AVTX-803 for Leukocyte Adhesion Deficiency

Overseen By Jennifer Lin

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: AUG Therapeutics

Disqualifiers: Severe anemia, Impaired renal function, Fucose intolerance, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 1 year

2 Participants Needed

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. ^{1 2 3 4 5}

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. ^{2 3 5 6 7}

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

Subject must have completed protocol AVTX-803-LAD-301

I have been diagnosed with LAD II through genetic and biochemical tests.

Exclusion Criteria

My hemoglobin level is below 8.0 g/dL.

My doctor thinks I did not respond to fucose treatment despite proper dosing.

My kidney function is reduced with an eGFR below 90 mL/min.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

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

1 year

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

The study assesses the long-term safety and efficacy of AVTX-803 in subjects with LAD II

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• AVTX-803

Trial Overview

The study aims to evaluate the long-term safety and effectiveness of a treatment called AVTX-803 in patients with LAD II, a rare genetic disorder affecting white blood cells' ability to adhere properly.

How Is the Trial Designed?

Group I: AVTX-803 Experimental Treatment 1 Intervention

AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day

Who Is Running the Clinical Trial?

AUG Therapeutics

Lead Sponsor

Trials

2

Recruited

6+

Avalo Therapeutics, Inc.

Lead Sponsor

Trials

13

Recruited

2,100+

Citations

1.

ir.avalotx.com

ir.avalotx.com/news-events-presentations/press-releases/detail/156/avalo-therapeutics-announces-first-patient-dosed-in-the-avtx-803-pivotal-trial-ladder-for-the-treatment-of-leukocyte-adhesion-deficiency-type-ii-lad-ii

Avalo Therapeutics Announces First Patient Dosed in the ...

AVTX-803 is a therapy developed and studied for the treatment of leukocyte adhesion deficiency type II (LAD II, also known as SLC35C1-CDG). AVTX ...

2.

mayo.edu

mayo.edu/research/clinical-trials/cls-20542200

AVTX-803 in Patients with Leukocyte Adhesion Deficiency ...

The purpose of this study is to assess the long-term safety and effectiveness of AVTX-803 in subjects with leukocyte adhesion deficiency Type II (LAD II).

3.

trialx.com

trialx.com/clinical-trials/listings/262027/an-extension-study-assessing-the-safety-and-efficacy-of-avtx-803-in-subjects-with-leukocyte-adhesion-deficiency-type-ii/

An Extension Study Assessing the Safety and Efficacy of ...

Overview. The primary objective of this extension study is to assess the long-term safety and efficacy of AVTX-803 in subjects with LAD II ...

4.

aging.networkofcare.org

aging.networkofcare.org/sanmateo/CommunityResources/ClinicalTrials/Detail/NCT05462587?keyword=%22Adhesion%22

A Study to Evaluate Efficacy and Safety of AVTX-803 in ...

The primary objective of this study is to evaluate the efficacy and safety of AVTX-803 compared to withdrawal in patients with Leukocyte Adhesion Deficiency ...

5.

withpower.com

withpower.com/trial/phase-3-leukocyte-adhesion-deficiency-syndrome-1-2023-a2ed7

AVTX-803 for Leukocyte Adhesion Deficiency

Trial Overview The study aims to evaluate the long-term safety and effectiveness of a treatment called AVTX-803 in patients with LAD II, a rare genetic disorder ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05462587

A Study to Evaluate Efficacy and Safety of AVTX-803 in ...

The primary objective of this study is to evaluate the efficacy and safety of AVTX-803 compared to withdrawal in patients with Leukocyte Adhesion Deficiency, ...

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05754450

NCT05754450 | An Extension Study Assessing the Safety ...

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). Official Title.