Leukocyte Adhesion DeficiencyPhase 3NCT05754450
An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II
A Phase 3 study testing AVTX-803 in people with Leukocyte Adhesion Deficiency, run by AUG Therapeutics at 1 site in United States.
Trial summary
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). 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
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Apr 2023to 31 Dec 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 have completed protocol AVTX-803-LAD-301
- Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)
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 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
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, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)
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).
- Conditions
- Leukocyte Adhesion Deficiency
- Being tested
- AVTX-803
- 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-803Drug
L-fucose crystalline powder
| Study group | Role | What they receive |
|---|---|---|
| AVTX-803 | Experimental | AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day Receives: Drug: AVTX-803 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.
Measured: Through study completion, an average of 1 year.
Secondary outcomes (5)
Sialyl-Lewis X antigen expression on leukocytes
Measured: At 6 months
Change from Baseline in the composite Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)
The NPCRS comprises 3 sections, with each section containing specific items relevant to the assessment of subjects with CDG. Section I (Current Function); Section II (System Specific Involvement); and Section III (Current Clinical Assessment). The sum of the scores is used to scale patients into 3 domains: mild (0-14), moderate (15-25), and severe (>26) category.
Measured: Through study completion, an average of 1 year.
Change from Baseline in the individual parameters of the Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)
The NPCRS comprises 3 sections, with each section containing specific items relevant to the assessment of subjects with CDG. Section I (Current Function); Section II (System Specific Involvement); and Section III (Current Clinical Assessment). The sum of the scores is used to scale patients into 3 domains: mild (0-14), moderate (15-25), and severe (>26) category.
Measured: Through study completion, an average of 1 year.
Goal Attainment Score (GAS)
The parent (or subject) formulates 3 individual goals for improving quality of life. Typically, these goals affect mobility, independence, and disease-related health aspects. These goals will be re-evaluated and scored by the parent or subject at each study visit within a scale of -2 to +2.
Measured: Through study completion, an average of 1 year.
Clinician Global Impression of Severity (CGI-S)
The CGI-S scale is a 7-point scale that assesses the severity of the subject's severity of disease/illness. 1=Normal, not ill at all; 2=borderline ill; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6= severely ill; 7=among the most extreme ill."
Measured: Through study completion, an average of 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2023
- Study start10 Apr 2023
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AUG Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Deyle, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05754450
- Sponsor's protocol ID
- AVTX-803-LAD-302
- Status last verified
- Apr 2026
- First submitted
- 6 Feb 2023
- 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, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)
- ClinicalTrials.gov ID
- NCT05754450
- Acronym / sponsor ID
- AVTX-803-LAD-302
- Conditions
- Leukocyte Adhesion Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 2 participants (estimated)
- Lead sponsor
- AUG TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 10 Apr 2023 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 3 Mar 2023 (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 extension study is to assess the long-term safety and efficacy of AVTX-803 in subjects with LAD II (SLC35C1-CDG).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AVTX-803 | Experimental | Drug: AVTX-803AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day |
Who may qualify
- Ages
- 6 months to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Subject must have completed protocol AVTX-803-LAD-301
- Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)
Key exclusion criteria (4 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 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-803L-fucose crystalline powder | Drug | AVTX-803 |
Primary outcome
Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.
Time frame: Through study completion, an average of 1 year.
5 secondary outcomes
Sialyl-Lewis X antigen expression on leukocytes
Time frame: At 6 months
Change from Baseline in the composite Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)
Time frame: Through study completion, an average of 1 year.
Change from Baseline in the individual parameters of the Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)
Time frame: Through study completion, an average of 1 year.
Goal Attainment Score (GAS)
Time frame: Through study completion, an average of 1 year.
Clinician Global Impression of Severity (CGI-S)
Time frame: Through study completion, an average of 1 year.
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 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 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 2 inclusion and 4 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?
Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803. (time frame: Through study completion, an average of 1 year.). 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.