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

Completed · Phase 3

Conditions studied: Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)

In brief

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).

Key facts

Study ID

NCT03905694

Run by

Alnylam Pharmaceuticals

People needed

18

Starts

2019-04-22

Expected to finish

2024-07-26

Last updated by the study team

2025-02-14

Who can join

Age: any, up to 5. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Clinical Trial Site — Rochester, Minnesota, United States
• Clinical Trial Site — Houston, Texas, United States
• Clinical Trial Site — Lyon, France
• Clinical Trial Site — Paris, France
• Clinical Trial Site — Bonn, Germany
• Clinical Trial Site — Haifa, Israel
• Clinical Trial Site — Jerusalem, Israel
• Clinical Trial Site — Nahariya, Israel
• Clinical Trial Site — London, United Kingdom

Full record on ClinicalTrials.gov

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