Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
Condition: Fabry Disease
MeSH terms: Fabry Disease
Intervention: GZ/SAR402671
Intervention description: Pharmaceutical form: Capsule; Route of administration: Oral
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 11.0
Sex: Male
Minimum age: 18 Years
Maximum age: 49 Years
Healthy volunteers: No
Keywords: Venglustat

Summary:

      Primary Objective:

      To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy
      of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants
      diagnosed with Fabry disease.
    

Eligibility criteria:

        Inclusion criteria:

          -  The participant was greater than equal to (>=) 18 years of age and less than (<) 50
             years of age.

          -  The participant was male.

          -  The participant had provided a signed informed consent.

          -  The participant had a confirmed diagnosis of Fabry disease as documented by leukocyte
             α- Galactosidase A (αGAL) activity of <4 nanomole/hour/milligram (nmol/hr/mg)
             leukocyte (preferred assay; results from a central laboratory) or plasma αGAL <1.5
             nanomole/hour/milliliter (nmol/hr/mL) (results from a central laboratory).

          -  The participant had a plasma globotriaosylsphingosine (lyso-GL3) >=65 nanogram per
             milliliter (ng/mL).

          -  The participant had never been treated with a Fabry disease-specific treatment.

          -  If the participant was on renin-angiotensin-aldosterone system (RAAS) blockers and
             antidepressants, the dose should be stable (i.e., prescribed dose and frequency) for
             at least the immediate 3 months prior to screening.

        Exclusion criteria:

          -  The participant had an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m^2
             using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI).

          -  The participant had a median urine protein/creatinine ratio (PCR) >=0.5 gram per gram
             (g/g) (median of 3 overnight urine collections. Collection of each of the 3 samples
             must occur between 4 and 7 days of each other, and all samples must be collected
             within a 15 day period). All 3 samples must be collected regardless of the results and
             results available prior to Day 1.

          -  The participant had undergone a kidney transplant.

          -  The participant had either active or a history of clinically significant organic
             disease (with the exception of the symptoms related to Fabry disease), including
             clinically significant cardiovascular, hepatic, pulmonary, hematologic, neurological
             or renal disease, or other medical condition, serious inter-current illness, or
             extenuating circumstances that, in the opinion of the Investigator, would preclude
             participation in the trial.

          -  The participant had abnormal liver function (serum total bilirubin > the upper limit
             of normal, or serum alanine aminotransferase ([ALT] and aspartate aminotransferase
             [AST] >2.0 times the upper limit of normal).

          -  The participant had, according to World Health Organization (WHO) grading a cortical
             cataract (COR) > one-quarter of the lens circumference (Grade COR-2) or a posterior
             subcapsular cataract (PSC) >2 millimeter (mm) (Grade PSC-2). Participants with nuclear
             cataracts were not excluded.

          -  The participant was currently receiving potentially cataractogenic medications.

          -  The participant had received strong or moderate inducers or inhibitors of Cytochrome
             P450 3A4 (CYP3A4) per Food and Drug Administration (FDA) classification within 14 days
             prior to enrollment or within 5 times the elimination half-life or PD half-life of the
             medication, whichever is longer.

          -  The participant was scheduled for in-patient hospitalization, including elective
             surgery, during the study.

          -  The participant had a positive result on any of the following tests: hepatitis B
             surface antigen (HBsAg), anti-hepatitis C virus (anti-HCV) antibodies, anti-human
             immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti-HIV2 Ab). Participants
             with a positive hepatitis B surface antibody (HBsAb) test with a history of prior
             hepatitis B immunization were eligible if other criteria met (i.e., negative tests
             for: HBsAg, hepatitis B core antibody [HBcAb], and hepatitis C virus antibody
             [HCVAb]).

          -  The participant had participated in a study involving an investigational drug within
             the past 30 days of the start of the trial.

          -  The participant was unwilling to comply with the requirements of the protocol.

          -  The participant was a sexually active man who was not willing to use 2 forms of birth
             control including a barrier method during the study until 6 weeks after the last
             treatment with investigational medicinal product (IMP).

          -  The participant had a history or ongoing clinically significant cardiac arrhythmia,
             defined as either atrial fibrillation, sustained or non-sustained ventricular
             tachycardia.

          -  The participant had any contraindication to magnetic resonance imaging (MRI).

          -  The participant had one of the following central nervous system exclusion criteria:

               -  Acute stroke, within 3 months of the screening visit.

               -  History of seizures.

        The above information is not intended to contain all considerations relevant to a
        participant's potential participation in a clinical trial.