What is the mechanism of action of this treatment?

The most common treatments for Leukocyte Adhesion Deficiency (LAD) focus on correcting or compensating for the molecular abnormalities that impair leukocyte function. AVTX-803, for example, is designed to enhance the adhesion capabilities of leukocytes, which is crucial for their proper functioning in immune responses. By improving leukocyte adhesion, these treatments aim to restore the ability of leukocytes to migrate to sites of infection or injury, thereby improving the immune response in LAD patients. This is vital for LAD patients as it helps in preventing recurrent infections and promoting better overall immune function.

What side effects are associated with this type of intervention?

Treatments for Leukocyte Adhesion Deficiency (LAD), such as AVTX-803, which aim to correct or compensate for molecular abnormalities in leukocyte function, may have side effects similar to other immunomodulatory therapies. Common side effects can include infections due to altered immune responses, hypersensitivity reactions, and potential hematologic abnormalities such as neutropenia. As with any therapy targeting immune function, monitoring for adverse effects is crucial to ensure patient safety.

What prior FDA approvals exist?

There are no specific FDA-approved treatments for Leukocyte Adhesion Deficiency (LAD) that directly address the underlying molecular defects similar to AVTX-803. AVTX-803 is designed to correct or compensate for the molecular abnormalities impairing leukocyte function in LAD II patients. Current management of LAD typically involves supportive care, including antibiotics to treat infections and hematopoietic stem cell transplantation (HSCT) for severe cases. The development of targeted therapies like AVTX-803 represents a novel approach in addressing the root cause of the disease.

What other types of research exist?

<ol> <li>Lipoxin A4 Analogs: These compounds, such as 15(R/S)-methyl-LXA4, 15-cyclohexyl-LXA4, and 16-phenoxy-LXA4, have been shown to inhibit neutrophil transmigration and adhesion, which could be beneficial for LAD patients by modulating leukocyte function and reducing inflammation.</li> <li></li> </ol> CXCR4 Inhibitors: AMD070, a selective CXCR4 receptor inhibitor, has demonstrated the ability to elevate white blood cell counts and could potentially enhance leukocyte function in LAD patients by modulating leukocyte mobilization and adhesion. 3. Alpha4-Integrin Blockade: The combination of alpha4-integrin blockade with CXCR4 inhibition has shown additive effects in mobilizing hematopoietic stem/progenitor cells, which might be leveraged to improve leukocyte function in LAD patients.

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AVTX-803 for Leukocyte Adhesion Deficiency

Phase 3

Recruiting

Led By David Deyle, MD

Research Sponsored by AUG Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up change from baseline at day 56, change from baseline at day 112

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a medication called AVTX-803 to see if it can help people with a rare immune system disorder called Leukocyte Adhesion Deficiency, Type II (LAD II). The medication aims to improve how their white blood cells work so they can better fight infections. An earlier version of this medication has shown promise in enhancing immune cell responses and treating various conditions.

Who is the study for?

This trial is for people aged 6 months to 75 years with Leukocyte Adhesion Deficiency Type II. Participants must have a history of recurrent or poorly responding infections and agree to use double-barrier contraception. It's not for those intolerant to fucose, with kidney issues (eGFR <90 mL/min), severe anemia, or who are pregnant.

What is being tested?

The study tests the effectiveness and safety of AVTX-803 (L-Fucose) in patients with LAD II compared to those who stop treatment. The goal is to see if this intervention can help manage symptoms associated with the condition.

What are the potential side effects?

While specific side effects aren't listed here, potential risks may include reactions related to intolerance or hypersensitivity to fucose or other ingredients in AVTX-803.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ change from baseline at day 56, change from baseline at day 112

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~change from baseline at day 56, change from baseline at day 112

This trial's timeline: 3 weeks for screening, Varies for treatment, and change from baseline at day 56, change from baseline at day 112 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: AVTX-803Experimental Treatment1 Intervention

Approximately 4 subjects will receive AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day for 8 weeks.

Group II: WithdrawalActive Control1 Intervention

Subject will be in withdrawal for 8 weeks.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Leukocyte Adhesion Deficiency (LAD) focus on correcting or compensating for the molecular abnormalities that impair leukocyte function. AVTX-803, for example, is designed to enhance the adhesion capabilities of leukocytes, which is crucial for their proper functioning in immune responses. By improving leukocyte adhesion, these treatments aim to restore the ability of leukocytes to migrate to sites of infection or injury, thereby improving the immune response in LAD patients. This is vital for LAD patients as it helps in preventing recurrent infections and promoting better overall immune function.

Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study.Volume of Gadolinium Enhancement and Successful Repair of the Blood-Brain Barrier in Cerebral Adrenoleukodystrophy.The Landscape of Hematopoietic Stem Cell Transplant and Gene Therapy for X-Linked Adrenoleukodystrophy.