A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency

Inclusion Criteria

• •Documented diagnosis of Arginase I deficiency
• •Adequate organ function: Hgb ≥ 10 g/dL, ANC ≥ 1.5 x 109/L, plt count ≥ 100,000/µL; liver transaminase levels ≤ 2.5x ULN, total bilirubin ≤ 2.0 mg/dL; serum creatinine \<1.5 x ULN
• •If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
• •If a sexually active (male or female), must be surgically sterile, post-menopausal (female), or must agree to use a physician-approved method of birth control during the study and for a minimum of 30 days after the last study drug administration
• •Patient or legal guardian is able and willing to provide written informed consent and to comply with all requirements of study participation (including all study procedures and continuation of prescribed diet without modification) prior to any screening procedures