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

NCT06309823 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: 1000080330
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (3)

• 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

  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

  5 years

• Patient/Parent Global Assessment of Disease Activity

  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

  5 years

Study Arms (1)

Active treatment

EXPERIMENTAL

Drug: MAS825

Interventions

MAS825 DRUG

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.

Active treatment

Eligibility Criteria

• Age: 13 Years - 14 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• N/A

Sponsors & Collaborators

• The Hospital for Sick Children: lead

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G1X8, Canada

Location

MeSH Terms

Conditions

Lymphoproliferative Syndrome, X-Linked, 2

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Project Investigator

Study Record Dates

• First Submitted: February 23, 2024
• First Posted: March 13, 2024
• Study Start: February 8, 2023
• Primary Completion: January 17, 2024
• Study Completion: January 17, 2024
• Last Updated: March 13, 2024

Record last verified: 2024-03

Locations

CA (1)