AVTX-803 for Leukocyte Adhesion Deficiency
LG
EC
JL
Overseen ByJennifer Lin
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: AUG Therapeutics
No Placebo GroupAll 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 DataThis treatment has passed at least one previous human trial
Trial Summary
What is the purpose of this trial?
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).
Research Team
DD
David Deyle
Principal Investigator
Mayo Clinic
Eligibility Criteria
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.
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Timeline
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
Treatment Details
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.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: AVTX-803Experimental Treatment1 Intervention
AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day
Find a Clinic Near You
Who Is Running the Clinical Trial?
AUG Therapeutics
Lead Sponsor
Trials
2
Recruited
6+
Avalo Therapeutics, Inc.
Lead Sponsor
Trials
13
Recruited
2,100+
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