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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03566017




Registration number
NCT03566017
Ethics application status
Date submitted
24/05/2018
Date registered
21/06/2018
Date last updated
21/11/2018

Titles & IDs
Public title
Extension Study of 1 mg/kg Pegunigalsidase Alfa 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
Secondary ID [1] 0 0
PB-102-F60
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - pegunigalsidase alfa

Experimental: Experimental open label - pegunigalsidase alfa


Other interventions: pegunigalsidase alfa
Recombinant human alpha galactosidase A

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Evaluation of treatment-related adverse events - CTCAE v4.03
Timepoint [1] 0 0
Through study completion, 4 years
Secondary outcome [1] 0 0
Kidney function - Estimated glomerular filtration rate (eGFRCKD-EPI)
Timepoint [1] 0 0
Once a month for the first 12 months, then every 3 months until the end of the study, 4 years
Secondary outcome [2] 0 0
Cardiac assessment - Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)
Timepoint [2] 0 0
Every 12 months up to the end of the study, 4 years
Secondary outcome [3] 0 0
Biomarkers for Fabry disease - Plasma Gb3, plasma Lyso-Gb3, and urine Lyso-Gb3
Timepoint [3] 0 0
Every 3 months the first 12 months and every 6 months up to the end of the study, 4 years
Secondary outcome [4] 0 0
Record of pain medication use - Frequency of pain medication use, or pre-infusion medication
Timepoint [4] 0 0
Every two weeks for 4 years
Secondary outcome [5] 0 0
Kidney function - Protein/Creatinine ratio, spot urine test (UPCR)
Timepoint [5] 0 0
Every 3 months the first 12 months and every 6 months up to the end of the study, 4 years
Secondary outcome [6] 0 0
Pain assessment - short form Brief Pain Inventory (BPI)
Timepoint [6] 0 0
Every 6 months up to the end of the study, 4 years
Secondary outcome [7] 0 0
Symptom assessment - Mainz Severity Score Index (MSSI)
Timepoint [7] 0 0
Every 6 months up to the end of the study, 4 years
Secondary outcome [8] 0 0
Quality of life assessment - quality of life (EQ-5D-5L)
Timepoint [8] 0 0
Every 6 months up to the end of the study, 4 years

Eligibility
Key inclusion criteria
- Completion of study PB-102-F20 or PB-102-F30

- The patient signs informed consent

- 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.
Minimum age
18 Years
Maximum age
60 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Utah
Country [10] 0 0
United States of America
State/province [10] 0 0
Virginia
Country [11] 0 0
Canada
State/province [11] 0 0
Nova Scotia
Country [12] 0 0
Czechia
State/province [12] 0 0
Czech Republic
Country [13] 0 0
Hungary
State/province [13] 0 0
Budapest
Country [14] 0 0
Netherlands
State/province [14] 0 0
Amsterdam
Country [15] 0 0
Norway
State/province [15] 0 0
Bergen
Country [16] 0 0
Slovenia
State/province [16] 0 0
Slovenj Gradec
Country [17] 0 0
Spain
State/province [17] 0 0
Zaragoza
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Cambridge
Country [19] 0 0
United Kingdom
State/province [19] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Protalix
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The objective of PB-102-F60 is to evaluate the long-term safety, tolerability, and efficacy
parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in
adult Fabry patients who have successfully completed studies PB-102-F20 or PB-102-F30.
Trial website
https://clinicaltrials.gov/show/NCT03566017
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03566017