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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03681184 | A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 | PHASE3 | COMPLETED | 39 | — | — | Nov 27, 2018 | Jan 12, 2024 | Aug 12, 2024 | 17 | United States, France +6 |
| NCT02706886 | Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1 | PHASE1 | COMPLETED | 52 | — | — | Mar 8, 2016 | Jan 23, 2019 | Jan 30, 2020 | 9 | France, Germany +3 |
Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline across Months 3 through 6. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.
An AE is any untoward medical occurrence in a clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
| Arm | Type | Description |
|---|---|---|
| Placebo/Lumasiran | PLACEBO_COMPARATOR | Lumasiran-matching placebo (normal saline \[0.9% NaCl\]) was administered subcutaneously (SC) at Day 1 and Months 1, 2 and 3 during the 6-Month Double-blind (DB) Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month Open-label Extension (OLE) period. |
| Lumasiran/Lumasiran | EXPERIMENTAL | Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period. |
| Part A: SAD: Placebo | PLACEBO_COMPARATOR | A single dose of matching placebo will be administered subcutaneously (SC). |
| Part A: SAD: Lumasiran 0.3 mg/kg | EXPERIMENTAL | A single dose of 0.3 mg/kg lumasiran will be administered SC. |
| Part A: SAD: Lumasiran 1.0 mg/kg | EXPERIMENTAL | A single dose of 1.0 mg/kg lumasiran will be administered SC. |
| Part A: SAD: Lumasiran 3.0 mg/kg | EXPERIMENTAL | A single dose of 3.0 mg/kg lumasiran will be administered SC. |
| Part A: SAD: Lumasiran 6.0 mg/kg | EXPERIMENTAL | A single dose of 6.0 mg/kg lumasiran will be administered SC. |
| Part B: MAD: Placebo | PLACEBO_COMPARATOR | Participants with primary hyperoxaluria type 1 (PH1) will be treated with placebo matching one of the lumasiran dosages in the lumasiran arms (one placebo participant for each lumasiran arm). At Day 85 these placebo treated participants will cross over to their respective Part B lumasiran arms in the Part B: MAD Study Day 85-End of Study Period and will then be treated with lumasiran. The estimated total time on study was up to 546 days. |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | EXPERIMENTAL | Participants with PH1 will be treated with 1.0 mg/kg lumasiran SC once monthly (qM) on Days 1, 29 and 57. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | EXPERIMENTAL | Participants with PH1 will be treated with 3.0 mg/kg lumasiran SC qM on Days 1, 29 and 57. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | EXPERIMENTAL | Participants with PH1 will be treated with 3.0 mg/kg lumasiran SC once every three months (q3M) on Days 1 and 85. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. |
| Name | Type | Description |
|---|---|---|
| Placebo | DRUG | Placebo by SC injection |
| Lumasiran | DRUG | Lumasiran by SC injection |
Inclusion Criteria: * Willing to provide written informed consent or assent and to comply with study requirements * Confirmation of PH1 disease * Meet the 24 hour urine oxalate excretion requirements * If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days Exclusi...