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

Phase 3

Recruiting

• Conditions: Leukocyte Adhesion Deficiency
• Interventions: Drug: AVTX-803

• Registration Number: NCT05754450
• Lead Sponsor: AUG Therapeutics

Brief 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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-02-06
• Study First Submitted QC: 2023-03-02
• Study First Posted: 2023-03-03
• Study Start: 2023-04-10
• Status Verified: 2024-03
• Last Update Submitted: 2024-10-30
• Last Update Posted: 2024-11-01
• Primary Completion: 2024-12-31
• Study Completion: 2024-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 2

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AVTX-803	AVTX-803	AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day

Primary Outcome Measures

Name	Time	Method
Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.	Through study completion, an average of 1 year.

Secondary Outcome Measures

Name	Time	Method
Clinician Global Impression of Severity (CGI-S)	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."
Goal Attainment Score (GAS)	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.
Change from Baseline in the individual parameters of the Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)	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.
Sialyl-Lewis X antigen expression on leukocytes	At 6 months
Change from Baseline in the composite Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)	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.

Trial Locations

Locations (1)

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States