Back to resultsAn Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II
Primary Purpose
Leukocyte Adhesion Deficiency
Status
Recruiting
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
AVTX-803
Sponsored by
About this trial
This is an interventional treatment trial for Leukocyte Adhesion Deficiency
Eligibility 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) 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
Sites / Locations
- Mayo ClinicRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AVTX-803
Arm Description
AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day
Outcomes
Primary Outcome Measures
Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.
Secondary Outcome Measures
Sialyl-Lewis X antigen expression on leukocytes
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.
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.
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.
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."
Full Information
NCT ID
NCT05754450
First Posted
February 6, 2023
Last Updated
July 20, 2023
Sponsor
Avalo Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT05754450
Brief Title
An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II
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)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 10, 2023 (Actual)
Primary Completion Date
December 31, 2023 (Anticipated)
Study Completion Date
December 31, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Avalo Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. 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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leukocyte Adhesion Deficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
AVTX-803
Arm Type
Experimental
Arm Description
AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day
Intervention Type
Drug
Intervention Name(s)
AVTX-803
Intervention Description
L-fucose crystalline powder
Primary Outcome Measure Information:
Title
Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.
Time Frame
Through study completion, an average of 1 year.
Secondary Outcome Measure Information:
Title
Sialyl-Lewis X antigen expression on leukocytes
Time Frame
At 6 months
Title
Change from Baseline in the composite Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)
Description
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.
Time Frame
Through study completion, an average of 1 year.
Title
Change from Baseline in the individual parameters of the Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)
Description
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.
Time Frame
Through study completion, an average of 1 year.
Title
Goal Attainment Score (GAS)
Description
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.
Time Frame
Through study completion, an average of 1 year.
Title
Clinician Global Impression of Severity (CGI-S)
Description
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."
Time Frame
Through study completion, an average of 1 year.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility 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)
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Emily Cooke
Phone
610.254.4201
Email
ecooke@avalotx.com
First Name & Middle Initial & Last Name or Official Title & Degree
Lisa Goldberg
Phone
610.254.4201
Email
lgoldberg@avalotx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Garry Neil, MD
Organizational Affiliation
Avalo Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Eva Morava-Kozicz, MD PhD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II
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