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Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

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ClinicalTrials.gov Identifier: NCT02489344
Recruitment Status : Completed
First Posted : July 3, 2015
Last Update Posted : January 9, 2019
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Brief Summary:

Primary Objective:

To assess the long-term safety of GZ/SAR402671 in adult male patients with Fabry disease who previously completed study ACT13739.

Secondary Objective:

To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male patients with Fabry disease who previously completed study ACT13739.


Condition or disease Intervention/treatment Phase
Fabry Disease Drug: GZ/SAR402671 Phase 2

Detailed Description:
The total duration of study period will be up to 31 months.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed With Fabry Disease
Actual Study Start Date : July 7, 2015
Actual Primary Completion Date : November 20, 2018
Actual Study Completion Date : November 20, 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: GZ/SAR402671
GZ/SAR402671 - Administered once a day, orally for 30 months
Drug: GZ/SAR402671

Pharmaceutical form:capsule

Route of administration: oral





Primary Outcome Measures :
  1. Characterization of the safety profile of GZ/SAR402671, including the frequency, duration, and severity of adverse events [ Time Frame: Through 30 months ]
  2. Clinically significant changes in laboratory parameters including hematology, biochemistry, and urinalysis [ Time Frame: Through 30 months ]
  3. Clinically significant changes in physical examinations including vital signs and body weight [ Time Frame: Through 30 months ]

Secondary Outcome Measures :
  1. Change from baseline in plasma globotriaosylceramide (GL-3) [ Time Frame: Through 30 months ]
  2. Change from baseline in plasma lyso GL-3 [ Time Frame: Through 30 months ]
  3. Change from baseline in plasma glucosylceramide (GL 1) [ Time Frame: Through 30 months ]
  4. Urine GL-3 change from baseline [ Time Frame: Through 30 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria :

  • Male patient with Fabry disease who previously completed study ACT13739.
  • Patients, willing and able to provide signed informed consent.
  • Sexually active patients, willing to practice true abstinence in line with their preferred and usual lifestyle or using two acceptable effective methods of contraception.

Exclusion criteria :

-Patients, in the opinion of the Investigator, unable to adhere to the requirements of the study.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02489344


Locations
United States, Georgia
Investigational Site Number 840002
Atlanta, Georgia, United States, 30322
United States, Ohio
Investigational Site Number 840003
Cincinnati, Ohio, United States, 45229
United States, Virginia
Investigational Site Number 840001
Fairfax, Virginia, United States, 22030
France
Investigational Site Number 250001
Garches, France, 92380
Poland
Investigational Site Number 616001
Warszawa, Poland, 04-730
Russian Federation
Investigational Site Number 643002
Moscow, Russian Federation, 125167
United Kingdom
Investigational Site Number 826002
Cambridge, United Kingdom, CB2 OQQ
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Clinical Sciences & Operations Sanofi

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT02489344     History of Changes
Other Study ID Numbers: LTS14116
2014-004995-49 ( EudraCT Number )
U1111-1165-9049 ( Other Identifier: UTN )
First Posted: July 3, 2015    Key Record Dates
Last Update Posted: January 9, 2019
Last Verified: January 2019

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders