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)
Verified date | March 2024 |
Source | AUG Therapeutics |
Contact | Emily Cooke |
Phone | 610.254.4201 |
ecooke[@]avalotx.com | |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
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 |
Country | Name | City | State |
---|---|---|---|
United States | Mayo Clinic | Rochester | Minnesota |
Lead Sponsor | Collaborator |
---|---|
AUG Therapeutics |
United States,
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. |
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