What is the mechanism of action of this treatment?

The most common treatments for X-linked Autoinflammation with Infantile Enterocolitis Syndrome (XLAIES) involve immunomodulatory agents that target specific immune pathways to control the hyperactive immune response. These treatments are essential for managing the excessive inflammation seen in XLAIES patients. For example, agents targeting IL-18 driven autoinflammatory pathways, similar to those studied in the MAS825 trial, can help reduce inflammation by modulating the activity of immune cells involved in these pathways. This targeted approach is crucial for improving clinical outcomes and reducing the severity of symptoms in XLAIES patients.

What side effects are associated with this type of intervention?

Treatments for X-linked Autoinflammation with Infantile Enterocolitis Syndrome, particularly those involving immunomodulatory agents like MAS825, can have side effects such as increased risk of infections, infusion-related reactions (fever, chills, rash), gastrointestinal symptoms (nausea, vomiting, diarrhea), and liver enzyme abnormalities. Severe but less common side effects may include cytokine release syndrome, capillary leak syndrome, and hematologic abnormalities.

What prior FDA approvals exist?

There is limited specific information on FDA-approved treatments for X-linked Autoinflammation with Infantile Enterocolitis Syndrome (XLAIES). However, treatments targeting similar immune pathways, such as those involved in NLRC4-GOF, XIAP deficiency, or CDC42 mutations, include immunomodulatory agents like canakinumab, which has shown efficacy in treating autoinflammatory diseases by inhibiting interleukin-1β. Other related treatments include abatacept, which modulates T-cell activation, and rituximab, which targets B cells. These treatments highlight the potential of immunomodulatory therapies in managing autoinflammatory conditions.

What other types of research exist?

Promising novel alternatives to MAS825 for treating X-linked Autoinflammation with Infantile Enterocolitis Syndrome (XLAIES) in 2024 may include other immunomodulatory agents such as canakinumab, which targets IL-1β, and anti-IL-23 agents like ustekinumab and mirikizumab, which have shown efficacy in related inflammatory conditions. These agents target specific immune pathways and may offer therapeutic benefits for XLAIES patients.

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MAS825 for Autoinflammatory Diseases (MASter-1 Trial)

Phase 2

Recruiting

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

For all Patients: Male and female patients weighing at least 3 kg

Must not have

Patients weighing >160 kg at Screening.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up screening through eos

Summary

This trial is testing a new medication called MAS825 in patients with rare genetic conditions that don't respond well to typical treatments. The study will take several years to evaluate the medication's effectiveness and safety. The goal is to see if MAS825 can help manage these conditions by targeting the underlying genetic problems.

Who is the study for?

This trial is for patients with specific genetic autoinflammatory diseases (NLRC4-GOF, XIAP deficiency, or CDC42 mutations) who weigh at least 3 kg. Participants must provide informed consent and have active disease symptoms. Excluded are those with hypersensitivity to study drugs, positive HIV/Hepatitis tests, tuberculosis infection, certain CDC42 mutation syndromes, recent anti-rejection/immunomodulatory drug use except some exceptions like glucocorticoids.

What is being tested?

The trial is testing the effectiveness of MAS825 compared to a placebo in treating autoinflammatory conditions caused by specific genetic mutations. It's a Phase 2 study focusing on safety and how well patients tolerate the treatment.

What are the potential side effects?

While not explicitly listed in the provided information, potential side effects may include reactions related to immune system modulation such as inflammation or allergic responses due to hypersensitivity history exclusion criteria.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I weigh at least 3 kg.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I weigh more than 160 kg.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ screening through eos (end of study)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~screening through eos (end of study)

This trial's timeline: 3 weeks for screening, Varies for treatment, and screening through eos (end of study) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Cohort 1: Occurrence of disease flare in patients with MAS825 treated patients compared with placebo during Period 2 assessed by Physician's Global Assessment and inflammatory markers

Secondary study objectives

All cohorts: Confirmation of serological markers of MAS825

All cohorts: Number and severity of safety assessments and adverse events

All cohorts: Patient / Parent global assessment of disease activity (PPGA) scale

+5 more

Side effects data

From 2021 Phase 2 trial • 140 Patients • NCT04382651

16%

Acute respiratory failure

13%

Acute kidney injury

Acute respiratory distress syndrome

Pneumonia bacterial

Hypoxia

Respiratory failure

Hypotension

Atrial fibrillation

Septic shock

Anxiety

COVID-19

Pancytopenia

Cardiac arrest

COVID-19 pneumonia

Sepsis

Multiple organ dysfunction syndrome

Urinary tract infection

Cardiogenic shock

Cardiac failure acute

Acute coronary syndrome

Metabolic encephalopathy

Haemophagocytic lymphohistiocytosis

Alanine aminotransferase increased

Fungaemia

Myocarditis

Hypothermia

Pneumomediastinum

Metabolic acidosis

Cerebrovascular accident

Pulmonary embolism

Respiratory arrest

Nephropathy

Pneumothorax

Distributive shock

Disseminated intravascular coagulation

Pneumonia klebsiella

Aspartate aminotransferase increased

100%

80%

60%

40%

20%

Study treatment Arm

MAS825 + SoC

Placebo + SoC

Total

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MAS825Experimental Treatment1 Intervention

Experimental drug

Group II: PlaceboPlacebo Group1 Intervention

matching placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

MAS825

2019

Completed Phase 3

~230

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for X-linked Autoinflammation with Infantile Enterocolitis Syndrome (XLAIES) involve immunomodulatory agents that target specific immune pathways to control the hyperactive immune response. These treatments are essential for managing the excessive inflammation seen in XLAIES patients. For example, agents targeting IL-18 driven autoinflammatory pathways, similar to those studied in the MAS825 trial, can help reduce inflammation by modulating the activity of immune cells involved in these pathways. This targeted approach is crucial for improving clinical outcomes and reducing the severity of symptoms in XLAIES patients.

Case report: Novel treatment regimen for enterovirus encephalitis in SCID.Case Report: Refractory Autoimmune Gastritis Responsive to Abatacept in LRBA Deficiency.Necrotizing enterocolitis as an adverse effect of recombinant interleukin-2 and Ch14.18 in maintenance therapy for high-risk neuroblastoma.