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

Phase 2

Recruiting

• Conditions: RC4-GOF, XIAP Deficiency, CDC42 Mutations

• Registration Number: JPRN-jRCT2041200077
• Lead Sponsor: Hirano Takamitsu

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-12-16
• Last Update Posted: 22 January 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 1

Inclusion Criteria

1.Male and female patients weighing at least 3 kg
2.Written informed consent by parent(s)/legal guardian(s) for the pediatric patients and assent by the pediatric patient (depending on local requirements) must be obtained before any study-specific assessment is performed. For adult patients, written informed consent by patients capable of giving consent, or when the patient is not capable of giving consent, by his/her legal/authorized representative (if allowed according to local requirements).
3.Patients with genetic diagnosis of NLRC4-GOF, XIAP deficiency, or CDC42 mutation
4.Clinical history and investigations consistent with autoinflammation with infantile enterocolitis (AIFEC/NLRC4-GOF), XIAP or CDC42. XIAP patients must have persistent disease or be resistant to escalating therapy.
5.At first treatment, evidence of active disease

Exclusion Criteria

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 systemic recurrent and/or evidence of active bacterial, fungal, parasitic or viral infections.
- 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 thewith 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.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. 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. Female patients of child-bearing potential (or Tanner stage 2 or above) who are or might become sexually active, 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified