CTIS2023-504419-34-00招募中1 期
A three-period multicenter study, with a randomized-withdrawal, double-blinded, placebo-controlled design to evaluate the clinical efficacy, safety and tolerability of MAS825 in patients with monogenic IL-18 driven autoinflammatory diseases, including NLRC4-GOF, XIAP deficiency, or CDC42 mutations. - CMAS825D12201
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 19
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 64（—）
- 性别
- All
入选标准
- •All cohorts: 1.Male and female patients weighing at least 3 kg, All cohorts: 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)., Cohort 1: 3.Patients with a genetic diagnosis of either NLRC4-GOF, XIAP deficiency, or CDC42 mutation., Cohort 1: 4.Clinical history and investigations consistent with autoinflammation with infantile enterocolitis (AIFEC/NLRC4-GOF), XIAP or CDC42., Cohort 1: 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.
排除标准
- •1.History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients., 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., 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.
研究者
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