The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.
A Study of EZN-2279 (Polyethylene Glycol Recombinant Adenosine Deaminase [PEG-rADA]) Administered as a Weekly Intramuscular Injection in Patients With Adenosine Deaminase (ADA)-Deficient Combined Immunodeficiency
Diagnosis of ADA-deficient combined immunodeficiency
Stable clinical status while receiving therapy with Adagen®. Patients previously receiving gene therapy or undergoing hematopoietic stem cell transplantation who still require Adagen® treatment are eligible. The dose of Adagen® must be stable for at least 3 months prior to study entry.
Have both during the Adagen® Lead-in phase of the study:
Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen®
Total erythrocyte dAXP ≤0.02 μmol/mL from a trough blood sample
Patients or parent/guardian must be capable of understanding the protocol requirements and risks and providing written informed assent/consent