Nedosiran for Primary Hyperoxaluria (PHYOX8 Trial)

Palo Alto (17 mi)

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.

Treatment Details

The 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.

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.