Leukocyte Adhesion Deficiency
Conditions
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).
Interventions
DRUGAVTX-803
L-fucose crystalline powder
Sponsors
AUG Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803. | Through study completion, an average of 1 year. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
| 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." |
Countries
United States
Contacts
CONTACTJennifer Lin
PRINCIPAL_INVESTIGATORDavid Deyle
Mayo Clinic
Outcome results
None listed