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

Primary Purpose

Fabry Disease

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Lucerastat
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:

  • 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.

Sites / Locations

  • University of Alabama at Birmingham - Nephrology Research Clinic
  • University of California Irvine
  • Department of Gastroenterology
  • University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation
  • Rush University Medical Center
  • University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
  • Massachusetts General Hospital
  • Infusion Associates
  • University of Pennsylvania
  • Renal Disease Research Institute
  • University of Utah
  • Lysosomal and Rare Disorders Research and Treatment Center
  • Royal Melbourne Hospital, Department of Respiratory Medicine
  • Royal Perth Hospital Unit - The University of Western Australia
  • Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse
  • University Hospital Leuven
  • London Health Sciences Centre - Victoria Hospital
  • M.A.G.I.C Clinic Ltd
  • Research Center, Hôpital Du Sacré-Coeur de Montréal
  • Vancouver Hospital & Health Sciences - Vancouver General Hospital
  • Health Sciences Center Winnipeg
  • Raymond Poincare University Hospital
  • Charité-Virchow Klinikum - Nephrologie und Internistische Intensivmedizin
  • SphinCS GmbH
  • Fachinternistische Gemeinschaftspraxis Markgräferland
  • Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie
  • Haukeland University Hospital Helse Bergen HF
  • 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
  • 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 Type

Experimental

Arm Label

Lucerastat

Arm Description

Dose will be based on subject's eGFR.

Outcomes

Primary Outcome Measures

Treatment-emergent adverse events (AEs)

Secondary Outcome Measures

Full Information

First Posted
November 6, 2018
Last Updated
August 15, 2023
Sponsor
Idorsia Pharmaceuticals Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT03737214
Brief Title
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 18, 2018 (Actual)
Primary Completion Date
October 29, 2025 (Anticipated)
Study Completion Date
November 29, 2025 (Anticipated)

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

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
Single Group Assignment
Model Description
Multi-center, open-label, uncontrolled, single-arm, extension study
Masking
None (Open Label)
Allocation
N/A
Enrollment
107 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Lucerastat
Arm Type
Experimental
Arm Description
Dose will be based on subject's eGFR.
Intervention Type
Drug
Intervention Name(s)
Lucerastat
Intervention Description
Administered in hard gelatin capsules containing 250 mg of lucerastat.
Primary Outcome Measure Information:
Title
Treatment-emergent adverse events (AEs)
Time Frame
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 Pre-specified Outcome Measures:
Title
Treatment-emergent serious adverse events (SAEs)
Time Frame
From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)
Title
Subject estimated glomerular filtration rate (eGFR) slope
Time Frame
From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Title
Change in left ventricular mass index (LVMI)
Time Frame
From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)
Title
Change in plasma globotriaosylceramide (Gb3)
Time Frame
From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
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
35294
Country
United States
Facility Name
University of California Irvine
City
Irvine
State/Province
California
ZIP/Postal Code
92697
Country
United States
Facility Name
Department of Gastroenterology
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
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
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Renal Disease Research Institute
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
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 Respiratory Medicine
City
Parkville
ZIP/Postal Code
3052
Country
Australia
Facility Name
Royal Perth Hospital Unit - The University of Western Australia
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 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
T2N 4Z6
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
R3E 3P4
Country
Canada
Facility Name
Raymond Poincare University Hospital
City
Garches
ZIP/Postal Code
92380
Country
France
Facility Name
Charité-Virchow Klinikum - Nephrologie und Internistische Intensivmedizin
City
Berlin
ZIP/Postal Code
10117
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
Haukeland University Hospital Helse Bergen HF
City
Bergen
ZIP/Postal Code
5021
Country
Norway
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
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
Citations:
PubMed Identifier
35782623
Citation
Wanner C, Kimonis V, Politei J, Warnock DG, Uceyler N, Frey A, Cornelisse P, Hughes D. Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study. Mol Genet Metab Rep. 2022 Mar 26;31:100862. doi: 10.1016/j.ymgmr.2022.100862. eCollection 2022 Jun.
Results Reference
derived

Learn more about this trial

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

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