A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients with Leukocyte Adhesion Deficiency Type II
NCT ID: NCT05462587
Last Updated: 2024-11-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
4 participants
INTERVENTIONAL
2022-07-28
2024-11-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
DOUBLE
Study Groups
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AVTX-803
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.
AVTX-803 (L-Fucose)
L-fucose crystalline powder
Withdrawal
Subject will be in withdrawal for 8 weeks.
No interventions assigned to this group
Interventions
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AVTX-803 (L-Fucose)
L-fucose crystalline powder
Eligibility Criteria
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Inclusion Criteria
* 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 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
6 Months
75 Years
ALL
No
Sponsors
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AUG Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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David Deyle, MD
Role: PRINCIPAL_INVESTIGATOR
Mayo Clinic
Locations
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Mayo Clinic
Rochester, Minnesota, United States
Countries
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Central Contacts
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Facility Contacts
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Eva Morava-Kozicz, MD PhD
Role: primary
Other Identifiers
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AVTX-803-LAD-301
Identifier Type: -
Identifier Source: org_study_id
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