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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03425539




Registration number
NCT03425539
Ethics application status
Date submitted
16/01/2018
Date registered
7/02/2018

Titles & IDs
Public title
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Scientific 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
Secondary ID [1] 0 0
ID-069A301
Universal Trial Number (UTN)
Trial acronym
MODIFY
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
Treatment: Drugs - Placebo

Experimental: Lucerastat -

Placebo comparator: Placebo -


Treatment: Drugs: 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.

Treatment: Drugs: 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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Neuropathic Pain Monthly Score: Change From Baseline to Month 6
Timepoint [1] 0 0
From baseline to Month 6 (duration: 6 months)
Secondary outcome [1] 0 0
Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6
Timepoint [1] 0 0
From baseline to Month 6 (duration: 6 months)
Secondary outcome [2] 0 0
Abdominal Pain Monthly Score: Change From Baseline to Month 6
Timepoint [2] 0 0
From baseline to Month 6 (duration: 6 months)
Secondary outcome [3] 0 0
Number of Days With Diarrhea: Change From Baseline to Month 6
Timepoint [3] 0 0
From baseline to Month 6 (duration: 6 months)

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/06/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
2/09/2021
Sample size
Target
Accrual to date
Final
118
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
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Oregon
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
South Carolina
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United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Utah
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United States of America
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Virginia
Country [16] 0 0
Austria
State/province [16] 0 0
Vienna
Country [17] 0 0
Belgium
State/province [17] 0 0
Ghent
Country [18] 0 0
Belgium
State/province [18] 0 0
Leuven
Country [19] 0 0
Canada
State/province [19] 0 0
Ontario
Country [20] 0 0
Canada
State/province [20] 0 0
Calgary
Country [21] 0 0
Canada
State/province [21] 0 0
Halifax
Country [22] 0 0
Canada
State/province [22] 0 0
Montréal
Country [23] 0 0
Canada
State/province [23] 0 0
Vancouver
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Canada
State/province [24] 0 0
Winnipeg
Country [25] 0 0
Germany
State/province [25] 0 0
Berlin
Country [26] 0 0
Germany
State/province [26] 0 0
Hochheim
Country [27] 0 0
Germany
State/province [27] 0 0
Mühlheim
Country [28] 0 0
Germany
State/province [28] 0 0
Würzburg
Country [29] 0 0
Ireland
State/province [29] 0 0
Dublin
Country [30] 0 0
Italy
State/province [30] 0 0
Monza
Country [31] 0 0
Italy
State/province [31] 0 0
Naples
Country [32] 0 0
Netherlands
State/province [32] 0 0
Amsterdam
Country [33] 0 0
Norway
State/province [33] 0 0
Bergen
Country [34] 0 0
Poland
State/province [34] 0 0
Krakow
Country [35] 0 0
Poland
State/province [35] 0 0
Warsaw
Country [36] 0 0
Spain
State/province [36] 0 0
Barcelona
Country [37] 0 0
Spain
State/province [37] 0 0
Madrid
Country [38] 0 0
Spain
State/province [38] 0 0
Zaragoza
Country [39] 0 0
Switzerland
State/province [39] 0 0
Zurich
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Birmingham
Country [41] 0 0
United Kingdom
State/province [41] 0 0
London
Country [42] 0 0
United Kingdom
State/province [42] 0 0
Salford

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03425539