Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ402671 in Treatment-naïve Adult Male Patients with Fabry Disease

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 8

Inclusion Criteria

The patient is =18 years of age and <50 years of age.
The patient is male.
The patient has provided a signed informed consent.
The patient had a confirmed diagnosis of Fabry disease as documented by leukocyte a-Galactosidase A (aGAL) activity of <4 nmol/hr/mg leukocyte (preferred assay; results from a central laboratory) or plasma aGAL <1.5 nmol/hr/mL (results from a central laboratory).
The patient has a plasma globotriaosylsphingosine (lyso-GL3) = 65 ng/mL.
The patient has never been treated with a Fabry disease-specific treatment.
If the patient is on renin-angiotensin-aldosterone system (RAAS) blockers and antidepressants, the dose should be stable (ie, prescribed dose and frequency) for at least the immediate 3 months prior to screening.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 8
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The patient has an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m^2 using (Kidney Disease Epidemiology Collaboration) CKD-EPI.
The patient has a median urine protein/creatinine ratio (PCR) =0.5 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 21 day period). All 3 samples must be collected regardless of the results and results available prior to Day 1.
The patient had undergone a kidney transplant.
The patient has 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 patient has 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 patient has, 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 mm (Grade PSC-2). Patients with nuclear cataracts are not excluded.
The patient is currently receiving potentially cataractogenic medications.
The patient has received strong or moderate inducers or inhibitors of Cytochrome P450 3A4 (CYP3A4) per Food and Drug Administration (FDA) classification within 14 days prior to enrolment or within 5 times the elimination half-life or PD half-life of the medication, whichever is longer.
The patient is scheduled for in-patient hospitalization, including elective surgery, during the study.
The patient has 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). Patients with a positive hepatitis B surface antibody (HBsAb) test with a history of prior hepatitis B immunization are eligible if other criteria are met (ie, negative tests for:
HBsAg, hepatitis B core antibody [HBcAb], and hepatitis C virus antibody [HCVAb]).
The patient has participated in a study involving an investigational drug within the past 30 days of the start of the trial.
The patient is unwilling to comply with the requirements of the protocol.
The patient is a sexually active man who is not willing to use 2 forms of birth control including a barrier method during the study.
The patient has history or ongoing clinically significant cardiac arrhythmia, defined as either atrial fibrillation, sustained or non-sustained ventricular tachycardia
The patient has any contraindication to magnetic resonance imaging (MRI).
The patient has one of the following central nervous system exclusion criteria:
Acute stroke, within 3 months of the screening visit.
History of seizures.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ402671 / SAR402671 in enzyme replacement therapy treatment-naïve adult male patients diagnosed with Fabry disease.;Secondary Objective: Not applicable;Primary end point(s): Change from baseline in globotriaosylceramide (GL-3) scores as evaluated by light microscopy (LM) in superficial skin capillary endothelium;Timepoint(s) of evaluation of this end point: 26 weeks

Secondary Outcome Measures

Name	Time	Method

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