Loading clinical trials...

A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients with Leukocyte Adhesion Deficiency Type II | Clinical Trials | Clareo Health

RECRUITINGPHASE3

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)

NCT05462587•AUG Therapeutics

View on ClinicalTrials.gov

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).

Eligibility

Age

0 - 75 years

Sex

ALL

Healthy Volunteers

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

Locations (1)

Mayo Clinic

Rochester, Minnesota, United States

Key Dates

Start Date

July 28, 2022

Primary Completion Date

November 30, 2024

Completion Date

November 30, 2024

Last Updated

November 1, 2024

Enrollment

ESTIMATED participants

Conditions

Leukocyte Adhesion Deficiency

Interventions

AVTX-803 (L-Fucose)

DRUG

Contacts

Jennifer Lin

CONTACT

3052833827 huiyil@augtx.com

Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

NCT06282432

Leukocyte Adhesion Deficiency

View study

COMPLETEDPHASE2

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

NCT01998633

Hemophagocytic LymphohistiocytosisChronic Active Epstein-Barr Virus Infection+4 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: November 1, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

Gene Therapy for Patients With Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)