X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)
Conditions
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.
Interventions
DRUGMAS825
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.
Sponsors
The Hospital for Sick Children
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
13 Years to 14 Years
Healthy volunteers
No
Inclusion criteria
* Known patient with XIAP genetic defect under the care of the investigator
Exclusion criteria
* N/A
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| 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 |
Countries
Canada
Outcome results
None listed