Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children.
Genet Med
; 24(3): 654-662, 2022 03.
Article
en En
| MEDLINE
| ID: mdl-34906487
ABSTRACT
PURPOSE:
Primary hyperoxaluria type 1 (PH1) is a rare, progressive, genetic disease with limited treatment options. We report the efficacy and safety of lumasiran, an RNA interference therapeutic, in infants and young children with PH1.METHODS:
This single-arm, open-label, phase 3 study evaluated lumasiran in patients aged <6 years with PH1 and an estimated glomerular filtration rate >45 mL/min/1.73 m2, if aged ≥12 months, or normal serum creatinine, if aged <12 months. The primary end point was percent change in spot urinary oxalate to creatinine ratio (UOxCr) from baseline to month 6. Secondary end points included proportion of patients with urinary oxalate ≤1.5× upper limit of normal and change in plasma oxalate.RESULTS:
All patients (N = 18) completed the 6-month primary analysis period. Median age at consent was 50.1 months. Least-squares mean percent reduction in spot UOxCr was 72.0%. At month 6, 50% of patients (9/18) achieved spot UOxCr ≤1.5× upper limit of normal. Least-squares mean percent reduction in plasma oxalate was 31.7%. The most common treatment-related adverse events were transient, mild, injection-site reactions.CONCLUSION:
Lumasiran showed rapid, sustained reduction in spot UOxCr and plasma oxalate and acceptable safety in patients aged <6 years with PH1, establishing RNA interference therapies as safe, effective treatment options for infants and young children.Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Hiperoxaluria Primaria
/
Tratamiento con ARN de Interferencia
Límite:
Child, preschool
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Humans
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Infant
Idioma:
En
Año:
2022
Tipo del documento:
Article