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

NCT ID: NCT05754450

Last Updated: 2024-11-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-04-10

Study Completion Date

2024-12-31

Brief Summary

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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).

Detailed Description

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Conditions

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Leukocyte Adhesion Deficiency

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AVTX-803

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

Group Type EXPERIMENTAL

AVTX-803

Intervention Type DRUG

L-fucose crystalline powder

Interventions

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AVTX-803

L-fucose crystalline powder

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

6 Months

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AUG Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David Deyle

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

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Mayo Clinic

Rochester, Minnesota, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Jennifer Lin

Role: CONTACT

3052833827

Facility Contacts

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Eva Morava-Kozicz, MD PhD

Role: primary

Other Identifiers

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AVTX-803-LAD-302

Identifier Type: -

Identifier Source: org_study_id

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