A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
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).
Inclusion Criteria:

 -  Has 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:

 -  Abnormal serum creatinine levels at screening for infants who are less than 1 year old

 -  Does not have relatively preserved kidney function

 -  Clinical evidence of systemic oxalosis

 -  History of kidney or liver transplant