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NCT Number: NCT05754450

An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type 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).

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Key information

Conditions

Leukocyte Adhesion Deficiency Leukocyte adhesion deficiency type 1

Age range

6 month–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location status: Recruiting

Location contact

Queenie Tan, MD PhD

CONTACT

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subject must have completed protocol AVTX-803-LAD-301
  • Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)

Exclusion criteria

  • 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

Treatment and study plan

AVTX-803

Drug

L-fucose crystalline powder

Primary outcomes

  1. Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.

    Time frame: Through study completion, an average of 1 year.

Secondary outcomes

  1. Sialyl-Lewis X antigen expression on leukocytes

    Time frame: At 6 months

  2. 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.

    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.

  3. 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.

    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.

  4. Goal Attainment Score (GAS)

    Time frame: Through study completion, an average of 1 year.

    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.

  5. Clinician Global Impression of Severity (CGI-S)

    Time frame: Through study completion, an average of 1 year.

    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."

Study contacts

Contact information is provided by the study sponsor or research team.

Jennifer Lin

CONTACT

[email protected]

3052833827

Sponsors and collaborators

Lead sponsor

AUG Therapeutics

Industry

Registry information

Official study 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)

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Mar 3, 2023
Registry last updated
May 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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