Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

17 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-12-18

Study Completion Date

2027-09-11

Brief Summary

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This study is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF, XIAP deficiency, or CDC42 mutations.

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Detailed Description

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This is a three-period study, with an open-label, single-arm active treatment in Period 1 followed by a randomized-withdrawal, double-blinded, placebo-controlled design in Period 2, and an open label, long-term safety follow-up in Period 3 and Period 3s. The total study duration is approximately 4 - 5 years.

Patients who enter Period 2 will be randomized to MAS825 or matching placebo in a 1:1 ratio.

Cohort 1 patients will complete all periods of the study, which will take approximately 5 years.

Cohort 2: Patients who are receiving MAS825 in a Novartis Managed Access Program with a diagnosis of NLRC4-GOF, XIAP deficiency, or CDC42 mutation who meet criteria will be eligible to directly enter into Period 3 and Period 3s for open-label long-term safety follow-up. Cohort 2 patients will be in the study for approximately 4 years.

Conditions

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NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 Mutations

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This study includes:

* Screening
* Period 1: Open-Label Treatment Period to identify responders to MAS825
* Period 2: Randomized Withdrawal Period consists of a randomized treatment withdrawal period to primarily assess the efficacy of MAS825 compared to placebo.
* Period 3: Open-Label, Long-Term Safety follow-up
* Period 3s: Open-Label, transition to new route of administration and safety follow-up
* End of Study

Patients will participate in all 3 periods of the study if they have never been treated with MAS825 before.

Patients who are enrolled from the Managed Access program will participate in Period 3 and Period 3s only after completing screening and baseline assessments.

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Subject, investigator, and sponsor blinding via manual randomization during Period 2, Randomized Withdrawal Period

Study Groups

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MAS825

Experimental drug

Group Type EXPERIMENTAL

MAS825

Intervention Type BIOLOGICAL

Experimental drug

Placebo

matching placebo

Group Type PLACEBO_COMPARATOR

Placebo

1. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients.
2. Signs and symptoms, in the judgment of the investigator, of clinically significant active bacterial, fungal, parasitic or viral infections, excluding chronic Epstein-Barr Virus (EBV).

\- COVID-19 specific: If in line with health and governmental authority guidance, it is highly recommended that testing to exclude COVID-19 using PCR or comparable approved methodology be completed within 1 week prior to first dosing.
3. Any conditions or significant medical problems, which in the opinion of the investigator places the patient at unacceptable risk for MAS825 therapy
4. Previous treatment with anti-rejection and/or immunomodulatory drugs within the past 28 days or 5 half-lives (whichever is the longer) for immunomodulatory therapeutic antibodies (or as listed in the prohibited medications section) prior to MAS825 treatment with the exceptions of glucocorticoids, cyclosporin and targeted binding or blocking therapies.
5. A positive HIV test result at Screening. Evidence of prior testing within 3 months is sufficient.
6. A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C test result at Screening. Evidence of prior testing within 3 months is sufficient.
7. Presence of tuberculosis infection as defined by a positive TB test at Screening. Evidence of prior testing within 3 months is sufficient.
8. Live vaccinations within 1 month prior to MAS825 treatment, during the trial, and up to 3 months following the last dose.
9. Pregnant or nursing (lactating) females.
10. Female patients of child-bearing potential (or Tanner stage 2 or above) who are or might become sexually active, agree to use highly effective contraceptive methods to prevent pregnancy while on MAS825 therapy
11. Patients weighing \>160 kg at Screening.
12. For CDC42 mutation patients: Takenouchi-Kosaki syndrome - CDC42 mutations associated with a diverse syndrome characterized by variable development delays, cardiac, brain and hematological abnormalities.

Minimum Eligible Age

0 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No