Nedosiran in Pediatric Patients from Birth to 11 Years of Age with PH1, PH2, or PH3 and Relatively Intact Renal Function

JRCT ID: jRCT2041220026

Registered date:04/06/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Primary hyperoxaluria
• Date of first enrollment: 28/04/2022
• Target sample size: 4
• Countries of recruitment: France,Japan,Germany,Japan,United Kingdom,Japan,United States,Japan,Canada,Japan,Spain,Japan,Italy,Japan,Lebanon,Japan,Poland,Japan,Turkey,Japan
• Study type: Interventional

Intervention(s)

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 participant's 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 weight at Day 90 for participants < 6 months of age at Screening.

Outcome(s)

• Primary Outcome: Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in PH1, PH2, or PH3 participant subgroups

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: < 12age old
• Gender: Both

Include 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

Exclude 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

Related Information

• Primary Sponsor: Bowman Thomas
• Secondary ID(s): NCT05001269