An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II
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)
1 other identifier
interventional
2
1 country
1
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Apr 2023
Typical duration for phase_3
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 6, 2023
CompletedFirst Posted
Study publicly available on registry
March 3, 2023
CompletedStudy Start
First participant enrolled
April 10, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 31, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 31, 2026
May 1, 2026
April 1, 2026
3.7 years
February 6, 2023
April 27, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.
Through study completion, an average of 1 year.
Secondary Outcomes (5)
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.
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.
Goal Attainment Score (GAS)
Through study completion, an average of 1 year.
Clinician Global Impression of Severity (CGI-S)
Through study completion, an average of 1 year.
Study Arms (1)
AVTX-803
EXPERIMENTALAVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day
Interventions
Eligibility Criteria
You may qualify if:
- Subject must have completed protocol AVTX-803-LAD-301
- Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AUG Therapeuticslead
Study Sites (1)
Mayo Clinic
Rochester, Minnesota, 55905, United States
MeSH Terms
Conditions
Study Officials
- PRINCIPAL INVESTIGATOR
David Deyle
Mayo Clinic
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 6, 2023
First Posted
March 3, 2023
Study Start
April 10, 2023
Primary Completion (Estimated)
December 31, 2026
Study Completion (Estimated)
December 31, 2026
Last Updated
May 1, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will not share