ILLUMINATE B - Evaluate Lumasiran in young children with PH1
Research type
Research Study
Full title
ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1
IRAS ID
257635
Contact name
Wesley Hayes
Contact email
Sponsor organisation
Alnylam Pharmaceticals, Inc
Eudract number
2018-004014-17
Duration of Study in the UK
5 years, 6 months, 1 days
Research summary
Research Summary
PH1 is a rare genetic disease which effects predominantly the liver and ultimately the kidneys. It is characterised by the excessive 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 8 patients from 37 weeks gestational age but less than 6 years old are expected to be enrolled across 9 clinical study centres in 5 countries. The study consists of 3 parts:
- Screening Period (up to 2 Months)
- Primary Analysis Period (6 Months)
- Long-Term Extension Period (Approximately 4 years)All eligible participants will be administered the investigational medicine, lumasiran, no control group will be assessed. The participants will not be assigned treatment groups, the dosing regimen will be determined by the participant’s weight. At month 3 and beyond, participants will receive lumasiran either monthly(participants weighing <10 kg) or every 3 months (participants weighing ≥10 kg) at the maintenance dose. Patients with weight increases crossing the threshold for the next weight-based dosing category (<10 kg to ≥10 kg or <20 kg to ≥20 kg) will follow the new dosing regimen for the remainder of the study or until the next dosing category threshold is reached.
The purpose of the study is to determine if lumasiran is able to reduce the production of oxalate in the liver, and evaluate the safety of lumasiran.
Summary of Results
The plain language summary is available for download on our clinical trial. https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fclick.pstmrk.it%2F3ts%2Fclinicaltrials.alnylam.com%252F%2FNBTI%2F-w68AQ%2FAQ%2F12e4be3d-461b-483f-94f9-b8331bf80d52%2F2%2F_SoNTmDCaS&data=05%7C02%7Cedgbaston.rec%40hra.nhs.uk%7C065419ad2dc74da8495908dd655f2799%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638778181535363026%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=88RQJb69kxzwNO8vnjFECVRiVtXa0EJD2m9SvjQDTt0%3D&reserved=0
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
19/WM/0014
Date of REC Opinion
1 Mar 2019
REC opinion
Further Information Favourable Opinion