EUCTR2021-000621-27-DE进行中（未招募）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). - EPPIK
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 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 clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •For All Subjects (Both Populations)
- •A subject must meet all of the following criteria to be eligible for participation in this study:
- •1. The subject or parent/legal guardian (as appropriate) is willing and able to provide signed informed consent/assent, and where required, the subject is willing to provide assent before any screening procedures per local requirements.
- •2. The subject has an eGFR =30 mL/min/1.73 m2 at screening.
- •3. The subject has a mean seated blood pressure between the 5th and 95th percentile for sex, and height (Banker 2016).
- •For Population 1
- •1. The subject is male or female =1 year at screening to <18 years of age at Day 1 (Baseline).
- •2. The subject 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.
- •For Population 2
- •1. The subject is male or female =2 years to <18 years of age at Day 1 (Baseline).
- •2. The subject 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])
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 57
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •For All Subjects (Both Populations)
- •A subject who meets any of the following criteria will be excluded from this study:
- •1. The subject weighs <7.3 kg at screening.
- •2. The subject has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies.
- •3. The subject has immunoglobulin A (IgA) glomerular deposits not in the context of primary IgAN or IgAV (ie, secondary to another condition eg, systemic lupus erythematosus and liver cirrhosis).
- •4. 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 (including, but not limited to, systemic corticosteroids, calcineurin inhibitors and mycophenolate mofetil abatacept, cyclophosphamide, rituximab, ofatumumab, and ocrelizumab) within 6 months before screening.
- •5. Subjects taking chronic immunosuppressive medications (including systemic steroids) not on a stable dose for =1 month before screening.
- •6. The subject requires any of the prohibited concomitant medications as defined in the study protocol.
- •7. The subject has undergone any organ transplantation, with the exception of corneal transplants.
- •8. The subject has a documented history of congenital or acquired heart failure (modified Ross heart failure classification for children Class II to Class IV [Ross 2012]) and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema.
- •9. The subject has hemodynamically significant cardiac valvular disease.
- •10. The subject has clinically significant congenital vascular disease.
- •11. The subject has jaundice, hepatitis, or known hepatobiliary disease, or alanine aminotransferase and/or aspartate aminotransferase >2 times the upper limit of the normal range at screening.
- •12. The subject has a history of malignancy within the past 2 years.
- •13. The subject has a screening hematocrit <27% (0.27 L/L) or a hemoglobin value <9 g/dL (90 g/L).
- •14. The subject has a screening potassium value >5.5 mEq/L (5.5 mmol/L).
- •15. The subject has any abnormal clinical laboratory screening values that are considered by the Investigator to be clinically significant.
- •16. The subject has a history of allergic response to any Ang II antagonist or ERA, including sparsentan, or has a hypersensitivity to any of the excipients in the study medication.
- •17. The female subject is pregnant, plans to become pregnant during the course of the study, or is breastfeeding
- •18. Female subjects of childbearing potential, beginning at menarche, who do not agree to use 1 highly reliable (ie, can achieve a failure rate of <1% per year) method of contraception from 7 d before the first dose of the study medication until 28 d after the last dose of study medication. Examples of highly reliable contraception methods include stable oral, implanted, transdermal, or injected contraceptive hormones associated with the inhibition of ovulation or an intrauterine device (IUD) in place for at least 3 m. One additional barrier method must also be used during vaginal sexual activity, such as a diaphragm, diaphragm with spermicide (preferred), or male partner’s use of male condom or male condom with spermicide (preferred), from Day 1/Randomization until 28 d after the last dose of study medication. Female subjects of childbearing potential are defined as those who are fertile after menarche, unless permanently
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