ILLUMINATE C - Evaluate Lumasiran in Patients with Advanced PH1
Research type
Research Study
Full title
ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1)
IRAS ID
266892
Contact name
Shabbir Moochhala
Contact email
Sponsor organisation
Alnylam Pharmaceticals, Inc
Eudract number
2019-001346-17
Duration of Study in the UK
5 years, 6 months, 22 days
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 ‘Lumasiran’ (ALN-GO1). It is thought that lumasiran may reduce the production of harmful oxalate and symptoms of PH1.
This is a global phase 3 study, approximately 16 patients from 37 weeks gestational age up to an undefined age limit are expected to be enrolled across 15 clinical study centres in 10 countries. All eligible patients will be administered open-label lumasiran; no control group will be assessed.
The study consists of 3 parts:
- Screening Period (2 months)
- Primary Analysis Period (6 months)
- Long-term extension period (54 months)The study will include 2 cohorts: Cohort A and Cohort B. Cohort A will include patients who do not yet require dialysis. Cohort B will include patients who are on dialysis therapy. Dialysis therapy will be restricted to patients on haemodialysis only. Cohort A patients who experience a progression of renal impairment over time and begin to require dialysis therapy will cross-over to Cohort B.
From Screening to Day 1, patients will have 4 plasma oxalate collections to establish a robust baseline measurement for plasma oxalate.
The study medicine Lumasiran will be administered subcutaneously using weight-based dosing.
The purpose of the study is to evaluate the effect of lumasiran on plasma oxalate in patients who are not on dialysis therapy and in patients who are on dialysis therapy.
REC name
London - London Bridge Research Ethics Committee
REC reference
19/LO/1903
Date of REC Opinion
22 Jan 2020
REC opinion
Further Information Favourable Opinion