Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
NCT ID: NCT02489344
Last Updated: 2021-03-23
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
8 participants
INTERVENTIONAL
2015-07-07
2018-11-20
Brief Summary
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To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344).
Secondary Objective:
To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male participants with Fabry disease who previously completed study ACT13739.
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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GZ/SAR402671
Participants received GZ/SAR402671 15 milligrams (mg) once daily orally for 30 months in this extension study (LTS14116).
GZ/SAR402671
Pharmaceutical form:capsule
Route of administration: oral
Interventions
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GZ/SAR402671
Pharmaceutical form:capsule
Route of administration: oral
Eligibility Criteria
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Inclusion Criteria
* Participants, willing and able to provide signed informed consent.
* Sexually active participants, willing to practice true abstinence in line with their preferred and usual lifestyle or using two acceptable effective methods of contraception.
Exclusion Criteria
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
18 Years
MALE
No
Sponsors
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Genzyme, a Sanofi Company
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Sciences & Operations
Role: STUDY_DIRECTOR
Sanofi
Locations
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Investigational Site Number 840002
Atlanta, Georgia, United States
Investigational Site Number 840003
Cincinnati, Ohio, United States
Investigational Site Number 840001
Fairfax, Virginia, United States
Investigational Site Number 250001
Garches, , France
Investigational Site Number 616001
Warsaw, , Poland
Investigational Site Number 643002
Moscow, , Russia
Investigational Site Number 826002
Cambridge, , United Kingdom
Countries
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References
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Deegan PB, Goker-Alpan O, Geberhiwot T, Hopkin RJ, Lukina E, Tylki-Szymanska A, Zaher A, Sensinger C, Gaemers SJM, Modur V, Thurberg BL, Sharma J, Najafian B, Mauer M, DasMahapatra P, Wilcox WR, Germain DP. Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study. Mol Genet Metab. 2023 Feb;138(2):106963. doi: 10.1016/j.ymgme.2022.11.002. Epub 2022 Nov 9.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2014-004995-49
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
U1111-1165-9049
Identifier Type: OTHER
Identifier Source: secondary_id
LTS14116
Identifier Type: -
Identifier Source: org_study_id
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