CompletedPhase 3

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

United States, France, Germany18 participantsStarted 2019-04-22

Plain-language 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).

Who can participate

Age range0 Years – 5 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

Timeframe: Baseline to Month 6

Trial details

NCT IDNCT03905694

SponsorAlnylam Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-06-29

Results submitted2021-06-29

View on ClinicalTrials.gov More Primary Hyperoxaluria trials