A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 (ILLUMINATE-B)

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Primary Hyperoxaluria Type 1 (PH1); Primary Hyperoxaluria

Treatments: Drug: Lumasiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT03905694

ALN-GO1-004

2018-004014-17 (EudraCT Number)

Details and patient eligibility

About

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).

Enrollment: 18 patients

Sex: All

Ages: Under 5 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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18 participants in 1 patient group

Lumasiran

Experimental group

Description:

Lumasiran will be administered by subcutaneous (SC) injection.

Treatment:

Drug: Lumasiran