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

AUG Therapeutics

Status and phase

Enrolling

Phase 3

Conditions

Leukocyte Adhesion Deficiency

Treatments

Drug: AVTX-803 (L-Fucose)

Study type

Interventional

Funder types

Industry

Identifiers

NCT05462587

AVTX-803-LAD-301

Details and patient eligibility

About

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).

Enrollment

4 estimated patients

Sex

All

Ages

6 months to 75 years old

Volunteers

No 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