Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Completed · Phase 3

Conditions studied: Fabry Disease

In brief

The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.

Key facts

Study ID

NCT03614234

Run by

Chiesi Farmaceutici S.p.A.

People needed

29

Starts

2018-11-13

Expected to finish

2026-04-13

Last updated by the study team

2026-05-22

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Completion of study PB-102-F50.
• The patient signs informed consent.
• Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.

You may not qualify if…

• Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.

Where it is running

• UAB Medicine — Birmingham, Alabama, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• University of Iowa Hospitals and Clinica — Iowa City, Iowa, United States
• Infusion Associates — Grand Rapids, Michigan, United States
• Renal Disease Research Institute, LLC — Dallas, Texas, United States
• University of Utah Hospitals & Clinics — Salt Lake City, Utah, United States
• O & O Alpan — Fairfax, Virginia, United States
• UZ Antwerpen — Edegem, Belgium
• Fakultní poliklinika Všeobecné fakultní nemocnice v Praze — Prague, Czechia
• Medical Endocrinology PE 2132, Rigshospitalet — Copenhagen, Denmark
• Azienda Ospedaliera Universitaria "Federico II" — Naples, Via Pansini, Italy
• Helse Bergen HF Haukeland Universitetssykehus — Bergen, Norway
• Addenbrooke's Hospital — Cambridge, United Kingdom
• The Royal Free Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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