Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

Study Identifier:
PB-102-F30
ClinicalTrials.gov Identifier:
NCT03018730
EudraCT Identifier:
N/A
EU CT ID:
N/A
Study Contact Information:
N/A
See Study Publication Below
Study Complete

Trial Documents

Protocol
Available Languages: English
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Statistical Analysis Plan
Available Languages: English
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Study Details

Medical Condition
  • Fabry Disease
Study Drug
  • Biological: PRX-102 (pegunigalsidase alfa)
Date
May 2017 - Dec 2019
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 60 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Age: 18-60 years
  • A documented diagnosis of Fabry disease
  • 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
  • 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
  • Treatment with agalsidase alfa for at least 2 years and on a stable dose (\>80% labelled dose/kg) for at least 6 months
  • eGFR ≥ 40 ml/min/1.73 m2 by CKD-EPI equation
  • Availability of at least 2 historical serum creatinine evaluations since starting agalsidase alfa treatment and not more than 2 years
  • 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
Exclusion Criteria
  • History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa
  • 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
  • Urine protein to creatinine ratio (UPCR) \> 0.5 g/g and not treated with an ACE inhibitor or ARB
  • Known history of hypersensitivity to Gadolinium contrast agent that was not managed by the use of premedication;
  • Females who are pregnant, planning to become pregnant during the study, or are breast feeding
  • Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before screening
  • Congestive heart failure NYHA Class IV
  • Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before screening
  • Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Monitor would interfere with the patient's compliance with the requirements of the study
Healthy Volunteers
No

Protocol 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.

Study Locations

Location
Status
Location
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Status
N/A
Location
Capital Health
Halifax, Nova Scotia, Canada, B3H 1V8
Status
N/A
Location
Vseobecna fakultni nemocnice v Praze
Prague, Czech Republic
Status
N/A
Location
Academisch Medisch Centrum
Amsterdam, Netherlands
Status
N/A
Location
Helse Bergen HF Haukeland Universitetssykehus
Bergen, Norway
Status
N/A
Location
General Hospital Slovenj Gradec
Slovenj Gradec, Slovenia, SI-2380
Status
N/A
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Study Publications

Azimpour K, Dorling P, Koulinska I, Kunduri S, Lan Z, Poritz J, Tremblay G, Raad-Faherty A. Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials. Adv Ther. 2025 Mar;42(3):1421-1434. doi: 10.1007/s12325-024-03095-2. Epub 2025 Jan 23.Linhart A, Dostalova G, Nicholls K, West ML, Tondel C, Jovanovic A, Giraldo P, Vujkovac B, Geberhiwot T, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Hughes D. Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study. Orphanet J Rare Dis. 2023 Oct 21;18(1):332. doi: 10.1186/s13023-023-02937-6.