NCT07024563招募中3 期
A Phase 3, Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Ravulizumab in Pediatric Participants (2 to < 18 Years of Age) With Primary Immunoglobulin A Nephropathy (IgAN)
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 36
- 试验地点
- 17
- 主要终点
- Change from Baseline in Proteinuria Based on Urine Protein to Creatinine Ratio (UPCR) at Week 34
研究概览
简要总结
The primary objectives of this study are to characterize ravulizumab pharmacokinetics (PK) and pharmacodynamics (PD), and to evaluate safety and efficacy following ravulizumab IV dosing in pediatric participants with IgAN or IgAVN.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be 2 to < 18 years of age at the time of signing the informed consent or assent.
- •Stable and maximum allowed or tolerated RAASI (ACEI and/or ARB) dose for ≥ 3 months prior to Screening with no planned change during Screening through Week
- •UPCR ≥ 1.0 g/g from the mean of 3 first morning voids (FMV) collected within 1 week during the Screening Period
- •Estimated GFR ≥ 30 mL/min/1.73 m2 during Screening
- •Meningococcal infection vaccine
- •Haemophilus influenzae type b and Streptococcus pneumoniae vaccine
- •Participants who are receiving SGLT2i, DEARA (eg, sparsentan), MRA, ERA, or GLP-1 agonists must be on a stable and maximum allowed or tolerated dose for ≥ 3 months prior to Screening with no planned change in dose through Week
- •Established diagnosis of primary IgAN diagnosis based on kidney biopsy within 3 years prior to Screening or during the Screening Period
排除标准
- •Diagnosis of rapidly progressive glomerulonephritis
- •Secondary forms of IgAN not in the context of primary IgAN or IgAV
- •Concomitant clinically significant renal disease other than IgAN or IgAVN
- •Clinical remission of IgAN/IgAVN or clinically significant improvement in proteinuria within the last 6 months.
- •Uncontrolled diabetes mellitus with HbA1c > 8.5%
- •History of kidney transplant or planned kidney transplant during the Primary Evaluation Period.
- •History of other solid organ (heart, lung, small bowel, pancreas, or liver) or bone marrow transplant
- •Splenectomy or functional asplenia
- •Participants with nephrotic syndrome receiving albumin infusions or with acute kidney injury requiring dialysis within the last 6 months prior to Screening.
- •Hemolytic uremic syndrome diagnosed any time prior to Screening.
- •Planned urological surgery expected to influence kidney function within the study time frame.
- •Congenital immunodeficiency
- •Active systemic bacterial, viral, or fungal infection within 14 days prior to enrollment
- •Received biologics for the treatment of IgAN or IgAVN within≤ 6 months prior to Screening
研究组 & 干预措施
Ravulizumab
Experimental
All participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and either once every 8 weeks (q8w) thereafter or once every 4 weeks (q4w) depending on weight.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Change from Baseline in Proteinuria Based on Urine Protein to Creatinine Ratio (UPCR) at Week 34
时间窗: Baseline, Week 34
次要结局
- Change from Baseline in eGFR(Baseline, Weeks 50 and 106)
- Number of Participants With Treatment Emergent Adverse Events, Treatment Emergent Serious Adverse Events and Adverse Events of Special Interest(Baseline up to Week 106)
- Maximum Observed Plasma Concentration (Cmax) of Ravulizumab(Baseline up to Week 34)
- Trough Serum Concentration (Ctrough) of Ravulizumab(Baseline up to Week 34)
- Change From Baseline in Serum Free Complement Component 5 (C5) Concentration(Baseline up to Week 34)
- Change from Baseline in proteinuria based on Urine Protein to Creatinine Ratio (UPCR) at Week 10(Baseline, Week 10)
- Change from Baseline in Albuminuria based on Urine Albumin to Creatinine Ratio (UACR) at Week 34(Baseline, Week 34)
- Number of Participants with Partial Remission(Week 34)
- Annualized Total Estimated Glomerular Filtraion Rate (eGFR) over 106 weeks(Baseline up to Week 106)
- Number of Participants with UPCR <0.5 gram of protein per gram of creatinine(Week 34)
- Number of Participants with Antidrug Antibodies to Ravulizumab and Neutralizing Antibodies(Baseline up to Week 106)
研究者
研究点 (17)
Loading locations...
相似试验
已完成
1 期
Study to Evaluate the Effect of IW-3718 on the Pharmacokinetics of Oral Contraceptive, Levothyroxine, Glyburide, and Digoxin in Healthy Adult VolunteersHealthy VolunteersNCT03856970Ironwood Pharmaceuticals, Inc.87
已完成
3 期
Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating MutationCystic FibrosisNCT01705145Vertex Pharmaceuticals Incorporated35
已完成
3 期
Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia GravisGeneralized Myasthenia GravisNCT04735432argenx110
已完成
2 期
Pharmacokinetics, Safety, and Efficacy of Elvitegravir/Cobicistat/Emtricitabine/Tenofovir Disoproxil Fumarate Single Tablet Regimen (STR) in AdolescentsAcquired Immunodeficiency SyndromeHIV InfectionsNCT01721109Gilead Sciences50
已完成
3 期
A Study of Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic ArthritisArthritis, JuvenileNCT05083182Janssen Research & Development, LLC48