Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients

A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-005544-18-ES

Enrollment

Registered

2015-02-16

Start date

2015-04-29

Completion date

Unknown

Last updated

2022-08-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fabry disease MedDRA version: 18.0 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: PRX102 Product Code: PRX102 Pharmaceutical Form: Infusion INN or Proposed INN: PRX102 CAS Number: 1333358-30-7 Current Sponsor code: PRX102 Concentration unit: mg/ml milligram(s)/millili

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Completion of study PB-102-F02 2. The patient signs informed consent 3. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used for 1 month after termination of treatment. 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 17 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1

Exclusion criteria

Exclusion criteria: 1. Pregnant or nursing 2. Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient’s compliance with the requirements of the study

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the ongoing safety, tolerability and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02- and are continuing to receive treatment at the dose each patient reeceived in study PB-102-F02.;Secondary Objective: Not applicable;Primary end point(s): Efficacy Variables The efficacy endpoints evaluated in Study PB-102-F02 will continue to be measured in this extension study. Baseline values for this extension study will be the values from the last infusion of study PB-102-F02. Safety Variables: Safety will be assessed by the frequency, severity, and duration of treatment-emergent adverse events (TEAEs), including clinically significant laboratory abnormalities, ECG changes from baseline, physical examination findings, assessment of the injection site reactions and existence of Anti PRX-102 antibodies after administration of the study drug.;Timepoint(s) of evaluation of this end point: Efficacy Variables Gb3 and Lyso-Gb3 concentrations in plasma: baseline/every 3 months Gastrointestinal symptoms, Kidney functions, BPI: baseline/every 3 months Gb3 concentration in kidney and in skin: last treatment visit Left ventricular mass and myocardial fibrosis: baseline/ every 12 months Cardiac function and stress test: baseline/every 12 months Cerebrovascular disease: baseline/last treatment of study MSSI: baseline/every 6 months Safety Variables Laboratory values and physical examination: screening and visits 7, 14, 20, 27, 33, 40, 46, 53 ECG: screening and visits 14, 40, 53 Anti PRX-102 antibodies: screening and visits 5, 7, 14, 20, 27, 33, 40,46,53 and 3 months after last infusion AE: every study visit Injection site reactions: Every study treatment visit	—

Measure

Time frame

Main Objective: To evaluate the ongoing safety, tolerability and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02- and are continuing to receive treatment at the dose each patient reeceived in study PB-102-F02.;Secondary Objective: Not applicable;Primary end point(s): Efficacy Variables The efficacy endpoints evaluated in Study PB-102-F02 will continue to be measured in this extension study. Baseline values for this extension study will be the values from the last infusion of study PB-102-F02. Safety Variables: Safety will be assessed by the frequency, severity, and duration of treatment-emergent adverse events (TEAEs), including clinically significant laboratory abnormalities, ECG changes from baseline, physical examination findings, assessment of the injection site reactions and existence of Anti PRX-102 antibodies after administration of the study drug.;Timepoint(s) of evaluation of this end point: Efficacy Variables Gb3 and Lyso-Gb3 concentrations in plasma: baseline/every 3 months Gastrointestinal symptoms, Kidney functions, BPI: baseline/every 3 months Gb3 concentration in kidney and in skin: last treatment visit Left ventricular mass and myocardial fibrosis: baseline/ every 12 months Cardiac function and stress test: baseline/every 12 months Cerebrovascular disease: baseline/last treatment of study MSSI: baseline/every 6 months Safety Variables Laboratory values and physical examination: screening and visits 7, 14, 20, 27, 33, 40, 46, 53 ECG: screening and visits 14, 40, 53 Anti PRX-102 antibodies: screening and visits 5, 7, 14, 20, 27, 33, 40,46,53 and 3 months after last infusion AE: every study visit Injection site reactions: Every study treatment visit

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Secondary

Measure	Time frame
Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable	—

Countries

Paraguay, Spain, United Kingdom, United States

Contacts

Public ContactUnidad de Puesta en Marcha

SERMES PLANIFICACION S.L

start-up@sermescro.com+34913275025

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026