Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
Approved for marketing · NCT04552691
Conditions: Fabry Disease
At a glance
- Research payments reported, 2025
- $167,740
- U.S. investigators named
- 4
- Sites paid
- 4
About the trial
- Sponsor
- Chiesi Farmaceutici S.p.A. (with Chiesi USA, Inc.)
- Tested
- Pegunigalsidase Alfa (drug)
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2024-07-31. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Chiesi USA, Inc. reported $167,740 in research payments for this study in 2025 (6 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Virginia E. Kimonis, MD Medical Genetics · Orange, CA
- William Wilcox Medical Genetics · Decatur, GA
- Ankit N. Mehta, M.D. Nephrology (Kidney) · Dallas, TX
- Ozlem Goker-alpan, M.D. Pediatrics · Fairfax, VA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Lysosomal and Rare Disorders Research and Treatment Center Inc $95,379
- Renal Disease Research Institute $47,477
- Uci Health-Orange, CA $13,452
- Emory University Hospital, GA $11,431
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.