Long-term safety and efficacy of AVTX-803 for 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)
This study is testing a new drug called AVTX-803 to see if it is safe and effective for people with Leukocyte Adhesion Deficiency Type II over a long period of time.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 2 (estimated) |
| Ages | 6 Months to 75 Years |
| Sex | All |
| Sponsor | AUG Therapeutics Industry-sponsored |
| Locations | 1 site (Rochester, Minnesota) |
| Trial ID | NCT05754450 on ClinicalTrials.gov |
What this trial studies
This extension study aims to evaluate the long-term safety and effectiveness of the investigational drug AVTX-803 in patients diagnosed with Leukocyte Adhesion Deficiency Type II (LAD II). Participants must have previously completed a related protocol and have confirmed biochemical and genetic evidence of the condition. The study will monitor participants for any adverse effects and assess the drug's efficacy over an extended period.
Who should consider this trial
Good fit: Ideal candidates are individuals who have completed the AVTX-803-LAD-301 protocol and have a confirmed diagnosis of LAD II.
Not a fit: Patients with severe anemia, impaired renal function, or known intolerance to fucose may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option that improves the health and quality of life for patients with LAD II.
How similar studies have performed: While this study builds on previous research, the specific approach with AVTX-803 in this context is novel and has not been extensively tested in similar studies.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Rochester, Minnesota
- Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
Study contacts
- Principal investigator: David Deyle — Mayo Clinic
- Study coordinator: Jennifer Lin
- Email: huiyil@Augtx.com
- Phone: 3052833827
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.