Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

Study Identifier:
PB-102-F90
ClinicalTrials.gov Identifier:
NCT04552691
EudraCT Identifier:
N/A
Study Contact Information:
N/A
Recruitment Complete

Study Details

Medical Condition
  • Fabry Disease
Study Drug
  • Drug: Pegunigalsidase Alfa
Date
N/A - N/A
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Healthy Volunteers
No

Protocol Summary

The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and a) cannot be adequately treated with currently approved FDA products and/or b) are not able or willing to participate in any of the on-going clinical trials in the United States.

Study Locations

Location
Status
Location
University of Alabama-Birmingham
Birmingham, Alabama, United States, 35294
Status
N/A
Location
Phoenix Children's Hospital, Inc.
Phoenix, Arizona, United States, 85006
Status
N/A
Location
University of California Irvine
Orange, California, United States, 92868
Status
N/A
Location
Central Coas Nephrology
Salinas, California, United States, 93901
Status
N/A
Location
University of Florida, Division of Pediatric Genetics
Jacksonville, Florida, United States, 32207
Status
N/A
Location
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Status
N/A