RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

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)

Quick Facts

Study Start:2022-07-28

Study Completion:2024-11-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05462587

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Months to 75 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	* 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

Inclusion Criteria

Exclusion 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.

* 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

Contacts and Locations

Study Contact

Jennifer Lin

CONTACT

3052833827

huiyil@augtx.com

Principal Investigator

David Deyle, MD

PRINCIPAL_INVESTIGATOR

Mayo Clinic

Study Locations (Sites)

Mayo Clinic

Rochester, Minnesota, 55905

United States

Collaborators and Investigators

Sponsor: AUG Therapeutics

David Deyle, MD, PRINCIPAL_INVESTIGATOR, Mayo Clinic

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-07-28

Study Completion Date2024-11-30

Study Record Updates

Study Start Date2022-07-28

Study Completion Date2024-11-30

Terms related to this study

Additional Relevant MeSH Terms

Leukocyte Adhesion Deficiency