A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (ILLUMINATE-C)

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Primary Hyperoxaluria; Primary Hyperoxaluria Type 1

Treatments: Drug: Lumasiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT04152200

2019-001346-17 (EudraCT Number)

2023-503382-29-00

ALN-GO1-005

Details and patient eligibility

About

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).

Enrollment: 21 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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21 participants in 1 patient group

Lumasiran

Experimental group

Description:

All patients will receive open-label lumasiran.

Treatment:

Drug: Lumasiran