A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (ILLUMINATE-A)

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Primary Hyperoxaluria Type 1 (PH1)

Treatments: Drug: Lumasiran; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03681184

ALN-GO1-003

2018-001981-40 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).

Enrollment: 39 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Willing to provide written informed consent or assent and to comply with study requirements
• Confirmation of PH1 disease
• Meet the 24 hour urine oxalate excretion requirements
• If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

Exclusion criteria

• Clinically significant health concerns (with the exception of PH1) or clinical evidence of extrarenal systemic oxalosis
• Clinically significant abnormal laboratory results
• Known active or evidence of HIV or hepatitis B or C infection
• An estimated GFR of < 30 mL/min/1.73m^2 at screening
• Received an investigational agent within 30 days or 5 half-lives before the first dose of study drug or are in follow-up of another clinical study
• History of kidney or liver transplant
• Known history of multiple drug allergies or allergic reaction to an oligonucleotide or GalNAc
• History of intolerance to subcutaneous injection
• Women who are pregnant, planning a pregnancy, or breast-feeding or those of child bearing potential and not willing to use contraception
• History of alcohol abuse within the last 12 months, or unable or unwilling to limit alcohol consumption throughout the study

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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39 participants in 2 patient groups, including a placebo group

Placebo/Lumasiran

Placebo Comparator group

Description:

Lumasiran-matching placebo (normal saline \[0.9% NaCl\]) was administered subcutaneously (SC) at Day 1 and Months 1, 2 and 3 during the 6-Month Double-blind (DB) Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month Open-label Extension (OLE) period.

Treatment:

Drug: Placebo

Drug: Lumasiran

Lumasiran/Lumasiran

Experimental group

Description:

Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Treatment:

Drug: Placebo

Drug: Lumasiran