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Recruiting NCT05754450

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

Phase III Interventional Leukocyte Adhesion Deficiency

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: AVTX-803.
Who it may be relevant to
Registry conditions: Leukocyte Adhesion Deficiency. Basic parameters: 6 months — 75 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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)

Overview

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).

Interventions

  • Drug AVTX-803
    L-fucose crystalline powder

Primary outcome measures

  • 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 outcome measures (5)
  • 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.]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Mayo Clinic — Rochester

Identifiers

NCT: NCT05754450 · AVTX-803-LAD-302

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗