2024-520167-13-00招募中3 期
A Phase 3, Open-Label, Single-Arm, 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)
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 6
- 主要终点
- To characterize the PK and PD of treatment with ravulizumab IV in pediatric participants to support the extrapolation of efficacy from the adult population
研究概览
简要总结
To characterize the PK and PD of treatment with ravulizumab IV in pediatric participants to support the extrapolation of efficacy from the adult population
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Single arm
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Participant must be ≥ 2 to < 18 years of age inclusive, at the time of signing the informed consent or assent.
- •The Investigator, or a person designated by the Investigator, will obtain written informed consent from each study participant’s legal guardian and the participant’s assent, when applicable, before any study-specific activity is performed. All legal guardians should be fully informed, and participants should be informed to the fullest extent possible, about the study in language and terms they are able to understand.
- •Legal guardian or primary caregiver must be able to accurately maintain the child’s take-home record, including items of general health.
- •Established diagnosis of primary IgAN diagnosis based on kidney biopsy obtained prior to Day
- •Established diagnosis of IgAVN based on kidney biopsy obtained prior to Day
- •If receiving immunosuppressive medications (eg, corticosteroid, cyclophosphamide, CNIs, mizoribine, or MMF) for the treatment of the renal manifestations of IgAVN, the medication must be stable for ≥ 1 month prior to Screening.
- •UPCR ≥ 0.5 g/g from the mean of 3 FMVs collected within 1 week during the Screening Period (should be collected at home).
- •eGFR ≥ 30 mL/min/1.73 m2 during Screening as calculated by the CKiD U25 equation (Pierce, 2021).
- •Adherence to and compliance with stable (weight-adjusted per PI's discretion) and maximum allowed or tolerated RASI (ACEI and/or ARB) dose for ≥ 3 months prior to Screening with no planned change in dose (weight-adjusted per PI's discretion) during Screening through Week
- •Participants with intolerance to RASI medications may be included.
- •Body weight ≥ 10 kg at Screening.
- •Male or female (according to their reproductive organs and functions assigned by chromosomal complement) (FDA, 2020) assigned at birth, inclusive of all gender identities.
- •Contraceptive and barrier use as well as pregnancy testing as appropriate for the age and sexual activity of pediatric participants and/or as required by local regulations as outlined in the protocol.
- •To reduce the risk of meningococcal infection (N meningitidis), all participants must be vaccinated against meningococcal infection from serogroups A, C, W135, and Y (and serogroup B, where available) within 3 years and at least 2 weeks prior to the first dose of study intervention. If vaccination occurs < 2 weeks from the first dose of study intervention, the participant will receive prophylactic antibiotics for at least 2 weeks after initial vaccination. Vaccinations must follow national/local guidelines.
- •Must have received vaccination against Haemophilus influenzae type b and Streptococcus pneumoniae unless previously vaccinated according to current national and local vaccination guidelines.
排除标准
- •Diagnosis of rapidly progressive glomerulonephritis as measured by eGFR loss ≥ 50% over a period of 3 months prior to Screening.
- •Drug or alcohol abuse or dependence within 1 year prior to Screening that interferes with ability to participate in the clinical study.
- •Secondary forms of IgAN not in the context of primary IgAN or IgAV (eg, due to systemic lupus erythematosus, cirrhosis, or celiac disease).
- •Concomitant clinically significant renal disease other than IgAN or IgAVN.
- •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; or planned transplant during the Primary Evaluation Period or Extension Period, except for corneal 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.
- •Systemic BP > 90th percentile for sex and height.
- •History of malignancy within 5 years of Screening, except for nonmelanoma skin cancer or carcinoma in situ of the cervix that has been treated with no evidence of recurrence.
- •Hemolytic uremic syndrome diagnosed any time prior to Screening.
- •Hypersensitivity to any ingredient contained in the study intervention, including murine proteins.
- •Received biologics for the treatment of IgAN or IgAVN ≤ 6 months prior to Screening.
- •Traditional Chinese medicines and Chinese proprietary medicines with systemic immunosuppressive properties including but not limited to Tripterygium wilfordii or Tripterygium wilfordii-containing medicines for the treatment of IgAN or IgAVN ≤ 6 months prior to Screening.
- •Received a complement inhibitor ≤ 30 days or 5 half-lives, whichever is longer, prior to Screening.
- •Current enrollment or past participation in any other clinical study in which an investigational intervention (eg, drug, vaccine, invasive device) was administered within 5 half-lives (if known) days before signing of consent in this clinical study.
- •Pregnant, breastfeeding, or intending to conceive during the study and within 8 months after the last dose of the study intervention.
- •Inability to travel to the clinic for specified visits during the study or fulfill the logistical requirements of study intervention administration.
- •Participant, parent, or legal guardian is involved in the planning and/or conduct of the study (applies to both Sponsor personnel and/or site personnel).
- •Received systemic immunosuppression (eg, corticosteroid, cyclophosphamide, CNIs, mizoribine, or MMF), or budesonide for the treatment of IgAN ≤ 3 months prior to Screening.
- •Received systemic immunosuppression ≤ 3 months of Screening for extrarenal manifestations of IgAV.
- •Planned urological surgery expected to influence kidney function within the study time frame.
- •History of severe IgAV symptoms (eg, including but not limited to orchitis, cerebral vasculitis, pulmonary hemorrhage, gastrointestinal bleeding) within 1 year before Screening.
- •Congenital immunodeficiency.
- •History of unexplained, recurrent infection.
- •Known medical or psychological condition(s), including substance abuse, or risk factor that, in the opinion of the Investigator, might interfere with the participant’s full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.
- •History of or unresolved N meningitidis infection.
- •Known history of HIV, active hepatitis B infection, or active hepatitis C infection.
- •Active systemic bacterial, viral, or fungal infection within 14 days prior to enrollment.
结局指标
主要结局
To characterize the PK and PD of treatment with ravulizumab IV in pediatric participants to support the extrapolation of efficacy from the adult population
To characterize the PK and PD of treatment with ravulizumab IV in pediatric participants to support the extrapolation of efficacy from the adult population
次要结局
未报告次要终点
研究者
European Clinical Trial Information
Scientific
Alexion Pharmaceuticals Inc.
研究点 (6)
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