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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03737214




Registration number
NCT03737214
Ethics application status
Date submitted
6/11/2018
Date registered
9/11/2018
Date last updated
3/07/2024

Titles & IDs
Public title
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Scientific 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
Secondary ID [1] 0 0
2018-002210-12
Secondary ID [2] 0 0
ID-069A302
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Lucerastat

Experimental: Lucerastat - Dose will be based on subject's eGFR.


Treatment: Drugs: Lucerastat
Administered in hard gelatin capsules containing 250 mg of lucerastat.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Treatment-emergent adverse events (AEs)
Timepoint [1] 0 0
From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up)

Eligibility
Key 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.
* Fertile male only if agreement 1) to use a condom, 2) to not father a child.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/12/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
29/11/2025
Actual
Sample size
Target
107
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Department of Nephrology - Parkville
Recruitment hospital [2] 0 0
Royal Perth Hospital, Department of Nephrology - Perth
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment postcode(s) [2] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Iowa
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
South Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
United States of America
State/province [11] 0 0
Utah
Country [12] 0 0
United States of America
State/province [12] 0 0
Virginia
Country [13] 0 0
Austria
State/province [13] 0 0
Vienna
Country [14] 0 0
Belgium
State/province [14] 0 0
Ghent
Country [15] 0 0
Belgium
State/province [15] 0 0
Leuven
Country [16] 0 0
Canada
State/province [16] 0 0
Calgary
Country [17] 0 0
Canada
State/province [17] 0 0
London
Country [18] 0 0
Canada
State/province [18] 0 0
Montréal
Country [19] 0 0
Canada
State/province [19] 0 0
Vancouver
Country [20] 0 0
Canada
State/province [20] 0 0
Winnipeg
Country [21] 0 0
France
State/province [21] 0 0
Garches
Country [22] 0 0
Germany
State/province [22] 0 0
Berlin
Country [23] 0 0
Germany
State/province [23] 0 0
Hochheim
Country [24] 0 0
Germany
State/province [24] 0 0
Müllheim
Country [25] 0 0
Germany
State/province [25] 0 0
Würzburg
Country [26] 0 0
Netherlands
State/province [26] 0 0
Amsterdam
Country [27] 0 0
Norway
State/province [27] 0 0
Bergen
Country [28] 0 0
Poland
State/province [28] 0 0
Kraków
Country [29] 0 0
Poland
State/province [29] 0 0
Warsaw
Country [30] 0 0
Spain
State/province [30] 0 0
Barcelona
Country [31] 0 0
Spain
State/province [31] 0 0
Madrid
Country [32] 0 0
Spain
State/province [32] 0 0
Zaragoza
Country [33] 0 0
Switzerland
State/province [33] 0 0
Zürich
Country [34] 0 0
United Kingdom
State/province [34] 0 0
London
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Idorsia Pharmaceuticals Ltd.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.
Trial website
https://clinicaltrials.gov/study/NCT03737214
Trial related presentations / publications
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.
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trials
Address 0 0
Idorsia Pharmaceuticals Ltd.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03737214