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

The Hospital for Sick Children1 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

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

Interventions

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.

Eligibility

Sex: MALEMin age: 13 YearsMax age: 14 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Known patient with XIAP genetic defect under the care of the investigator Exclusion Criteria: * N/A

Locations (1)

The Hospital for Sick Children

Toronto, Ontario, Canada

Outcomes

Primary Outcomes

Impact of MAS825 on Physician Global Assessment Score

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

Time frame: 5 years

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

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

Time frame: 5 years

Patient/Parent Global Assessment of Disease Activity

Time frame: 5 years

Data from ClinicalTrials.gov

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