Study detail
RecruitingPhase 3
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)
AUG Therapeutics
Summary
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).
Eligibility
- Age range
- 0–75 years
- Sex
- All
- Healthy volunteers
- No
Detailed criteria
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
Interventions
- DrugAVTX-803
L-fucose crystalline powder
Location
- Mayo ClinicRochester, Minnesota