A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease
NCT ID: NCT02930655
Last Updated: 2018-07-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
14 participants
INTERVENTIONAL
2015-02-01
2016-02-01
Brief Summary
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The secondary objectives were to investigate the effects of lucerastat on plasma and urine levels of biomarkers, to assess its effects on renal and cardiac functions and to determine the pharmacokinetic profile of lucerastat at steady-state.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Lucerastat group
Ten subjects with Fabry Disease received 1000 mg of oral lucerastat twice daily for 12 weeks in addition to their standard of care treatment (enzyme replace therapy).
Lucerastat
Hard gelatin capsules for oral administration formulated at a strength of 250 mg, and administered as 4 capsules in the morning and 4 capsules in the evening.
Enzyme replacement therapy (ERT)
All the subjects received an ERT as background therapy for at least 24 months prior to the screening visit and they had to continue receiving this treatment during the conduct of the study.
Control group
Four subjects with Fabry Disease under enzyme replace therapy (ERT) as standard of care treatment were included as a control group.
Enzyme replacement therapy (ERT)
All the subjects received an ERT as background therapy for at least 24 months prior to the screening visit and they had to continue receiving this treatment during the conduct of the study.
Interventions
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Lucerastat
Hard gelatin capsules for oral administration formulated at a strength of 250 mg, and administered as 4 capsules in the morning and 4 capsules in the evening.
Enzyme replacement therapy (ERT)
All the subjects received an ERT as background therapy for at least 24 months prior to the screening visit and they had to continue receiving this treatment during the conduct of the study.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Male and female adult subjects with a diagnosis of Fabry Disease (FD) based on historical assessments (residual α-GAL A activity level below lower limit of normal for males and presence of a galactosidase alpha mutation for females) and a history of clinical symptoms of FD
* On ERT for at least 24 months without any change in dose within the last 6 months prior to screening
Exclusion Criteria
* Severe residual neurologic deficit
* Clinically significant unstable cardiac disease
* Any circumstances or conditions, which, in the opinion of the investigator, may have affected full participation in the study or compliance with the protocol
18 Years
ALL
No
Sponsors
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Idorsia Pharmaceuticals Ltd.
INDUSTRY
Responsible Party
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Principal Investigators
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Nicolas Guérard
Role: STUDY_DIRECTOR
Actelion
Locations
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Investigator Site
Würzburg, , Germany
Countries
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References
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Guerard N, Morand O, Dingemanse J. Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects. Orphanet J Rare Dis. 2017 Jan 14;12(1):9. doi: 10.1186/s13023-017-0565-9.
Other Identifiers
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AC-069-104
Identifier Type: -
Identifier Source: org_study_id
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