Nedosiran for Primary Hyperoxaluria

(PHYOX8 Trial)

Recruiting at 16 trial locations

Overseen By Medical Information

Age: < 18

Sex: Any

Trial Phase: Phase 2

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

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

25 Participants Needed

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.

Research Team

Sarb Shergill, PhD

Principal Investigator

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

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).

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

Up to 5 weeks

Treatment

Participants receive monthly subcutaneous injections of DCR-PHXC based on age and weight

24 weeks

Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• nedosiran

Trial Overview 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.

Participant Groups

Group I: Open-Label DCR-PHXC Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Lead Sponsor

Trials

19

Recruited

580+

Dicerna Pharmaceuticals, Inc.

Lead Sponsor

Trials

15

Recruited

500+

Founded

2007

Headquarters

Watertown, USA

Known For

RNA Therapeutics

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