Overview

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

Status:
Active, not recruiting
Trial end date:
2024-08-19
Target enrollment:
0
Participant gender:
All
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).
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Alnylam Pharmaceuticals
Criteria
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