Study PB-102-F50 PRX-102 in Fabry Disease Patients Treated with ERT

• Research type

  Research Study

• Full title

  A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy: Fabrazyme (agalsidase beta) or Replagal (agalsidase alfa)

• IRAS ID

  231231

• Contact name

  Derralynn Hughes

• Contact email

  rmgvdah@ucl.ac.uk

• Sponsor organisation

  Protalix Ltd.

• Eudract number

  2017-001528-23

• Duration of Study in the UK

  1 years, 7 months, 2 days

• Research summary

  Research Summary
  The purpose of this research study is to test the experimental drug called pegunigalsidase alfa (PRX-102) as an enzyme replacement therapy (ERT) for Fabry disease. Fabry disease is a rare, inherited disease caused by the deficiency of a certain enzyme which results in reduced kidney function. Patients who are currently being treated with the standard enzyme replacement therapies agalsidase alfa (Replagal™) or agalsidase beta (Fabrazyme®) will be invited to participate in the study.

  This is an open-label switchover study to assess the safety, efficacy, and pharmacokinetics (seeing how the body uses a drug) of pegunigalsidase alfa treatment of 2 mg/kg every 4 weeks in patients previously treated with ERT, agalsidase alfa or agalsidase beta, for at least 3 years and on a stable dose for at least the last 6 months. Following screening, patients will be enrolled and switched from their current ERT to receive intravenous (IV) infusions of pegunigalsidase alfa 2 mg/kg every 4 weeks for 52 weeks (total of 14 infusions).

  Approximately 30 participants will take part in the study globally, with a target of 12 participants to be recruited in the UK amongst up to 4 UK research sites.

  Summary of Results
  The PB-102-F50, BRIGHT, study was a phase III multicenter, multinational, open-label, switch-over study designed to evaluate the safety, efficacy and pharmacokinetics of pegunigalsidase alfa treatment, 2 mg/kg every 4 weeks for one year in up to 30 adult patients with Fabry disease who have previously received a commercially available enzyme replacement therapy (ERT) for at least three years and on a stable dose (agalsidase alfa – Replagal® or agalsidase beta – Fabrazyme®).

• REC name

  London - Westminster Research Ethics Committee

• REC reference

  17/LO/1350

• Date of REC Opinion

  18 Oct 2017

• REC opinion

  Further Information Favourable Opinion