An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II

A Phase 3, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)

The primary objective of this extension study is to assess the long-term safety and efficacy of AVTX-803 in subjects with LAD II (SLC35C1-CDG).

Inclusion Criteria:

• Subject must have completed protocol AVTX-803-LAD-301
• Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)

Exclusion Criteria:

• Subject has severe anemia defined as hemoglobin <8.0 g/dL (<4.9 mmol/L)
• Subject has impaired renal function as defined by an eGFR <90 mL/min
• Subject has known or suspected intolerance or hypersensitivity to fucose or any ingredients of the investigational product
• In the investigator's opinion, subject has a history of failure to respond to fucose at adequate dosing