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

The Hospital for Sick Children

Status and phase: Completed; Phase 3

Conditions: X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)

Treatments: Drug: MAS825

Study type: Interventional

Funder types: Other

Identifiers

NCT06309823

1000080330

Details and patient eligibility

About

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.

Enrollment: 1 patient

Sex: Male

Ages: 13 to 14 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

• N/A

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

Active treatment

Experimental group

Treatment:

Drug: MAS825