Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
a study on Fabry Disease
Summary
- Eligibility
- for people ages 18-60 (full criteria)
- Location
- at Orange, California and other locations
- Dates
- study startedestimated completion
Description
Summary
This is a randomized, double blind, active control study of PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients treated for approximately 1 year with agalsidase beta and on a stable dose for at least 6 months will be screened and then randomized to continue treatment with 1mg/kg agalsidase beta or to treatment with 1 mg/kg of PRX-102. The identity of the enzyme will be blinded to the patient and the investigator. Patients will receive intravenous infusions every two weeks. Patients will be randomized in a 2:1 ratio of PRX-102 to agalsidase beta. Randomization will be stratified by urinary protein to creatinine ratio (UPCR) of < or ≥ 1 g/g by spot urine sample. No more than 50% of the patients will be female.
Official Title
A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta
Keywords
Fabry Disease Glomerular filtration rate Proteinuria PRX-102 pegunigalsidase alfa PRX-102 (pegunigalsidase alfa) agalsidase beta
Eligibility
You can join if…
Open to people ages 18-60
- Symptomatic adult Fabry disease patients, age 18-60 years
- Males: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than 30% mean normal levels and one or more of the characteristic features of Fabry disease
- neuropathic pain
ii. cornea verticillata
iii. clustered angiokeratoma
- Females:
- historical genetic test results consistent with Fabry pathogenic mutation and one or more of the described characteristic features of Fabry disease:
- neuropathic pain
ii. cornea verticillata
iii. clustered angiokeratoma
- or in the case of novel mutations a first degree male family member with Fabry disease with the same mutation, and one or more of the characteristic features of Fabry disease
- neuropathic pain
ii. cornea verticillata
iii. clustered angiokeratoma
- Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m²
- Linear negative slope of eGFR based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit) of ≥ 2 mL/min/1.73 m²/year
- Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months.
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.
You CAN'T join if...
- History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
- Known non-pathogenic Fabry mutations
- History of renal dialysis or transplantation
- 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)
- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
- Patient with a screening eGFR value between 91-120 mL/min/1.73 m², having an historical eGFR value higher than 120 mL/min/1.73 m² (during 9 to 18 months before screening)
- Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
- Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
- Congestive heart failure NYHA Class IV
- Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
- Known history of hypersensitivity to Gadolinium contrast agent that is not managed by the use of pre-medication
- Female subjects who are pregnant, planning to become pregnant during the study, or are breastfeeding
- 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
Locations
- University of California Irvine Center
Orange California 92868 United States - University of California Los Angeles
Los Angeles California 90024 United States
Details
- Status
- in progress, not accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- Protalix
- ID
- NCT02795676
- Phase
- Phase 3
- Study Type
- Interventional
- Last Updated