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

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)

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).

Study Overview

• Status: Recruiting
• Conditions: Leukocyte Adhesion Deficiency
• Intervention / Treatment: Drug: AVTX-803 (L-Fucose)

• Study Type: Interventional
• Enrollment (Estimated): 4
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Lisa Goldberg; Phone Number: 6102544201; Email: lgoldberg@avalotx.com
• Study Contact Backup: Name: Emily Cooke; Phone Number: 6102544201; Email: ecooke@avalotx.com

Study Locations

• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic
      • Contact: Eva Morava-Kozicz, MD PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AVTX-803; Approximately 4 subjects will receive AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day for 8 weeks.	Drug: AVTX-803 (L-Fucose); L-fucose crystalline powder
No Intervention: Withdrawal; Subject will be in withdrawal for 8 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: AUG Therapeutics
• Investigators: Study Director: Garry Neil, MD, Avalo Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 28, 2022
• Primary Completion (Estimated): September 30, 2024
• Study Completion (Estimated): September 30, 2024

Study Registration Dates

• First Submitted: June 28, 2022
• First Submitted That Met QC Criteria: July 14, 2022
• First Posted (Actual): July 18, 2022

Study Record Updates

• Last Update Posted (Actual): March 5, 2024
• Last Update Submitted That Met QC Criteria: March 4, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immunologic Deficiency Syndromes; Immune System Diseases; Genetic Diseases, Inborn; Fibrosis; Cicatrix; Primary Immunodeficiency Diseases; Tissue Adhesions; Leukocyte-Adhesion Deficiency Syndrome
• Other Study ID Numbers: AVTX-803-LAD-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No