Summary

for people ages 18-60 (full criteria)
at San Diego, California and other locations
study started
estimated completion:
Bruce Barshop

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

  1. Females:
  2. historical genetic test results consistent with Fabry pathogenic mutation and one or more of the described characteristic features of Fabry disease:
  3. neuropathic pain

ii. cornea verticillata

iii. clustered angiokeratoma

  1. 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
  2. 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 San Diego accepting new patients
    San Diego California 92093 United States
  • University of California Irvine Center accepting new patients
    Orange California 92868 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Protalix
ID
NCT02795676
Phase
Phase 3
Lead Scientist
Bruce Barshop
Study Type
Interventional
Last Updated
January 19, 2018