Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

Completed · Phase 3

Conditions studied: Fabry Disease

In brief

This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients who had been treated for approximately 1 year with agalsidase beta and who had been on a stable dose of that product for at least 6 months were randomized in a 2:1 ratio to either switch to PRX-102 or to continue treatment with agalsidase beta. Both treatments were delivered by intravenous infusions every two weeks, at a dosage of 1 mg/kg.

Key facts

Study ID

NCT02795676

Run by

Protalix

People needed

78

Starts

2016-06-01

Expected to finish

2022-07-01

Last updated by the study team

2023-09-13

Who can join

Age: 18 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• i. neuropathic pain
• ii. cornea verticillata
• iii. clustered angiokeratoma
• Females:
• a. historical genetic test results consistent with Fabry pathogenic mutation and one or more of the described characteristic features of Fabry disease:
• i. neuropathic pain
• ii. cornea verticillata
• iii. clustered angiokeratoma
• b. 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
• i. 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 may not qualify 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

Where it is running

• UAB Medicine — Birmingham, Alabama, United States
• Phoenix Children's Hospital — Phoenix, Arizona, United States
• University of California Irvine Center — Orange, California, United States
• University of California San Diego — San Diego, California, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• University of Iowa Hosptials and Clinics — Iowa City, Iowa, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Infusion Associates — Grand Rapids, Michigan, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Institute of Metabolic Disease, Baylor Healthcare — Dallas, Texas, United States
• Renal Disease Research Institute, LLC - Dallas — Dallas, Texas, United States
• Eccles Primary Children's Outpatient Services Building — Salt Lake City, Utah, United States
• O+O Alpan LLC — Fairfax, Virginia, United States
• Medical College of Wisconsin — Milwaukee, Wisconsin, United States
• Vseobecna fakultni nemocnice v Praze — Prague, Czech Republic, Czechia
• Turku University Central Hospital — Turku, Finland
• Hôpital Raymond Poincaré — Paris, France
• Semmelweis Egyetem — Budapest, Hungary
• Azienda Ospedaliera Universitaria "Federico II" — Naples, Italy
• Academisch Medisch Centrum — Amsterdam, Netherlands
• Haukeland University Hospital Klinisk Forskningspost — Bergen, Norway
• General Hospital Slovenj Gradec — Slovenj Gradec, Slovenia
• Hospital de Dia Quiron Zaragoza — Zaragoza, Spain
• Klinik und Poliklinik für Innere Medizin UniversitätsSpital Zürich — Zurich, Switzerland

Full record on ClinicalTrials.gov

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