EUCTR2020-003596-17-CZ进行中（未招募）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.
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •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
- •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
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.
- •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.
研究者
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