Leukocyte Adhesion DeficiencyPhase 3NCT05462587
A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients With Leukocyte Adhesion Deficiency Type II
A Phase 3 study testing AVTX-803 (L-Fucose) in people with Leukocyte Adhesion Deficiency, run by AUG Therapeutics at 1 site in United States.
Trial summary
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, Type II (LAD II). Read more about this study
- Who is it for?
- Ages 6 months to 75 years
- Conditions the study is for
- Leukocyte Adhesion Deficiency
- What is being tested?
- AVTX-803 (L-Fucose)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Jul 2022to 30 Nov 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject must be between 6 months and 75 years old
- 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
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- Women of childbearing potential (WOCBP) meeting the criteria below:
- Non-lactating and has a negative pregnancy test at screening -AND-
- 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.
Who cannot join
- Subject has severe anemia defined as hemoglobin <8.0 g/dL (<4.9 mmol/L)
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Queenie Tan, MD PhD
About this study
Official title: A Phase 3, Randomized, Double-blind, Two-period, Crossover, Withdrawal Study to Assess the Efficacy and Safety of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)
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, Type II (LAD II).
- Conditions
- Leukocyte Adhesion Deficiency
- Being tested
- AVTX-803 (L-Fucose)
- Medical subject headings
- Leukocyte adhesion deficiency type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AVTX-803 (L-Fucose)Drug
L-fucose crystalline powder
| Study group | Role | What they receive |
|---|---|---|
| AVTX-803 | Experimental | 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. Receives: Drug: AVTX-803 (L-Fucose) |
| Withdrawal | No intervention | Subject will be in withdrawal for 8 weeks. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen
To assess the effect of AVTX-803 in subjects with leukocyte adhesion deficiency Type II (LAD II) by evaluating Sialyl-Lewis X on leukocytes as a percentage present at the end of each 8 week treatment period.
Measured: Change from Baseline at Day 56, Change from Baseline at Day 112
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2022
- Study start28 Jul 2022
- Primary completion30 Nov 2026 (estimated)
- Study completion30 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukocyte Adhesion DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 4 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AUG Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Deyle, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05462587
- Sponsor's protocol ID
- AVTX-803-LAD-301
- Status last verified
- Apr 2026
- First submitted
- 28 Jun 2022
- Last updated
- 1 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-blind, Two-period, Crossover, Withdrawal Study to Assess the Efficacy and Safety of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)
- ClinicalTrials.gov ID
- NCT05462587
- Acronym / sponsor ID
- AVTX-803-LAD-301
- Conditions
- Leukocyte Adhesion Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 4 participants (estimated)
- Lead sponsor
- AUG TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 28 Jul 2022 (actual)
- Primary completion
- 30 Nov 2026 (estimated)
- Study completion
- 30 Nov 2026 (estimated)
- First posted
- 18 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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, Type II (LAD II).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AVTX-803 | Experimental | Drug: AVTX-803 (L-Fucose)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. |
| Withdrawal | No intervention | –Subject will be in withdrawal for 8 weeks. |
Who may qualify
- Ages
- 6 months to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Subject must be between 6 months and 75 years old
- 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
Key exclusion criteria (7 listed)
- Subject has severe anemia defined as hemoglobin <8.0 g/dL (<4.9 mmol/L)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AVTX-803 (L-Fucose)L-fucose crystalline powder | Drug | AVTX-803 |
Primary outcome
Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen
Time frame: Change from Baseline at Day 56, Change from Baseline at Day 112
To assess the effect of AVTX-803 in subjects with leukocyte adhesion deficiency Type II (LAD II) by evaluating Sialyl-Lewis X on leukocytes as a percentage present at the end of each 8 week treatment period.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
David Deyle, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AVTX-803 (L-Fucose) in people with Leukocyte Adhesion Deficiency, run by AUG Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jennifer Lin (3052833827, huiyil@augtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen (time frame: Change from Baseline at Day 56, Change from Baseline at Day 112). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2026.