CTIS2023-505497-14-00招募中1 期
A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects with Selected Proteinuric Glomerular Diseases (EPPIK) - RTRX-RE021-201
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 57
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 17（—）
- 性别
- All
入选标准
- •1. For All Subjects (Both Populations) - The subject or parent/legal guardian is willing and able to provide signed informed consent/assent, the subject is willing to provide assent before any screening procedures., 2. For All Subjects (Both Populations) - The subject has an eGFR =30 mL/min/1.73 m2 at screening., 3. For All Subjects (Both Populations) - The subject has a mean seated blood pressure between the 5th and 95th percentile for sex and height., 1. For Population 1 - Male or female =1 year at screening to <18 years of age at Day 1., 2. For Population 1 - Has a UP/C =1.5 g/g (170 mg/mmol) at screening AND one of the following: • Kidney biopsy-proven FSGS or MCD histological patterns and clinical presentation consistent with primary FSGS or MCD and qualifying proteinuria at screening despite history or ongoing treatment with corticosteroids and/or other immunosuppressive disease-modifying agents • Documentation of a genetic mutation in a podocyte protein associated with FSGS or MCD. Subjects with a documented podocytic mutation do not require kidney biopsy • Kidney biopsy-proven FSGS histological pattern with medical history and clinical presentation consistent with maladaptive cause of the lesion., 1. For Population 2 - Male or female =2 years to <18 years of age at Day 1 (Baseline)., 2.For Population 2 - Has UP/C =0.6 g/g (68 mg/mmol) at screening AND one of the following: • Kidney biopsy-confirmed IgAN, IgAV or AS • Diagnosis of AS by genetic testing (pathogenic X-linked COL4A5 mutation OR autosomal-recessive mutations in both alleles of COL4A3 and/or COL4A4 OR autosomal-dominant COL4A3 and/or COL4A4 and digenic mutations [ie, simultaneous mutations in 2 of the COL4A3, COL4A4, and COL4A5 genes].
排除标准
- •Weighs <7.3 kg at screening, Has clinically significant congenital vascular disease, Has jaundice, hepatitis, or known hepatobiliary disease, or ALT and/or AST >2 times the UL of normal at screening, Has a history of malignancy within the past 2 years, Has a screening hematocrit <27% (0.27 L/L) or a hemoglobin value <9 g/dL (90 g/L), Has a screening potassium value >5.5 mEq/L (5.5 mmol/L), Has any abnormal clinical laboratory screening values that are considered to be clinically significant, Has a history of allergy to any Ang II antagonist or ERA, including sparsentan, or has a hypersensitivity to any of the excipients, Female is pregnant, plans to become pregnant during the course of the study, or is breastfeeding, Female of childbearing potential who do not agree to use 1 highly reliable method of contraception from 7 days before the first dose of the study medication until 28 days after the last dose of study medication and have a - serum pregnancy test at screening and a - urine pregnancy, with + results confirmed by serum, at every study visit, Has participated in a study of another study medication within 28 days before screening or plans to participate in such a study during the course of this study, Has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies, The subject has had prior exposure to sparsentan, The subject or parent/legal guardian is unable to adhere to the requirements of the study, Has immunoglobulin A (IgA) glomerular deposits not in the context of primary IgAN or IgAV (ie, secondary to another condition, The subject has had an acute onset or presentation of glomerular disease or a diagnostic biopsy or a relapse of glomerular disease requiring new or different class of immunosuppressive treatment within 6 months before screening, Taking chronic immunosuppressive medications and not on a stable dose for =1 month before screening, Requires any of the prohibited concomitant medications, Has undergone any organ transplantation, with the exception of corneal transplants, Has a documented history of congenital or acquired heart failure and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema, Has hemodynamically significant cardiac valvular disease
研究者
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