EUCTR2018-004014-17-FR进行中（未招募）1 期
ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 8
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
- •1.Have reached at least 37 weeks estimated gestational age (full-term infant) but less than 6 years of age at consent.
- •2.Documentation of PH1 as determined by genetic analysis prior to initial dosing.
- •3.Urinary oxalate:creatinine ratio greater than the upper limit of normal based on age on at least 2 of 3 single-void collections during screening.
- •4.If taking therapeutic vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days before screening, and is able to remain on this stable regimen until at least the Month 6 visit. Dose adjustments for interval weight gain are acceptable.
- •5.Legal guardian(s) is (are) willing and able to comply with the study requirements and to provide written informed consent; the patient should provide assent per local and national requirements.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 8
- •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
- •1.Has any of the following laboratory parameter assessments:
- •a.Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× ULN for age
- •b.Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is <2× ULN
- •2.Has known active human immunodeficiency virus (HIV) infection; or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection.
- •3.If =12 months old at screening, has an estimated glomerular filtration (GFR) of =45 mL/min/1.73m2 (calculation will be based on the Schwartz Bedside Formula); if <12 months old at screening, has serum creatinine value per the central laboratory above the ULN for age.
- •4.Received an investigational agent within the last 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to randomization. Consultation with Medical Monitor required if treated with unapproved products prior to consent.
- •5.Medical history includes clinical evidence of extrarenal systemic oxalosis as determined by the Investigator.
- •6.Has undergone renal or liver transplantation or a liver transplant is anticipated in the 6 months after the initial dose of lumasiran.
- •7.Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation.
- •8.History of allergic reaction to an oligonucleotide or GalNAc.
- •9.History of intolerance to subcutaneous (SC) injection(s).
- •10.For female patients who may achieve menarche during the study, is not willing to comply with the contraceptive requirements during the study period
研究者
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