Expanded Access To Provide Pegzilarginase To Patients with Arginase 1 Deficiency on Individual INDs with Pegzilarginase
Pegzilarginase EAP
What is the goal of the study?
For patients deficient in arginase 1 there is a significant unmet need for a therapy that will lower arginine levels to within the normal range and promote the lifelong maintenance of normal arginine levels. This Expanded Access Program (EAP) through sIND will provide continued access to treatment with pegzilarginase for patients previously enrolled in interventional clinical trials CAEB1102-102A or CAEB1102-300A or previously approved sIND. This EAP will also provide treatment to naive patients who have not previously received pegzilarginase.The EAP is planned to continue until pegzilarginase is commercially available for patients with ARG1-D. Pegzilarginase is a cobalt-substituted, pegylated, recombinant human arginase 1. Pegzilarginase should be administered via intravenous (IV) infusion and may be switched to subcutaneous (SC) injection once dosing established, using the same dose.
Who can participate in the study?
Please contact the study team listed below to learn more.