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

• Register: EUCTR
• Last refreshed on: 8 August 2022
• Main ID: EUCTR2014-005544-18-ES
• Date of registration: 16/02/2015
• Prospective Registration: Yes
• Primary sponsor: Protalix Ltd.
• Public title: Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients
• Scientific title: A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients
• Date of first enrolment: 29/04/2015
• Target sample size: 18
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-005544-18
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Paraguay, Spain, United Kingdom, United States

Contacts

• Name: Unidad de Puesta en Marcha
• Affiliation: SERMES PLANIFICACION S.L

• Name: Unidad de Puesta en Marcha
• Affiliation: SERMES PLANIFICACION S.L

Key inclusion & exclusion 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:
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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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

Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Intervention(s)

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)/millilitre
Concentration type: equal
Concentration number: 2-

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)/millilitre
Concentration type: equal
Concentration number: 2-

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)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

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.

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

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.

Secondary Objective: Not applicable

Secondary Outcome(s)

Secondary end point(s): Not applicable

Timepoint(s) of evaluation of this end point: Not applicable

Secondary ID(s)

PB-102-F03

Source(s) of Monetary Support

Protalix Ltd.

Ethics review

Status: Approved
Approval date: 15/04/2015
Contact:

Results

• Results available: Yes
• Date Posted: 18/04/2022
• Date Completed: 26/08/2020
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-005544-18/results