An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II
Study Details
Study Description
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).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: AVTX-803 AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day |
Drug: AVTX-803
L-fucose crystalline powder
|
Outcome Measures
Primary Outcome Measures
- 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
- 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."
Eligibility Criteria
Criteria
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)
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Subject has impaired renal function as defined by an eGFR <90 mL/min
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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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Mayo Clinic | Rochester | Minnesota | United States | 55905 |
Sponsors and Collaborators
- Avalo Therapeutics, Inc.
Investigators
- Study Director: Garry Neil, MD, Avalo Therapeutics, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- AVTX-803-LAD-302