Clinical Research Details

Clinical Research

Expanded Access Treatment with Open-Label Pegunigalsidase Alfa for Fabry Patients

Study Description

The purpose of this treatment program is to provide access to pegunigalsidase alfa to patients with Fabry disease whose clinical condition, in the opinion of their Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and are not able to participate in one of the ongoing clinical trials with pegunigalsidase alfa in the United States.

Pegunigalsidase alfa is an investigational product. An investigational product is one that is not yet approved by the United States Food and Drug Administration (FDA).

Pegunigalsidase alfa is given by IV infusion every 2 weeks. The drug is provided by the Sponsor. You will have routine exams, blood and urine tests, EKGs, and Echocardiograms. You will also complete questionnaires to let the Sponsor know how you feel. You will provide additional blood for tests to monitor the drug in your body. These blood draws will occur at the same time as the routine blood tests.

You may be able to stay on the program until the drug is commercially available or it is no longer in your best interest to remain on the investigational product. It is anticipated that your participation will be up to 5 years.

Open Enrollment

Contact Name: Amanda Prince
Contact Phone: (904) 427-7079
Contact Email: amanda.prince@jax.ufl.edu

Investigators


Co Investigator
Pamela L. Trapane, M.D.
Pediatrics

Principal Investigator
Anatalia Labilloy, M.D., Ph.D., M.P.H.
Pediatrics