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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03566017
Ethics application status
Date submitted
24/05/2018
Date registered
21/06/2018
Date last updated
20/05/2025

Titles & IDs
Public title
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks 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
CLI-06657AA1-04
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(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - pegunigalsidase alfa

Experimental: Experimental open label - pegunigalsidase alfa


Treatment: Drugs: pegunigalsidase alfa
Recombinant human alpha galactosidase A
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Evaluation of treatment-related adverse events
Assessment method [1] 0 0
CTCAE v4.03
Timepoint [1] 0 0
Throughout the study, 7 years
Secondary outcome [1] 0 0
Kidney function 1
Assessment method [1] 0 0
Estimated glomerular filtration rate (eGFRCKD-EPI)
Timepoint [1] 0 0
Every 6 months throughout trial, 7 years
Secondary outcome [2] 0 0
Cardiac assessment
Assessment method [2] 0 0
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 to end of the study, 7 years
Secondary outcome [3] 0 0
Biomarkers for Fabry disease
Assessment method [3] 0 0
plasma Lyso-Gb3 and Gb3
Timepoint [3] 0 0
Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months.
Secondary outcome [4] 0 0
Record of pain medication use
Assessment method [4] 0 0
Frequency of pain medication use, or pre-infusion medication
Timepoint [4] 0 0
Every two weeks for 7 years
Secondary outcome [5] 0 0
Kidney function 2
Assessment method [5] 0 0
Protein/Creatinine ratio, spot urine test (UPCR)
Timepoint [5] 0 0
Every 6 months to the end of the study, 7 years
Secondary outcome [6] 0 0
Pain assessment
Assessment method [6] 0 0
short form Brief Pain Inventory (BPI)
Timepoint [6] 0 0
Every 6 months up to the end of the study, 7 years
Secondary outcome [7] 0 0
Symptom assessment
Assessment method [7] 0 0
Mainz Severity Score Index (MSSI)
Timepoint [7] 0 0
Every 12 months up to the end of the study, 7 years
Secondary outcome [8] 0 0
Quality of life assessment
Assessment method [8] 0 0
quality of life (EQ-5D-5L)
Timepoint [8] 0 0
Every 6 months up to the end of the study, 7 years

Eligibility
Key inclusion criteria
1. Completion of study PB-102-F20, PB-102-F03, 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. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.
Minimum age
18 Years
Maximum age
60 Years
Sex
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, would interfere with patient compliance with the requirements of the study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/09/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
21/01/2025
Sample size
Target
Accrual to date
Final
97
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
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
United States of America
State/province [11] 0 0
Utah
Country [12] 0 0
United States of America
State/province [12] 0 0
Virginia
Country [13] 0 0
United States of America
State/province [13] 0 0
Wisconsin
Country [14] 0 0
Canada
State/province [14] 0 0
Nova Scotia
Country [15] 0 0
Czechia
State/province [15] 0 0
Czech Republic
Country [16] 0 0
Finland
State/province [16] 0 0
Turku
Country [17] 0 0
France
State/province [17] 0 0
Garches
Country [18] 0 0
Hungary
State/province [18] 0 0
Budapest
Country [19] 0 0
Italy
State/province [19] 0 0
Via Pansini
Country [20] 0 0
Netherlands
State/province [20] 0 0
Amsterdam
Country [21] 0 0
Norway
State/province [21] 0 0
Bergen
Country [22] 0 0
Slovenia
State/province [22] 0 0
Slovenj Gradec
Country [23] 0 0
Spain
State/province [23] 0 0
Zaragoza
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Birmingham
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Greater Manchester
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Cambridge
Country [27] 0 0
United Kingdom
State/province [27] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Chiesi Farmaceutici S.p.A.
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT03566017