A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients With Leukocyte Adhesion Deficiency Type II

NCT05462587 | Recruiting Phase 3 FDA Drug

• 1 other identifier: AVTX-803-LAD-301
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Leukocyte Adhesion Deficiency

Outcome Measures

Primary Outcomes (1)

• 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.

  Change from Baseline at Day 56, Change from Baseline at Day 112

Study Arms (2)

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.

Drug: AVTX-803 (L-Fucose)

Withdrawal

NO INTERVENTION

Subject will be in withdrawal for 8 weeks.

Interventions

AVTX-803 (L-Fucose) DRUG

L-fucose crystalline powder

AVTX-803

Eligibility Criteria

• Age: 6 Months - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• AUG Therapeutics: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

MeSH Terms

Conditions

Leukocyte adhesion deficiency type 1

Interventions

Fucose

Intervention Hierarchy (Ancestors)

Deoxy Sugars; Carbohydrates

Study Officials

• David Deyle, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jennifer Lin

CONTACT

3052833827; huiyil@augtx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2022
• First Posted: July 18, 2022
• Study Start: July 28, 2022
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2026
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)