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

Primary Purpose

Leukocyte Adhesion Deficiency

Status
Recruiting
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
AVTX-803 (L-Fucose)
Sponsored by
Avalo Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leukocyte Adhesion Deficiency

Eligibility Criteria

6 Months - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Mayo ClinicRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

AVTX-803

Withdrawal

Arm Description

Approximately 4 subjects will receive AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day for 8 weeks.

Subject will be in withdrawal for 8 weeks.

Outcomes

Primary Outcome Measures

Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen
To assess the effect of AVTX-803 in subjects with leukocyte adhesion deficiency Type II (LAD II) by evaluating Sialyl-Lewis X on leukocytes as a percentage present at the end of each 8 week treatment period.

Secondary Outcome Measures

Full Information

First Posted
June 28, 2022
Last Updated
February 8, 2023
Sponsor
Avalo Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05462587
Brief Title
A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients With Leukocyte Adhesion Deficiency Type 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)
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 28, 2022 (Actual)
Primary Completion Date
December 31, 2023 (Anticipated)
Study Completion Date
December 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Avalo Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief 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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leukocyte Adhesion Deficiency

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
4 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
AVTX-803
Arm Type
Experimental
Arm Description
Approximately 4 subjects will receive AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day for 8 weeks.
Arm Title
Withdrawal
Arm Type
No Intervention
Arm Description
Subject will be in withdrawal for 8 weeks.
Intervention Type
Drug
Intervention Name(s)
AVTX-803 (L-Fucose)
Intervention Description
L-fucose crystalline powder
Primary Outcome Measure Information:
Title
Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen
Description
To assess the effect of AVTX-803 in subjects with leukocyte adhesion deficiency Type II (LAD II) by evaluating Sialyl-Lewis X on leukocytes as a percentage present at the end of each 8 week treatment period.
Time Frame
Change from Baseline at Day 56, Change from Baseline at Day 112

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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: Non-lactating and has a negative pregnancy test at screening -AND- 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Lisa Goldberg
Phone
6102544201
Email
lgoldberg@avalotx.com
First Name & Middle Initial & Last Name or Official Title & Degree
Emily Cooke
Phone
6102544201
Email
ecooke@avalotx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Garry Neil, MD
Organizational Affiliation
Avalo Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Eva Morava-Kozicz, MD PhD

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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