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RNAi Therapeutics

Nedosiran for Primary Hyperoxaluria (PHYOX8 Trial)

Phase 2
Recruiting
Research Sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 180 days
Awards & highlights

Summary

This trial will study the effects of nedosiran in children with Primary Hyperoxaluria, a kidney disease.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While specific side effects of nedosiran are not listed here, similar drugs can cause reactions like flu-like symptoms, liver issues indicated by blood tests changes, severe skin reactions at injection sites, and potential allergic responses.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~180 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and 180 days for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Efficacy: Absolute change in urinary oxalate to creatinine ratio
Efficacy: Percent change in urinary oxalate to creatinine ratio
Secondary outcome measures
Efficacy: eGFR changes
Safety: Changes from baseline in ECG: PR interval
Safety: Changes from baseline in ECG: QRS duration
+21 more
Other outcome measures
Change from Baseline in Plasma Oxalate Concentration
Change from Baseline number of kidney stones
Change from baseline PedsQL™
+1 more

Trial Design

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.

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Primary Hyperoxaluria (PH) treatments focus on reducing oxalate production to prevent kidney stones and renal damage. Nedosiran, an RNA interference therapy, targets and silences the gene for glycolate oxidase, an enzyme critical in oxalate synthesis. By reducing the activity of this enzyme, nedosiran lowers oxalate levels, addressing the root cause of the disease. This is vital for PH patients as it can decrease the incidence of kidney stones and help maintain kidney function.
The role of reactive oxygen species derived from different NADPH oxidase isoforms and mitochondria in oxalate-induced oxidative stress and cell injury.In vivo oxalate degradation by liposome encapsulated oxalate oxidase in rat model of hyperoxaluria.

Find a Location

Who is running the clinical trial?

Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyLead Sponsor
17 Previous Clinical Trials
540 Total Patients Enrolled
1 Trials studying Primary Hyperoxaluria
43 Patients Enrolled for Primary Hyperoxaluria
Dicerna Pharmaceuticals, Inc.Lead Sponsor
14 Previous Clinical Trials
492 Total Patients Enrolled
1 Trials studying Primary Hyperoxaluria
43 Patients Enrolled for Primary Hyperoxaluria
Alexandra Haagensen, MD, MBAStudy ChairDicerna Pharmaceuticals, Inc.
3 Previous Clinical Trials
33 Total Patients Enrolled

Media Library

nedosiran (RNAi Therapeutics) Clinical Trial Eligibility Overview. Trial Name: NCT05001269 — Phase 2
Primary Hyperoxaluria Research Study Groups: Open-Label DCR-PHXC
Primary Hyperoxaluria Clinical Trial 2023: nedosiran Highlights & Side Effects. Trial Name: NCT05001269 — Phase 2
nedosiran (RNAi Therapeutics) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05001269 — Phase 2
~3 spots leftby Dec 2024