Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Completed · Phase 3

Conditions studied: Fabry Disease

In brief

The objective of CLI-06657AA1-04 (formerly PB-102-F60) was to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who had successfully completed studies PB-102-F20, PB-102-F30, or at least 48 months in study PB-102-F03.

Key facts

Study ID

NCT03566017

Run by

Chiesi Farmaceutici S.p.A.

People needed

97

Starts

2018-09-16

Expected to finish

2025-01-21

Last updated by the study team

2026-03-24

Who can join

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

You may qualify if…

• Completion of study PB-102-F20, study PB-102-F30, or at least 48 months in study PB-102-F03.
• The participant signed informed consent.
• Female participants and male participants whose co-partners were of child-bearing potential agreed to use a medically acceptable method of contraception. These included 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, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or sexual abstinence. Contraception had to 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
• Phoenix Children's Hospital — Phoenix, Arizona, United States
• University of California San Diego — La Jolla, California, United States
• University of California Irvine Center — Orange, California, United States
• University of Florida — Gainesville, Florida, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
• Infusion Associates — Grand Rapids, Michigan, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Renal Disease Research Institute, LLC - Dallas — Dallas, Texas, United States
• Institute of Metabolic Disease — Dallas, Texas, United States
• University of Utah Hospitals & Clinics — Salt Lake City, Utah, United States
• O+O Alpan LLC — Fairfax, Virginia, United States
• Medical College of Wisconsin — Milwaukee, Wisconsin, United States
• Royal Melbourne Hospital — Parkville, Victoria, Australia
• Capital District Health Authority — Halifax, Nova Scotia, Canada
• Vseobecna fakultni nemocnice v Praze — Prague, Czech Republic, Czechia
• Turku University Central Hospital — Turku, Finland
• Hospital Raymond-Poincaré — Garches, France
• Semmelweis Egyetem — Budapest, Hungary
• Azienda Ospedaliera Universitaria "Federico II" — Naples, Via Pansini, Italy
• Academisch Medisch Centrum — Amsterdam, Netherlands
• Haukeland University Hospital Klinisk Forskningspost — Bergen, Norway
• General Hospital Slovenj Gradec — Slovenj Gradec, Slovenia

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.