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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active treatment | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Impact of MAS825 on Physician Global Assessment Score | Likert Scale: 0 = Absent (no) disease associated clinical 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
| 5 years |
| Patient/Parent Global Assessment of Disease Activity | Likert Scale: 0 = Absent (no) disease associated clinical signs and symptoms
| 5 years |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Hospital for Sick Children | Toronto | Ontario | M5G1X8 | Canada |
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| ID | Term |
|---|---|
| C564469 | Lymphoproliferative Syndrome, X-Linked, 2 |
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