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Trial record 1 of 1 for:    NCT05462587
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The Primary Objective of This Study is to Evaluate Efficacy and Safety of AVTX-803 in Patients With Leukocyte Adhesion Deficiency Type II

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05462587
Recruitment Status : Recruiting
First Posted : July 18, 2022
Last Update Posted : August 5, 2022
Sponsor:
Information provided by (Responsible Party):
Avalo Therapeutics, Inc.

Study Description
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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).

Condition or disease Intervention/treatment Phase
Leukocyte Adhesion Deficiency Drug: AVTX-803 (L-Fucose) Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 4 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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)
Actual Study Start Date : July 28, 2022
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : December 31, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Adhesions

Arms and Interventions
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Arm Intervention/treatment
Experimental: AVTX-803
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.
 Drug: AVTX-803 (L-Fucose)
L-fucose crystalline powder
No Intervention: Withdrawal
Subject will be in withdrawal for 8 weeks.


Outcome Measures
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Primary Outcome Measures :
  1. Effect of AVTX-803 on the percent of leukocytes expressing Sialyl-Lewis X antigen [ Time Frame: Change from Baseline at Day 56, Change from Baseline at Day 112 ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   6 Months to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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:

    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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05462587


Contacts
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Contact: John Boland 6102544201 jboland@avalotx.com
Contact: Emily Cooke 4845885264 ecooke@avalotx.com

Locations
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United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Eva Morava-Kozicz, MD PhD         
Sponsors and Collaborators
Avalo Therapeutics, Inc.
More Information
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Responsible Party: Avalo Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05462587    
Other Study ID Numbers: AVTX-803-LAD-301
First Posted: July 18, 2022    Key Record Dates
Last Update Posted: August 5, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Leukocyte-Adhesion Deficiency Syndrome
Tissue Adhesions
Cicatrix
Fibrosis
Pathologic Processes
 Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases