ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
Who is this study for? Children and adults with primary hyperoxaluria type 1
What treatments are being studied? Lumasiran
Status: Completed
Location: See all (17) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• 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
Locations
United States
California
Clinical Trial Site
San Diego
Florida
Clinical Trial Site
Jacksonville
Minnesota
Clinical Trial Site
Rochester
New York
Clinical Trial Site
New York
Ohio
Clinical Trial Site
Cleveland
Other Locations
France
Clinical Trial Site
Lyon
Clinical Trial Site
Paris
Germany
Clinical Trial Site
Bonn
Israel
Clinical Trial Site
Haifa
Clinical Trial Site
Jerusalem
Clinical Trial Site
Nahariya
Netherlands
Clinical Trial Site
Amsterdam
Switzerland
Clinical Trial Site
Bern
United Arab Emirates
Clinical Trial Site
Dubai
United Kingdom
Clinical Trial Site
Birmingham
Clinical Trial Site
London
Clinical Trial Site
London
Time Frame
Start Date: 2018-11-27
Completion Date: 2024-01-12
Participants
Target number of participants: 39
Treatments
Placebo_comparator: 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.
Experimental: Lumasiran/Lumasiran
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.
Related Therapeutic Areas
Sponsors
Leads: Alnylam Pharmaceuticals