Nedosiran for Primary Hyperoxaluria
(PHYOX8 Trial)
Recruiting at 16 trial locations
MI
Overseen ByMedical Information
Age: < 18
Sex: Any
Trial Phase: Phase 2
Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
What You Need to Know Before You Apply
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.
Who Is on the Research Team?
SS
Sarb Shergill, PhD
Principal Investigator
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Are You a Good Fit for This Trial?
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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Timeline for a Trial Participant
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
What Are the Treatments Tested in This Trial?
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.
How Is the Trial Designed?
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.
Find a Clinic Near You
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
Top Products
GalXC, GalXC-Plus
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