Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Nedosiran
DCR-PHXC · 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...
DCR-PHXC is being developed for primary hyperoxaluria, including primary hyperoxaluria type 1 and type 3. It is an investigational small molecule from Novo Nordisk A/S, studied in nephrology across placebo-controlled trials that have enrolled 138 participants in total.
DCR-PHXC is developed by Novo Nordisk A/S, which trades under the ticker NVO. The company is running the clinical program for the drug in primary hyperoxaluria, spanning phase 1, phase 2 and phase 3 studies.
DCR-PHXC is in clinical development, with trials across phase 1, phase 2 and phase 3. It is investigational and has not been approved. Three trials are listed, two currently active and one completed, with 138 participants enrolled overall.
DCR-PHXC trials include NCT04580420, a recruiting phase 2 study in primary hyperoxaluria type 1 with end stage renal disease; NCT04555486, a completed phase 1 study in type 3; NCT04042402, an active long term extension; and NCT03847909, a completed phase 2 study.
Yes. Two DCR-PHXC trials enroll participants aged 6 years and older: the completed phase 1 study NCT04555486 in primary hyperoxaluria type 3, and the completed phase 2 study NCT03847909 in primary hyperoxaluria type 1 and type 2. Both are open to all sexes.
DCR-PHXC trials run across North America, Europe, the Middle East and Asia-Pacific. Countries include the United States, Canada, the United Kingdom, Germany, France, Italy, Spain, the Netherlands, Japan, Australia, Lebanon and the United Arab Emirates, among others.