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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AVTX-803 | Experimental | AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AVTX-803 | Drug | L-fucose crystalline powder |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803. | Through study completion, an average of 1 year. |
| Measure | Description | Time Frame |
|---|---|---|
| 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) | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jennifer Lin | Contact | 3052833827 | huiyil@Augtx.com |
| Name | Affiliation | Role |
|---|---|---|
| David Deyle | Mayo Clinic | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic | Recruiting | Rochester | Minnesota | 55905 | United States |
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| ID | Term |
|---|---|
| C535887 | Leukocyte adhesion deficiency type 1 |
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| 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) | 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. |
| 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. |
| 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. |