A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (ILLUMINATE-A)
Primary Hyperoxaluria Type 1 (PH1)
About this trial
This is an interventional treatment trial for Primary Hyperoxaluria Type 1 (PH1) focused on measuring PH1, Primary hyperoxaluria, RNAi therapeutic, siRNA, AGT, Oxalate
Eligibility Criteria
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
Sites / Locations
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Arms of the Study
Arm 1
Arm 2
Placebo Comparator
Experimental
Placebo
Lumasiran
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.
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.