Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
DCR-PHXC · 5 trials · 10 indications
To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1
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 |
| 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 an investigational small molecule being developed for the treatment of Primary Hyperoxaluria, including Type 1, Type 2, and Type 3. It is also being studied in patients with Primary Hyperoxaluria Type 1 who have end-stage renal disease. The drug is in clinical development for these nephrology indications.
DCR-PHXC is being developed by Novo Nordisk A/S, a pharmaceutical company traded on the New York Stock Exchange under the ticker NVO. The company is conducting clinical trials of DCR-PHXC in multiple countries, including the United States, France, Germany, and the United Kingdom.
DCR-PHXC is in Phase 1 clinical development, with additional trials in Phase 2 and Phase 3. The Phase 1 trials have been completed, a Phase 2 trial is recruiting patients, and a Phase 3 long-term extension study is active but not recruiting. The drug is investigational and not yet approved.
DCR-PHXC has been studied in several clinical trials. NCT03392896 was a Phase 1 study in healthy volunteers and patients with Primary Hyperoxaluria, completed with 43 participants. NCT04042402 is a Phase 3 long-term extension study in patients with Primary Hyperoxaluria, active with 75 participants. NCT04555486 was a Phase 1 study in Type 3 patients, completed with 6 participants. NCT04580420 is a Phase 2 study in PH1 patients with end-stage renal disease, currently recruiting.
DCR-PHXC is also known by the name DCR-PHXC-101, as referenced in the title of the Phase 1 clinical trial NCT03392896. This alternative name may be used in some clinical trial records or publications, but the drug is the same investigational compound.