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

Phase 3

Completed

• Conditions: X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)
• Interventions: Drug: MAS825

• Registration Number: NCT06309823
• Lead Sponsor: The Hospital for Sick Children

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-02-08
• Primary Completion: 2024-01-17
• Study Completion: 2024-01-17
• Study First Submitted: 2024-02-23
• Status Verified: 2024-03
• Study First Submitted QC: 2024-03-07
• Last Update Submitted: 2024-03-07
• Study First Posted: 2024-03-13
• Last Update Posted: 2024-03-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 1

Inclusion Criteria

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

Exclusion Criteria

• N/A

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Active treatment	MAS825	-

Primary Outcome Measures

Name	Time	Method
Impact of MAS825 on Physician Severity Assessment of Disease Signs and Symptoms	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
Patient/Parent Global Assessment of Disease Activity	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
Impact of MAS825 on Physician Global Assessment Score	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

Trial Locations

Locations (1)

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada