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Label indication RIVFLOZA is indicated to lower urinary oxalate levels in children 2 years of age and older and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function, e.g., eGFR ≥30 mL/min/1.73 m 2 [see Clinical Pharmacology ( 12.3 )], Clinical Studies ( 14.1 )]. FDA label
Also known as DCR-PHXC
nedosiran · 6 trials · 11 indications
To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1
This outcome measure reported percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed primary hyperoxaluria type 1 (PH1), primary hyperoxaluria type 2 (PH2), or primary hyperoxaluria type 3 (PH3) subgroups.
This outcome measure reported absolute change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed PH1, PH2, or PH3 subgroups.
To assess the efficacy of DCR-PHXC in lowering Pox in participants with PH1 and severe renal impairment, with or without hemodialysis or peritoneal dialysis.
The AUC of 24-hour urinary oxalate (Uox) from Day 90 to Day 180, based on percent change from baseline, was compared between the active treatment group and placebo group. A multiple imputation approach was used to handle missing Uox data and then calculate the AUC.
Number of patients with abnormalities in clinically significant laboratory results, vital signs, and 12-lead ECG findings
| Arm | Type | Description |
|---|---|---|
| Open Label | EXPERIMENTAL | Open label, monthly subcutaneous injection |
| Open-Label DCR-PHXC | EXPERIMENTAL | Open-Label monthly subcutaneous injection of DCR-PHXC based on age and weight. |
| DCR-PHXC | EXPERIMENTAL | Intervention, drug, DCR-PHXC |
| Placebo - Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | Placebo, sterile normal saline (0.9% NaCl) for subcutaneous (SC) injection |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | Participants will receive a single dose of Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo. |
| Group A Active (DCR-PHXC) | EXPERIMENTAL | HVs, single ascending doses of DCR-PHXC. |
| Group A Placebo | PLACEBO_COMPARATOR | HVs, normal saline 0.9% injection to match active doses. |
| Group B Active (DCR-PHXC) | EXPERIMENTAL | PH1 and PH2 patients, open label, single ascending doses of DCR-PHXC. |
| Name | Type | Description |
|---|---|---|
| DCR-PHXC | DRUG | Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection |
| nedosiran | DRUG | Monthly subcutaneous dosing throughout study period |
| Sterile Normal Saline (0.9% NaCl) | DRUG | Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo |
| Placebo | DRUG | Single SC administration of placebo, which will be a sterile, preservative-free normal saline 0.9% solution for SC injection, which is of similar osmolality to the DCR-PHXC formulation. |
Key Inclusion Criteria: •Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have ge...
Nedosiran is being developed for the treatment of primary hyperoxaluria, a rare inherited disorder. It is an investigational RNA therapy studied in pediatric patients from birth to 11 years of age with primary hyperoxaluria and relatively intact renal function. It has not been approved by the FDA.
Nedosiran is a small interfering RNA (siRNA) therapy, part of the -siran drug class. siRNA therapies are designed to reduce production of specific proteins by interfering with messenger RNA. The specific molecular target of nedosiran is not stated in the available information.
Nedosiran is being developed by Novo Nordisk A/S, which trades under the ticker NVO. Novo Nordisk is the company responsible for the clinical development program for this investigational RNA therapy in primary hyperoxaluria.
Nedosiran is in Phase 2 clinical development. It is an investigational therapy and has not been approved by the FDA. The Phase 2 trial has been completed, and no active trials are currently listed for nedosiran.
Nedosiran has been studied in one completed Phase 2 clinical trial, NCT05001269, titled "Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function." The trial enrolled 27 participants across the United States, Canada, Germany, Italy, Japan, Lebanon, Poland, Spain, Turkey, the United Arab Emirates, and the United Kingdom.