Primary hyperoxaluria
Conditions
Interventions
Participants will receive monthly SC doses (3.5 mg/kg, not exceed 170 mg) of nedosiran over 6 months.The maximum injection volume in participants is 0.5 mL
if the participants weight requires an injection volume > 0.5 mL, the total dose will be administered as 2 SC injections.
No dose modifications are allowed, except for dose adjustments based upon weig
Sponsors
Bowman Thomas
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - 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
Exclusion criteria
Exclusion criteria: - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in PH1, PH2, or PH3 participant subgroups | — |
Countries
Canada, France, Germany, Italy, Japan, Lebanon, Poland, Spain, Turkey, United Kingdom, United States
Contacts
Public Contactintellim inquiry desk for JRCT
intellim Corporation
Outcome results
None listed