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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03018730




Registration number
NCT03018730
Ethics application status
Date submitted
9/01/2017
Date registered
12/01/2017
Date last updated
5/01/2021

Titles & IDs
Public title
Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
Scientific title
An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
Secondary ID [1] 0 0
PB-102-F30
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 - PRX-102 (pegunigalsidase alfa)

Experimental: PRX-102 - PRX-102 infusion every 2 weeks


Other interventions: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks

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

Outcomes
Primary outcome [1] 0 0
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Timepoint [1] 0 0
Throughout the 12 months study
Secondary outcome [1] 0 0
Mean annualised change in eGFR - Mean annualised change in eGFR
Timepoint [1] 0 0
Every 4 weeks for 12 months
Secondary outcome [2] 0 0
Left Ventricular Mass Index (g/m2) preferably by MRI - Left Ventricular Mass Index (g/m2) preferably by MRI (ECG can be used as an alternative)
Timepoint [2] 0 0
Every 6 months for 12 months
Secondary outcome [3] 0 0
Plasma Lyso-Gb3
Timepoint [3] 0 0
Every 3 months for 12 months
Secondary outcome [4] 0 0
Plasma Gb3
Timepoint [4] 0 0
Every 3 months for 12 months
Secondary outcome [5] 0 0
Urine Lyso-Gb3
Timepoint [5] 0 0
Every 3 months for 12 months
Secondary outcome [6] 0 0
Protein/Creatinine ratio - Protein/Creatinine ratio spot urine test
Timepoint [6] 0 0
Every 3 months for 12 months
Secondary outcome [7] 0 0
Frequency of pain medication use
Timepoint [7] 0 0
Every 2 weeks for 12 months
Secondary outcome [8] 0 0
Exercise tolerance (Stress Test)
Timepoint [8] 0 0
Every 6 months for 12 months
Secondary outcome [9] 0 0
Short Form Brief Pain Inventory (BPI)
Timepoint [9] 0 0
Every 3 months for 12 months
Secondary outcome [10] 0 0
Mainz Severity Score Index (MSSI)
Timepoint [10] 0 0
Every 6 months for 12 months
Secondary outcome [11] 0 0
Quality of life EQ-5D-5L
Timepoint [11] 0 0
Every 6 months for 12 months

Eligibility
Key inclusion criteria
1. Age: 18-60 years

2. A documented diagnosis of Fabry disease

3. Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less
than lower limit of normal according to laboratory range and one or more of the
characteristic features of Fabry disease i. Neuropathic pain ii. Cornea verticillata
iii. Clustered angiokeratoma

4. Females: historical genetic test results consistent with Fabry mutations, or in the
case of novel mutations a first degree male relative with Fabry disease, and one or
more of the characteristic features of Fabry disease i. Neuropathic pain ii. Cornea
verticillata iii. Clustered angiokeratoma

5. Treatment with agalsidase alfa for at least 2 years and on a stable dose (>80%
labelled dose/kg) for at least 6 months

6. eGFR = 40 ml/min/1.73 m2 by CKD-EPI equation

7. Availability of at least 2 historical serum creatinine evaluations since starting
agalsidase alfa treatment and not more than 2 years

8. 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
Minimum age
18 Years
Maximum age
60 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa

2. History of renal dialysis or transplantation

3. History of acute kidney injury in the 12 months prior to screening, including specific
kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic
renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as
extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive
nephropathy)

4. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB)
therapy initiated or dose changed in the 4 weeks prior to screening

5. Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE
inhibitor or ARB

6. Known history of hypersensitivity to Gadolinium contrast agent

7. Females who are pregnant, planning to become pregnant during the study, or are breast
feeding

8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period
before screening

9. Congestive heart failure NYHA Class IV

10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before
screening

11. 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

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
Completed
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
Canada
State/province [1] 0 0
Nova Scotia
Country [2] 0 0
Czechia
State/province [2] 0 0
Prague
Country [3] 0 0
Germany
State/province [3] 0 0
Wurzburg
Country [4] 0 0
Netherlands
State/province [4] 0 0
Amsterdam
Country [5] 0 0
Norway
State/province [5] 0 0
Bergen
Country [6] 0 0
Slovenia
State/province [6] 0 0
Slovenj Gradec
Country [7] 0 0
Spain
State/province [7] 0 0
Zaragoza
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Birmingham
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Cambridge
Country [10] 0 0
United Kingdom
State/province [10] 0 0
London
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Salford

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

Ethics approval
Ethics application status

Summary
Brief summary
This is an open label switch over study to assess the safety and efficacy of PRX-102
(pegunigalsidase alfa). Patients treated with agalsidase alfa for at least 2 years and on a
stable dose (>80% labelled dose/kg) for at least 6 months. Patients will be screened and
evaluated over 3 months while continuing on agalsidase alfa. Following the screening period,
the patient will be enrolled and switched from their agalsidase alfa treatment to receive
intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. No more than 25%
of treated patients will be female.
Trial website
https://clinicaltrials.gov/show/NCT03018730
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Raul Chertkoff, MD
Address 0 0
Protalix Ltd.
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
Other publications