An Extension of the PB-102-F02 study with PRX-102 in adult patients with Fabry Disease.

• Register: EUCTR
• Last refreshed on: 9 November 2020
• Main ID: EUCTR2014-005544-18-GB
• Date of registration: 17/08/2015
• Prospective Registration: Yes
• Primary sponsor: Protalix Ltd.
• Public title: An Extension of the PB-102-F02 study with PRX-102 in adult patients with Fabry Disease.
• Scientific title: A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients. - PB102F03: Extension Study of PRX-102 in Fabry Disease
• Date of first enrolment: 16/09/2015
• Target sample size: 16
• 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: no Placebo: no Other: no
• 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: Rainer Schuckelt
• Affiliation: CATO Europe GmbH

• Name: Rainer Schuckelt
• Affiliation: CATO Europe GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
Eligible patients must fulfill the following 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 must 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: 0
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:
The presence of any of the following excludes a patient from study enrollment:
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: PRX-102
Product Code: PRX-102
Pharmaceutical Form: Infusion
INN or Proposed INN: PRX-102
CAS Number: 1333358-30-7
Current Sponsor code: PRX-102
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: - Clinical lab tests and physical examination will be assessed at screening and then every 3 months for 24 months.
- ECG will be performed every 6 months, and visit 53(month 24).
- Vital Signs, adverse events and injection site reactions will be assessed at every visit.
- Vital signs will be assessed at the screening visit and visit 1 up to visit 53.
- Anti-PRX-102 antibodies will be assessed at Screening, visit 5 (month 2), visit 7 (month 3), visit 14 (month 6), visit 20 (month 9), visit 27 (month 12), visit 33 (month 15), visit 40 (mnonth 18), visit 46 (month 21), visit 53 (month 24) and 3 months after last infusion.

Secondary Objective: All exploratory efficacy endpoints that were evaluated during the PB-102-F01 study and its first extension (PB-102-F02) will continue to be assessed in this second extension study PB-102-F03. These include:

- Globotriaosylceramide (Gb3) concentration in kidney (assessed histologically in kidney biopsy).
- Gb3 concentration in skin (assessed histologically in skin punch biopsy)
- Gb3 concentrations in plasma and urine sediment
- Globotriaosylsphingosine (Lyso-Gb3) concentration in plasma
- Assessment of gastrointestinal symptoms
- Kidney functions (eGFR and proteinuria)
- Short Form Brief Pain Inventory (BPI)
- Left ventricular mass (LVM) and myocardial fibrosis assessment by cardiac MRI
- Cardiac function assessment by echocardiography and stress test
- Cerebrovascular disease assessment (clinical and MRI evaluation)
- Mainz Severity Score Index (MSSI)

Primary end point(s): The main outcome will be evaluation of safety in PRX-102 treated subjects at each dose. 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 and assessment of the injection site reactions after administration of the study drug. Also Anti-PRX-102 antibodies will be assessed.

Main Objective: To evaluate the ongoing safety and tolerability of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in the PB-102-F01 study and its first extension PB-102-F02.

Secondary Outcome(s)

Secondary end point(s): The following 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.

- Gb3 concentrations in plasma and urine sediment
- Globotriaosylsphingosine (Lyso-Gb3) concentration in plasma
- Assessment of gastrointestinal symptoms
- Kidney functions (eGFR and proteinuria)
- Short Form Brief Pain Inventory (BPI)
- Gb3 concentration in kidney (assessed histologically in kidney biopsy samples)
- Gb3 concentration in skin (assessed histologically in skin biopsy samples)
- Left ventricular mass and myocardial fibrosis assessment by cardiac MRI
- Cardiac function (assessed by echocardiography) and stress test
- Cerebrovascular disease (clinical and MRI evaluation)
- Mainz Severity Score Index (MSSI)

Timepoint(s) of evaluation of this end point: - Gb3 (plasma & urine) at baseline and every 3 months during the study
- Lyso-Gb3 concentration at baseline and every 3 months during the study
- Assessment of gastrointestinal symptoms at baseline and every 3 months during the study
- eGFR and proteinuria at baseline and every 3 months during the study
- BPI at baseline and every 3 months during the study
- Gb3 (kidney and skin) at last visit of this study
- Cardiac MRI at baseline and every 12 months during the study
- Echocardiography and stress test at baseline and every 12 months during the study
- Cerebrovascular disease at baseline and at last visit of this study (total treatment of 3 years)
- MSSI at baseline and every 6 months during this study

Secondary ID(s)

2014-005544-18-ES

PB-102-F03

NCT01981720

Source(s) of Monetary Support

Protalix Ltd.

Ethics review

Status: Approved
Approval date: 16/09/2015
Contact: