A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

• Status: Active, not recruiting
• Trial end date: 2025-07-01
• 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 patients with Advanced Primary Hyperoxaluria Type 1 (PH1).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria:

• Has documented diagnosis of primary hyperoxaluria type 1 (PH1)

• Estimated glomerular filtration rate (eGFR) ≤45 mL/min/1.73 m^2 for patients ≥12 months of age (<12 months of age, must have serum creatinine considered elevated for age)

• Meets plasma oxalate level requirements

• If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

• If on dialysis, may be on hemodialysis therapy only and must have been on a stable regimen for at least 4 weeks

Exclusion Criteria:

• Hemodialysis/peritoneal dialysis combination therapy or peritoneal dialysis alone

• Diagnosis of conditions other than PH1 contributing to renal insufficiency

• History of liver transplant

• History of kidney transplant and currently receiving immunosuppressants