Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

ConditionFabry Disease

Trial statusRecruiting

Trial phasePhase 2, Phase 3

Trial typeInterventional

Biological sexAll

Age13-70

SponsorChiesi Farmaceutici S.p.A.

About this trial

The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of approximately 16 male and female Fabry disease patients between the ages of 13 and 70 years to be part of the study. The study is conducted in Japan.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Must have been born in Japan and have their biological parents and all 4 grandparents of Japanese descent

Males: Plasma and/or leukocyte alpha-galactosidase-A activity (by activity assay) that is ≤ 5% of mean normal laboratory levels or, if the enzymatic activity is above the 5% limit but still under the normal level, a confirmed disease-causing mutation of the GLA gene

Females: Historical genetic test results consistent with Fabry mutations or, in the case of novel mutations, a first-degree male relative with Fabry disease

All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma

Disqualifiers

Administration of ERT for Fabry disease within 14 days before baseline, substrate reduction therapy for Fabry disease within 3 days before baseline, or chaperone therapy for Fabry disease within 3 days before baseline

History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or to any component of the study drug

Cohort A only: eGFR value of >90 to ≤120 mL/min/1.73 m2 at screening and a historical eGFR value >120 mL/min/1.73 m2 in the 9 to 24 months before screening, indicating absence of renal impairment. eGFR to be calculated using the JPN-CKD-EPI creatinine equation (2009).

Urine protein to creatinine ratio (UPCR) >0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB

Trial design

Design model

Single group

Treatments tested in this trial

PRX-102 1 mg/kg every 2 weeks
Drug
PRX-102 1 mg/kg every 2 weeks
PRX-102 2 mg/kg every 4 weeks
Drug
PRX-102 2 mg/kg every 4 weeks

Treatment groups

16 Participants

are divided into 1 treatment group

Group A: PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeksExperimental treatment 2 interventions