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Completed

NCT Number: NCT06309823

A Single-patient Clinical Trial of MAS825 in a Patient With XIAP Deficiency

MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1 beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2. Clinical trials are currently examining its efficacy in other diseases associated with elevations of these cytokines, including NLRC4-associated disease and hidradenitis suppurativa. This study proposes to assess the effectiveness of MAS825 in a single patient with XLP-2, who has previously demonstrated response to blockade of IL-1 beta and IL-18. Given the lack of alternative pharmaceutical options for XLP-2, this represents the only known medication option that avoids the toxicity associated with high-dose corticosteroids and the morbidity associated with hematopoietic stem cell transplantation.

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Key information

Conditions

X-linked Lymphoproliferative Syndrome Type 2 (XLP-2) Lymphoproliferative Syndrome, X-Linked, 2

Age range

13 year–14 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

The Hospital for Sick Children

Toronto, Ontario, M5G1X8, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Known patient with XIAP genetic defect under the care of the investigator

Exclusion criteria

  • N/A

Treatment and study plan

MAS825

Drug

MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2.

Primary outcomes

  1. Impact of MAS825 on Physician Global Assessment Score

    Time frame: 5 years

    Likert Scale:

    0 = Absent (no) disease associated clinical signs and symptoms

    • = Minimal disease associated signs and symptoms
    • = Mild disease associated signs and symptoms
    • = Moderate disease associated signs and symptoms
    • = Severe disease associated signs and symptoms

  2. Impact of MAS825 on Physician Severity Assessment of Disease Signs and Symptoms

    Time frame: 5 years

    The signs and symptoms assessed include: 1) Abdominal pain 2) Diarrhea 3) Fever using a Likert scale:

    0 = Absent

    • = Minimal
    • = Mild
    • = Moderate
    • = Severe

  3. Patient/Parent Global Assessment of Disease Activity

    Time frame: 5 years

    Likert Scale:

    0 = Absent (no) disease associated clinical signs and symptoms

    • = Minimal disease associated signs and symptoms
    • = Mild disease associated signs and symptoms
    • = Moderate disease associated signs and symptoms
    • = Severe disease associated signs and symptoms

Sponsors and collaborators

Lead sponsor

The Hospital for Sick Children

Other

Registry information

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Mar 13, 2024
Registry last updated
Mar 13, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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