United States	Greenwood Genetics 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 - Dept of Medicine	Philadelphia	Pennsylvania
United States	University of Utah - Division of Medical Genetics, Clinical Genetics Research	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Idorsia Pharmaceuticals Ltd.

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Treatment-emergent serious adverse events (SAEs)		From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)
Other	Subject estimated glomerular filtration rate (eGFR) slope		From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Other	Change in left ventricular mass index (LVMI)		From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Other	Change in plasma globotriaosylceramide (Gb3)		From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Primary	Treatment-emergent adverse events (AEs)		From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)