Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04042402 | Long Term Extension Study in Patients With Primary Hyperoxaluria | PHASE3 | ACTIVE NOT_RECRUITING | 75 | — | — | Jul 9, 2019 | Apr 1, 2030 | May 30, 2025 | 23 | United States, Australia +11 |
| NCT03847909 | A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 | PHASE2 | COMPLETED | 35 | — | — | Oct 28, 2019 | Jun 29, 2021 | May 22, 2024 | 28 | United States, Australia +13 |
To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1
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.
| Arm | Type | Description |
|---|---|---|
| Open Label | EXPERIMENTAL | Open label, monthly subcutaneous injection |
| 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 |
| 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 |
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...