A Phase 2 Open-Label Multicenter Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Nedosiran in Pediatric Patients from Birth to 11 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function - PHYOX8

Bowman Thomas0 个研究点目标入组 4 人开始时间: 2022年6月4日最近更新: 2023年10月17日

适应症

试验速览

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•Birth to 11 years of age inclusive, at the time of signing the informed consent.
•- Documented diagnosis of PH1, PH2, or PH3 confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
•- Average spot Uox to creatinine ratio at Screening above 2 times the 95th percentile for age
•- Estimated GFR at screening >= 30 mL/min normalized to 1.73 m2 BSA
•- Participants must have been on a stable treatment regimen for PH for 3 months prior to Day 1 and parent(s)/legal guardian should be willing to ensure participant remains on the same stable treatment regimen during the study
•- Male or female
•- Participant's parent or legal guardian is capable of giving signed informed consent, which includes complia
•nce with the requirements and restrictions listed in the ICF and in this protocol
•- A legal guardian or primary caregiver must be available to help the study-site personnel ensure follow up; accompany the participant to the study site on each assessment day according to the SoA (e.g., able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); consistently and consecutively be available to provide information on the participant using the rating scales during the scheduled study visits; accurately and reliably dispense study intervention as directed
•- Affiliated with or is a beneficiary of a health insurance system

•- Prior renal or hepatic transplantation; or planned transplantation within the study period
•- Currently receiving dialysis or anticipating requirement for dialysis during the study period
•- Plasma oxalate (Pox) > 30 micromol/L
•- Documented evidence of clinical manifestations of severe systemic oxalosis
•- Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact participant's safety
•- Use of an RNAi drug within the last 6 months
•- History of reactions to an oligonucleotide-based therapy
•- Participation in any clinical study in which they received an IMP within 4 months or 5 times the half-life of the drug (whichever is longer) before Screening
•- Liver function test (LFT) abnormalities : alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 1.5 x ULN for age and gender
•- Known hypersensitivity to nedosiran, or any of its ingredients
•- Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations detailed in protocol

发起方

Bowman Thomas