A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

Study Overview
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Brief Summary
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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).

Official Title
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ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

Conditions
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Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)

Intervention / Treatment
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* Drug: Lumasiran


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Lumasiran<br>Lumasiran will be administered by subcutaneous (SC) injection. | Drug: Lumasiran<br>* Lumasiran will be administered by subcutaneous (SC) injection.<br>* Other names: ALN-GO1;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. | Baseline to Month 6 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage Change in Urinary Oxalate Excretion From Baseline to End of Study (Month 60) |  | Up to 60 months | 
| Absolute Change in Urinary Oxalate Excretion From Baseline |  | Up to 60 months | 
| Percentage of Time That Spot Urinary Oxalate:Creatinine Ratio ≤ Near-normalization Threshold (≤1.5 × ULN) |  | Up to 60 months | 
| Percentage of Participants With Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN |  | Up to 60 months | 
| Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60) |  | Up to 60 months | 
| Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60) |  | Up to 60 months | 
| Maximum Observed Plasma Concentration (Cmax) of Lumasiran |  | Up to 24 months | 
| Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran |  | Up to 24 months | 
| Elimination Half-life (t1/2beta) of Lumasiran |  | Up to 24 months | 
| Area Under the Concentration-time Curve (AUC) of Lumasiran |  | Up to 24 months | 
| Apparent Clearance (CL/F) of Lumasiran |  | Up to 24 months | 
| Apparent Volume of Distribution (V/F) of Lumasiran |  | Up to 24 months | 
| Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline |  | Up to 60 months | 
| Frequency of Adverse Events (AEs) |  | Up to 60 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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siRNA, AGT, PH1, Primary Hyperoxaluria, Hyperoxaluria, RNAi Therapeutic