Evaluating AVTX-803 for treating Leukocyte Adhesion Deficiency Type II
A Phase 3, Randomized, Double-blind, Two-period, Crossover, Withdrawal Study to Assess the Efficacy and Safety of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)
This study is testing a new treatment called AVTX-803 to see if it can help people with Leukocyte Adhesion Deficiency Type II have fewer infections and feel better.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 4 (estimated) |
| Ages | 6 Months to 75 Years |
| Sex | All |
| Sponsor | AUG Therapeutics Industry-sponsored |
| Locations | 1 site (Rochester, Minnesota) |
| Trial ID | NCT05462587 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the efficacy and safety of AVTX-803, a treatment involving L-Fucose, in patients diagnosed with Leukocyte Adhesion Deficiency Type II (LAD II). Participants will be compared to a control group that will undergo withdrawal from their current treatment. The study will include individuals aged 6 months to 75 years who have a confirmed diagnosis of LAD II and a history of recurrent infections. The goal is to determine if AVTX-803 can improve patient outcomes and reduce infection rates.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 6 months to 75 years with biochemically and genetically confirmed LAD II and a history of recurrent infections.
Not a fit: Patients without a confirmed diagnosis of LAD II or those who do not have a history of recurrent infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life and health outcomes for patients with Leukocyte Adhesion Deficiency Type II.
How similar studies have performed: While this specific approach is novel, similar studies targeting genetic deficiencies have shown promise in improving patient outcomes.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subject must be between 6 months and 75 years old * Subject has biochemically and genetically proven LAD II (SLC35C1-CDG) * Subject has a documented history of Lewis antigen deficiency * Subject has a history of recurrent infections, opportunistic infections or infections that did not respond well to standard of care treatment * Subject or parent (for subjects under legal age for consent) has provided written informed consent for this study. Additionally, written informed assent has been provided, as appropriate, for minors of older age, per local institutional review board (IRB)/ethics committee (EC) policy and requirements * Subject is willing and able to comply with the protocol * Women of childbearing potential (WOCBP) meeting the criteria below: 1. Non-lactating and has a negative pregnancy test at screening -AND- 2. Uses an acceptable double-barrier method of contraception as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug. * Male subjects must agree to use an acceptable double-barrier method of contraception with their partner as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug. 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 a total absence of fucosylation on red blood cells and the presence of anti-H antigen * 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 * In the investigator's opinion, subject is not able or not willing to comply with the study requirements. * Subject is pregnant
Where this trial is running
Rochester, Minnesota
- Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
Study contacts
- Principal investigator: David Deyle, MD — 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.