Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

• Register: EUCTR
• Last refreshed on: 28 February 2019
• Main ID: EUCTR2018-001148-67-ES
• Date of registration: 16/11/2018
• Prospective Registration: Yes
• Primary sponsor: Protalix Ltd.
• Public title: Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
• Scientific title: Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
• Date of first enrolment: 08/02/2019
• Target sample size: 110
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-001148-67
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, Denmark, Germany, Hungary, Italy, Netherlands, Norway, Slovenia, Spain, Switzerland, Turkey, United Kingdom, United States

Contacts

• Name: Raul Chertkoff
• Affiliation: Protalix Ltd.

• Name: Raul Chertkoff
• Affiliation: Protalix Ltd.

Key inclusion & exclusion criteria

Inclusion criteria:
1. Completion of study PB-102-F20 or PB-102-F30
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 treatment termination.
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 110
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with patient 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 (a-galactosidase A deficiency)
MedDRA version: 20.0 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Intervention(s)

Product Name: Pegunigalsidase alfa
Product Code: PRX-102
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: PEGUNIGALSIDASE ALFA
CAS Number: 1644392-61-9
Current Sponsor code: PRX-102
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

Primary end point(s): SAFETY ENDPOINTS:
Changes from baseline in:
*Clinical laboratory tests
*Physical examination
*Assessment of the injection site
*Electrocardiography (ECG)
*Treatment-emergent adverse events (TEAE)
*Ability to taper off infusion pre-medication at the start of the study
*Requirement for use of pre-medication overall to manage infusion reactions
*Treatment-emergent anti-pegunigalsidase alfa antibodies

Main Objective: To evaluate the ongoing safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa (PRX-102) every other week in adult Fabry patients who have successfully completed studies PB-102-F20 or PB-102-F30.

Secondary Objective: N/A

Timepoint(s) of evaluation of this end point: Patients who received at least one dose of study drug will be included in the safety analysis. Results of safety evaluations (including TEAEs, injection site reactions, physical examinations, ECG, and laboratory analyses, anti-drug antibodies) will be summarized and described.
No formal statistical analyses will be performed. The study endpoints will be evaluated by various summary analyses by study visit and by change from baseline data for each efficacy endpoint. Safety and efficacy endpoints will be compared to baseline using summary statistics. Data will not be analysed by inferential statistics.
An interim analysis may be performed for administrative purposes during the course of the study; available efficacy and safety parameters will be summarized using descriptive statistics.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: N/A

Secondary end point(s): EFFICACY ENDPOINTS:
*Estimated glomerular filtration rate (eGFRCKD-EPI)
*Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI)
*Plasma Gb3
*Plasma Lyso-Gb3
*Urine Lyso-Gb3
*Protein/Creatinine ratio, spot urine test (UPCR)
*Frequency of pain medication use
*Exercise tolerance (Stress Test)
*Short Form Brief Pain Inventory (BPI)
*Mainz Severity Score Index (MSSI)
*Quality of life (EQ-5D-5L)
*Clinical events

Secondary ID(s)

PB-102-F60

2018-001148-67-GB

Source(s) of Monetary Support

Protalix Ltd.

Ethics review

Status: Approved
Approval date:
Contact: