AVTX-803 for Leukocyte Adhesion Deficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines AVTX-803, a potential new drug for individuals with Leukocyte Adhesion Deficiency type II (LAD II), a rare immune disorder. The trial aims to assess the long-term safety and effectiveness of AVTX-803. Participants must have previously participated in an earlier study on AVTX-803 and have confirmed LAD II through tests. Those with severe anemia or kidney problems are not eligible for this trial. As a Phase 3 trial, it represents the final step before FDA approval, providing participants an opportunity to contribute to the potential availability of a new treatment.
Do I have to stop taking my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.
Is there any evidence suggesting that AVTX-803 is likely to be safe for humans?
Research shows that AVTX-803 has been studied for its safety in treating leukocyte adhesion deficiency type II (LAD II). Earlier studies have examined its effectiveness and patient tolerance. Although detailed safety information isn't provided, the phase 3 trial indicates that AVTX-803 has already demonstrated a reasonable level of safety in previous studies. Phase 3 trials typically involve treatments tested for safety in people, with researchers now assessing long-term effectiveness and safety. This suggests that AVTX-803 is generally well-tolerated by patients.12345
Why do researchers think this study treatment might be promising?
Researchers are excited about AVTX-803 for treating Leukocyte Adhesion Deficiency because it offers a novel approach compared to existing treatments, which typically focus on bone marrow transplants. AVTX-803 works by providing a new active ingredient that is taken orally, potentially increasing convenience and improving patient adherence. This treatment targets the underlying cause of the condition with a specific dosing regimen that can be adjusted up to five times a day, offering a personalized approach to managing the disease.
What evidence suggests that AVTX-803 might be an effective treatment for Leukocyte Adhesion Deficiency?
Research has shown that AVTX-803 is being developed to treat leukocyte adhesion deficiency type II (LAD II), a rare genetic disorder. This treatment addresses a specific genetic problem causing LAD II. Early results suggest that AVTX-803 can improve symptoms and possibly enhance overall health in affected individuals. Although detailed information on its efficacy is still being collected, ongoing studies on long-term safety and effectiveness indicate that scientists remain hopeful about its potential.23567
Who Is on the Research Team?
David Deyle
Principal Investigator
Mayo Clinic
Are You a Good Fit for This Trial?
This trial is for individuals who have completed the AVTX-803-LAD-301 protocol and are confirmed to have Leukocyte Adhesion Deficiency Type II. They must not have severe anemia, impaired kidney function, or intolerance to fucose or any ingredients in AVTX-803.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
The study assesses the long-term safety and efficacy of AVTX-803 in subjects with LAD II
What Are the Treatments Tested in This Trial?
Interventions
- AVTX-803
Find a Clinic Near You
Who Is Running the Clinical Trial?
AUG Therapeutics
Lead Sponsor
Avalo Therapeutics, Inc.
Lead Sponsor