Nedosiran for Primary Hyperoxaluria
(PHYOX8 Trial)

Recruiting in Palo Alto (17 mi)

+16 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

This trial is testing nedosiran, a medication, in children with a rare kidney condition called Primary Hyperoxaluria. The goal is to see if it can reduce harmful oxalate levels and protect their kidneys.

Eligibility Criteria

This trial is for children from birth to 11 years with Primary Hyperoxaluria (PH), a genetic disorder affecting the kidneys, who have normal or near-normal kidney function. Participants need a legal guardian to consent and help with study requirements, must not be pregnant or breastfeeding if applicable, and should be on stable PH treatment for at least 3 months.

Inclusion Criteria

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 (if applicable per national regulations)

Documented diagnosis of PH1 or PH2 or PH3 confirmed by genotyping (historically available genotype information is acceptable for study eligibility).

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Exclusion Criteria

- Severe intercurrent illness

Prior renal or hepatic transplantation; or planned transplantation within the study period

Currently receiving dialysis or anticipating requirement for dialysis during the study period

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Treatment Details

Interventions

nedosiran (RNAi Therapeutics)

Trial OverviewThe trial is testing nedosiran, a medication intended to treat Primary Hyperoxaluria in young patients. It aims to evaluate its effectiveness and safety in those who still have relatively good kidney function.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Open-Label DCR-PHXCExperimental Treatment1 Intervention

Open-Label monthly subcutaneous injection of DCR-PHXC based on age and weight.