Phase 3
N=18
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Primary Hyperoxaluria · Primary Hyperoxaluria Type 1 (PH1)
Bottom Line
View on ClinicalTrials.gov: NCT03905694 ↗Enrolled (actual)
18
Serious AEs
11.1%
Results posted
Jul 2021
Primary outcome: Primary: Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 — -71.97 percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Lumasiran (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Alnylam Pharmaceuticals
- Primary completion
- Jun 2020
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 | — |
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).
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). 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.