Study Details

Testing the Drug Pegunigalsidase Alfa for Children and Teenagers with Fabry Disease

(IRB#: IRB_00180688)

Fabry disease is a rare genetic condition that keeps the body from breaking down certain fatty materials that build up in the body's cells. This can result in health issues such as pain in the hands and feet, damage to kidneys, brain and heart. This study will test a drug called Pegunigalsidase Alfa (Elfabrio) to treat children and teenagers with this disease. The drug has already been tested in adults with Fabry disease. The study wants to see if the drug will help children and teenagers with the disease and is safe. Participants will currently be in Stage I or Stage II of the study and be asked to continue participation. Participants who choose to continue will first attend in person visits for screening at the study clinic over about 6 weeks. Then if study criteria are met participants will attend in person visits at the study clinic for about 8 hours every two weeks to receive the study drug. Study participation will last up to 1 year. Medical tests will be done during the study to track the health of participants.

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