Study to evaluate the efficacy and safety of MAS825 in patients with autoinflammatory diseases.

Phase 1

• Conditions: Autoinflammatory diseases including NLRC4-Gain of Function (GOF) also known as AIFEC (autoinflammation with infantile enterocolitis), X-linked Inhibitor of Apoptosis Protein (XIAP) deficiency, or Cell division control protein 42 homolog (CDC42) mutation.; MedDRA version: 23.1Level: PTClassification code 10084306Term: Autoinflammation with infantile enterocolitisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 22.0Level: PTClassification code 10078961Term: Immune-mediated enterocolitisSystem Organ Class: 10017947 - Gastrointestinal disorders; MedDRA version: 20.0Level: SOCClassification code 10021428Term: Immune system disordersSystem Organ Class: 10021428 - Immune system disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2020-003596-17-CZ
• Lead Sponsor: ovartis Pharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-01-27
• Last Update Posted: 18 June 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

All cohorts:
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 or her legal/authorized representative (if allowed according to local requirements).
3.Patients with a genetic diagnosis of either NLRC4-GOF, XIAP deficiency, or CDC42 mutation.
4.Clinical history and investigations consistent with autoinflammation with infantile enterocolitis (AIFEC/NLRC4-GOF), XIAP or CDC42.
5.At first treatment, evidence of active disease
Cohort 2:
6.Patients with a genetic diagnosis of NLRC4-GOF, XIAP deficiency, or CDC42 mutations who are being treated with MAS825 in a Novartis managed access program.
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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, excluding chronic Epstein-Barr Virus (EBV).
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 prior to MAS825 treatment with the exceptions of:
-glucocorticoids
-cyclosporin
-targeted binding or blocking therapies, which must be discontinued prior to treatment with MAS825
-drugs listed as prohibited medication in the protocol, for which the washout periods should be followed as outlined in the protocol.
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. 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, agree to use highly effective contraceptive methods to prevent pregnancy while on MAS825 therapy
10.Patients weighing >160 kg at Screening.
11.For CDC42 mutation patients: Takenouchi-Kosaki syndrome – CDC42 mutations associated with a diverse syndrome characterized by variable development delays, cardiac, brain and hematological abnormalities.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified