ILLUMINATE A - Phase III Efficacy and Safety of Lumasiran in PH1
Research type
Research Study
Full title
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
IRAS ID
251137
Contact name
Sally-Anne Hulton
Contact email
Sponsor organisation
Alnylam Pharmaceticals, Inc
Eudract number
2018-001981-40
Duration of Study in the UK
5 years, 1 months, 0 days
Research summary
PH1 is a rare genetic disease which effects predominantly the liver and ultimately the kidneys. It is characterised by the above than normal production by the liver of a small molecule called oxalate, and is caused by a mutation in a gene. The oxalate mixes with small molecule called Calcium, which crystallise in the urinary tract, causing the formation of painful kidney stones, infections and ultimately kidney disease and failure.
Alnylam Pharmaceutical, Inc., is developing an injection called ‘Lumarsiran’ (ALN-GO1). It is thought that lumasiran may reduce the production of harmful oxalate and symptoms of PH1.
This is a Global Phase III study, approximately 30 patients from 6 years old are expected to be enrolled across 20 research sites globally. The study consists of parts:
- Screening Period (2 Months)
- Double-Blind Treatment Period (6 Months)
- Blinded Treatment Extension Period (3 Months)
- Open-Label Extension (4 Years)
If participants are eligible for the main study, they will enter the double-blind period, and have 66% chance of initially receiving Lumasiran, and 33% chance of initially receiving a placebo. A placebo looks like a lumasiran, however there is no active ingredient. In the blinded treatment period, participants who received placebo during the previous period will receive lumasiran in months 7 and 8. Participants who received lumasiran in the previous period will receive placebo for months 7 and 8. All participants will receive lumarisran for month 9. All participants who complete the double-blind treatment period and enter the open-label extension phase after month 9 of the study will receive lumasiran every 3 months for approximately 4 years (end of the study).
The purpose of the study is to determine of lumasiran is able to reduce the production of oxalate in the liver, and evaluate the safety of lumasiran.REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
18/NE/0294
Date of REC Opinion
20 Nov 2018
REC opinion
Further Information Favourable Opinion