2024-511214-20-00撤回2 期
The MEMORINEPH study: Memory B cell level-based tailored treatment with Rixathon in children with complicated forms of steroid-sensitive Nephrotic syndrome
Ospedale Pediatrico Bambino Gesu'2 个研究点 分布在 1 个国家目标入组 80 人开始时间: 2024年10月25日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 入组人数
- 80
- 试验地点
- 2
- 主要终点
- Evaluation of the number of RTX re-infusions comparing the two therapeutic regimens based on flow cytometry assessment of total B cell reconstitution (Arm A) or memory (Arm B) during a 24-month follow-up
研究概览
简要总结
To define the optimal number of RTX re-infusions in children with complicated forms of FRNS/SDNS through an open-label randomized controlled trial comparing two different regimens for repeated RTX treatment based on the reconstitution of total B cells (Arm A) or memory B cells (Arm B).
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •1.Prior to any study procedure, informed consent must be obtained in written form from the adult patient or from both parents/legal guardians of the pediatric patient. For adolescent and school-aged patients, assent will be obtained with age-appropriate information. 2.Children and young adults (aged 3 to 24 years) with complicated forms of FRNS/SDNS (see "Definitions") who have at least 2 relapses per year while on mycophenolate mofetil (MMF) and/or a calcineurin inhibitor at appropriate doses (3-5 mg/kg in 2 daily doses). 3.Previous infusions of RTX will be allowed. 4.Patients, if of legal age, and parents or legal guardians if minors, must be able to communicate adequately with the medical team and understand and carry out the necessary procedures for completing the study. 5.For patients with reproductive potential (females), the use of a reliable contraceptive method (e.g., abstinence, barrier contraception, hormonal contraception) for the entire duration of study participation is required.
排除标准
- •At screening, patients with any of the following characteristics will not be considered eligible for enrollment:
- •Corticosteroid resistance, defined as lack of response to 4 weeks of oral prednisone at standard dosage.
- •Onset of nephrotic syndrome after the age of
- •Secondary nephrotic syndrome.
- •Past or present malignant tumors of any organ or system, even in the absence of active or previous metastatic signs or symptoms.
- •Pregnancy (defined as the condition of a female individual from conception until the end of gestation, confirmed by a positive laboratory test for beta human chorionic gonadotropin (hCG)) or breastfeeding. Women of childbearing age (from menarche onward) must use an effective contraceptive method (complete abstinence, barrier contraception, or hormonal contraception) throughout the duration of the study and for 12 months following the last study RTX infusion.
- •Lack of response to rituximab or mycophenolate mofetil, defined as persistence of nephrotic proteinuria >4 weeks despite such therapy.
- •Active severe infection, for example, hepatitis B (positive HBsAg, HBcAb), sepsis, or active tuberculosis (positive Quantiferon).
- •Severe immunosuppression, defined as congenital immunodeficiency and/or as circulating immunoglobulin levels 2x lower than the minimum normal range for age and/or as circulating neutrophil granulocytes <1.5x10^3/μl.
- •Renal insufficiency with eGFR < 15 ml/min/1.73 m² calculated using the modified Schwartz formula (for children) or the Cockcroft-Gault formula (for adults).
- •Severe heart failure (New York Heart Association class IV) or severe uncontrolled heart disease.
- •Known hypersensitivity to RTX treatment.
- •Known hypersensitivity to concomitant medications (prednisone, mycophenolate mofetil/mycophenolic acid, calcineurin inhibitors).
- •Use of live attenuated vaccines within 28 days prior to enrollment and throughout the duration of the study.
- •Weight < 10 kg.
- •Thrombocytopenia, defined as platelet level <100,000/mm².
- •Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels >2.5x the upper limit of the normal range for age and sex.
结局指标
主要结局
Evaluation of the number of RTX re-infusions comparing the two therapeutic regimens based on flow cytometry assessment of total B cell reconstitution (Arm A) or memory (Arm B) during a 24-month follow-up
Evaluation of the number of RTX re-infusions comparing the two therapeutic regimens based on flow cytometry assessment of total B cell reconstitution (Arm A) or memory (Arm B) during a 24-month follow-up
次要结局
- Assessment during a 24-month follow-up of: • The number of relapses (defined as the reappearance of massive proteinuria (>40 mg/m²/h in children or urine protein/creatinine ratio > 2 g/g) or a positive urine dipstick (≥3+ for 3 days or positive for 7 days), with or without edema); • The number of hospitalization days; • The number of severe infections (defined as infections requiring hospitalization); • The time to the first relapse or a positive urine dipstick
- Exploratory endpoints: Evaluation using immunoturbidimetry and ELISA tests of serum levels of total immunoglobulins IgG, IgM, and IgA, as well as antibody titers specific to tetanus and HBV induced by vaccination. • Assessment of the incidence of prolonged hypogammaglobulinemia (defined as IgG levels < age-specific normal range) and/or severe hypogammaglobulinemia (IgG < 250 mg/dl), with potential need for the introduction of replacement therapy with IgG.
研究者
Marina Vivarelli
Scientific
Ospedale Pediatrico Bambino Gesu'
研究点 (2)
Loading locations...
相似试验
招募中
4 期
Evaluating, clinical and immunological effects of rituximab with cyclophosphamide compared to rituximab alone in AAV patients2023-507868-39-00Academisch Ziekenhuis Leiden100
招募中
2 期
Bosutinib in pediatric CML patients2023-504311-32-00Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)30
招募中
3 期
Comparison of neurocognitive outcome in two standard regimen for treatment of low-risk medulloblastoma (COGNITO-MB)2024-517133-40-00GPOH gGmbH46
招募中
2 期
Evaluation of the efficacy and safety of Rituximab in patients with Amyotrophic Lateral Sclerosis (ALS)2022-502743-35-00Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.52
尚未招募
3 期
Comparison of Neurocognitive Outcome in Two Standard Regimen for Treatment of Low-risk MedulloblastomaNCT07291102Nationwide Children's Hospital96