MedTrace Logo

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

NCT06309823 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2024-03-13

No results posted yet for this study

Summary

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.

Conditions

  • X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)

Interventions

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.

Sponsors & Collaborators

  • The Hospital for Sick Children

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
13 Years
Max Age
14 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-08
Primary Completion
2024-01-17
Completion
2024-01-17
FDA Drug
Yes

Countries

  • Canada

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06309823 on ClinicalTrials.gov