Approval Probability
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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 being developed for primary hyperoxaluria, a group of rare inherited disorders that cause excess oxalate production and kidney damage. The program covers primary hyperoxaluria type 1, type 2, and type 3, including patients with type 1 who also have end stage renal disease. It is an investigational therapy and has not been approved.
DCR-PHXC is being developed by Novo Nordisk A/S, which trades under the ticker NVO. The company is running the clinical program across multiple countries, including the United States, several European nations, Canada, Australia, Japan, and the Middle East.
DCR-PHXC is in clinical development, with trials spanning Phase 1 through Phase 3. A Phase 1 study in primary hyperoxaluria type 3 has completed, Phase 2 studies in type 1 and type 2 have completed or are recruiting, and a Phase 3 long term extension study is active but not recruiting. It is investigational and not approved.
DCR-PHXC has been studied in several trials. NCT04555486 was a completed Phase 1 study in primary hyperoxaluria type 3. NCT03847909 was a completed Phase 2 study in types 1 and 2. NCT04580420 is a recruiting Phase 2 study in type 1 with end stage renal disease, and NCT04042402 is an active Phase 3 long term extension study.
Across the reported studies, total enrollment is about 110 participants. The Phase 3 long term extension study NCT04042402 enrolled 75 patients, the completed Phase 2 study NCT03847909 enrolled 35, the recruiting Phase 2 study NCT04580420 plans 28, and the Phase 1 study NCT04555486 enrolled 6.
DCR-PHXC is being evaluated across primary hyperoxaluria type 1, type 2, and type 3. The Phase 3 extension study NCT04042402 includes patients with type 1, type 2, and type 3, while NCT04580420 focuses on type 1 patients who also have end stage renal disease and NCT04555486 studied type 3.