NCT06309823

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: The Hospital for Sick Children · Academic / Other
• Sex: Male
• Age: 13 Years – 14 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
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.

Timeline

• Start date: 2023-02-08
• Primary completion: 2024-01-17
• Completion: 2024-01-17
• First posted: 2024-03-13
• Last updated: 2024-03-13

Locations

1 site across 1 country: Canada

Regulatory

• FDA-regulated drug study