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

Leukocyte Adhesion Deficiency Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05754450
• Other study ID #: AVTX-803-LAD-302
• Status: Recruiting
• Phase: Phase 3
• Start date: April 10, 2023
• Est. completion date: December 31, 2024

Study information

• Verified date: March 2024
• Source: AUG Therapeutics
• Contact: Emily Cooke
• Phone: 610.254.4201
• Email: ecooke[@]avalotx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 2
• Est. completion date: December 31, 2024
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Leukocyte Adhesion Deficiency
• Leukocyte-Adhesion Deficiency Syndrome
• Tissue Adhesions

Intervention

Drug:
• AVTX-803
L-fucose crystalline powder

Locations

Country	Name	City	State
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
AUG Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.		Through study completion, an average of 1 year.
Secondary	Sialyl-Lewis X antigen expression on leukocytes		At 6 months
Secondary	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.	Through study completion, an average of 1 year.
Secondary	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.	Through study completion, an average of 1 year.
Secondary	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.	Through study completion, an average of 1 year.
Secondary	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."	Through study completion, an average of 1 year.