Phase 3
Completed N=18
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Primary Hyperoxaluria · Primary Hyperoxaluria Type 1 (PH1)
Source: ClinicalTrials.gov NCT03905694 ↗
Enrolled (actual)
18
Serious AEs
11.1%
Results posted
Jul 2021
Primary outcomePrimary: Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 — -71.97 percent change
◆ Published Evidence
Established
74citations · ~19 / year
Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children.
Summary
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
Linked Publications (4)
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Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children.
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Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial.
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Next-generation therapeutics for rare genetic disorders.
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Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 |
-71.97 | — |
| SECONDARY Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60]) |
-74.48 | — |
| SECONDARY Percentage of Time That Spot Urinary Oxalate: Creatinine Ratio is at or Below the Near-normalization Threshold (≤1.5 × Upper Limit of Normal (ULN) for Age) |
68.95 | — |
| SECONDARY Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline |
-0.4880; -0.5179 | — |
| SECONDARY Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age |
100 | — |
| SECONDARY Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age |
100 | — |
| SECONDARY Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60) |
-32.06; -24.78 | — |
| SECONDARY Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60) |
-5.03; -5.03 | — |
| SECONDARY Maximum Observed Plasma Concentration (Cmax) of Lumasiran |
886; 869; 1180; 878; 790 | — |
| SECONDARY Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran |
3.74; 4; 4; 3.64; 3.73 | — |
| SECONDARY Elimination Half-life (t1/2beta) of Lumasiran |
5.46; 5.96; 3.52; 3.74; 4.99 | — |
| SECONDARY Area Under the Concentration-time Curve From 0 to 24 Hours (AUC0-24) of Lumasiran |
8050; 8450; 10800; 8420; 7420 | — |
| SECONDARY Area Under the Concentration-time Curve From 0 to Last Quantifiable Concentration (AUC0-last) of Lumasiran |
7870; 6620; 8480; 5770; 7100 | — |
| SECONDARY Area Under the Concentration-time Curve From 0 to Infinity (AUC0-infinity) of Lumasiran |
9180; 8840; 11900; 11600; 9610 | — |
| SECONDARY Apparent Clearance (CL/F) of Lumasiran |
8.62; 10.2; 7.06; 6.87; 8.22 | — |
| SECONDARY Apparent Volume of Distribution (V/F) of Lumasiran |
57.2; 81; 36; 43.1; 57.6 | — |
| SECONDARY Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) |
-0.263; -4.525 | — |
| SECONDARY Number of Participants With Adverse Events (AEs) |
18 | — |
Eligibility Criteria
Inclusion Criteria
- Has genetic confirmation of primary hyperoxaluria type 1 (PH1)
- Meets urinary oxalate excretion requirements
- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
Exclusion Criteria
- If <12 months old at screening, has an abnormally high serum creatinine
- If ≥12 months old at screening, has an estimated glomerular filtration rate (GFR) of ≤45 mL/min/1.73m^2
- Clinical evidence of systemic oxalosis
- History of kidney or liver transplant
Data sourced from ClinicalTrials.gov (NCT03905694) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.