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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03737214
Ethics application status
Date submitted
6/11/2018
Date registered
9/11/2018
Date last updated
26/03/2020

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 25 months (24 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 , 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.
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Pregnant / planning to be become pregnant up to 30 days after study treatment
discontinuation 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
N/A
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Melbourne Hospital, Department of Respiratory Medicine - Parkville
Recruitment hospital [2] 0 0
Royal Perth Hospital Unit - The University of Western Australia - Perth
Recruitment postcode(s) [1] 0 0
3052 - 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
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United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
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Florida
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Georgia
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Illinois
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Iowa
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Massachusetts
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Michigan
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Minnesota
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New Jersey
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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South Carolina
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United States of America
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Texas
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Utah
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United States of America
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Virginia
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Austria
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Vienna
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Belgium
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Ghent
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Belgium
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Leuven
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Canada
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Ontario
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Canada
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Calgary
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Canada
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Halifax
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Canada
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Montréal
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Canada
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Vancouver
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Canada
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Winnipeg
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Germany
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Berlin
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Germany
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Hochheim
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Germany
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Mühlheim
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Germany
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Würzburg
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Ireland
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Dublin
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Italy
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Florence
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Italy
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Monza
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Italy
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Naples
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Netherlands
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Amsterdam
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Norway
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Bergen
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Poland
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Katowice
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Poland
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Krakow
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Poland
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Warsaw
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Barcelona
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Spain
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Madrid
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Spain
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Zaragoza
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Switzerland
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Zurich
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United Kingdom
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Birmingham
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United Kingdom
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Cardiff
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United Kingdom
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London
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United Kingdom
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Salford

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
Trial website
https://clinicaltrials.gov/show/NCT03737214
Trial related presentations / publications
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
Clinical Trial Disclosure Desk
Address 0 0
Country 0 0
Phone 0 0
+18566613721
Fax 0 0
Email 0 0
clinical-trials-disclosure@idorsia.com
Contact person for scientific queries

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