A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients With Leukocyte Adhesion Deficiency Type II

Leukocyte Adhesion Deficiency Clinical Trial

Official title:

A Phase 3, Randomized, Double-blind, Two-period, Crossover, Withdrawal Study to Assess the Efficacy and Safety of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05462587
• Other study ID #: AVTX-803-LAD-301
• Status: Recruiting
• Phase: Phase 3
• Start date: July 28, 2022
• Est. completion date: September 30, 2024

Study information

• Verified date: March 2024
• Source: AUG Therapeutics
• Contact: Lisa Goldberg
• Phone: 6102544201
• Email: lgoldberg[@]avalotx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The primary objective of this study is to evaluate the efficacy and safety of AVTX-803 compared to withdrawal in patients with Leukocyte Adhesion Deficiency, Type II (LAD II).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 4
• Est. completion date: September 30, 2024
• Est. primary completion date: September 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 75 Years

Eligibility

Inclusion Criteria:

• Subject must be between 6 months and 75 years old
• Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)
• Subject has a documented history of Lewis antigen deficiency
• Subject has a history of recurrent infections, opportunistic infections or infections that did not respond well to standard of care treatment
• Subject or parent (for subjects under legal age for consent) has provided written informed consent for this study. Additionally, written informed assent has been provided, as appropriate, for minors of older age, per local institutional review board (IRB)/ethics committee (EC) policy and requirements
• Subject is willing and able to comply with the protocol
• Women of childbearing potential (WOCBP) meeting the criteria below:

1. Non-lactating and has a negative pregnancy test at screening -AND-

2. Uses an acceptable double-barrier method of contraception as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug.

• Male subjects must agree to use an acceptable double-barrier method of contraception with their partner as determined by the investigator or sub-investigator for the duration of the study and 30 days following the last dose of study drug.

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 a total absence of fucosylation on red blood cells and the presence of anti-H antigen
• 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
• In the investigator's opinion, subject is not able or not willing to comply with the study requirements.
• Subject is pregnant

Related Conditions & MeSH terms

• Leukocyte Adhesion Deficiency
• Leukocyte-Adhesion Deficiency Syndrome
• Tissue Adhesions

Intervention

Drug:
• AVTX-803 (L-Fucose)
L-fucose crystalline powder

Locations

Country	Name	City	State
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
AUG Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen	To assess the effect of AVTX-803 in subjects with leukocyte adhesion deficiency Type II (LAD II) by evaluating Sialyl-Lewis X on leukocytes as a percentage present at the end of each 8 week treatment period.	Change from Baseline at Day 56, Change from Baseline at Day 112