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

March 7, 2024 updated by: Dilan Dissanayake, The Hospital for Sick Children
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G1X8
        • The Hospital for Sick Children

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • N/A

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Active treatment
Drug: MAS825
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Impact of MAS825 on Physician Global Assessment Score
Time Frame: 5 years

Likert Scale:

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

  1. = Minimal disease associated signs and symptoms
  2. = Mild disease associated signs and symptoms
  3. = Moderate disease associated signs and symptoms
  4. = Severe disease associated signs and symptoms
5 years
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

  1. = Minimal
  2. = Mild
  3. = Moderate
  4. = Severe
5 years
Patient/Parent Global Assessment of Disease Activity
Time Frame: 5 years

Likert Scale:

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

  1. = Minimal disease associated signs and symptoms
  2. = Mild disease associated signs and symptoms
  3. = Moderate disease associated signs and symptoms
  4. = Severe disease associated signs and symptoms
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Hospital for Sick Children

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 8, 2023

Primary Completion (Actual)

January 17, 2024

Study Completion (Actual)

January 17, 2024

Study Registration Dates

First Submitted

February 23, 2024

First Submitted That Met QC Criteria

March 7, 2024

First Posted (Actual)

March 13, 2024

Study Record Updates

Last Update Posted (Actual)

March 13, 2024

Last Update Submitted That Met QC Criteria

March 7, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 1000080330

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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