An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects with Leukocyte Adhesion Deficiency Type II

AUG Therapeutics

Status and phase: Enrolling; Phase 3

Conditions: Leukocyte Adhesion Deficiency

Treatments: Drug: AVTX-803

Study type: Interventional

Funder types: Industry

Identifiers

NCT05754450

AVTX-803-LAD-302

Details and patient eligibility

About

The primary objective of this extension study is to assess the long-term safety and efficacy of AVTX-803 in subjects with LAD II (SLC35C1-CDG).

Enrollment: 2 estimated patients

Sex: All

Ages: 6 months to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subject must have completed protocol AVTX-803-LAD-301
• Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)

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 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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2 participants in 1 patient group

AVTX-803

Experimental group

Description:

AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day

Treatment:

Drug: AVTX-803