ALN-G01-002 Extension study of long-term ALN-G01 in Patients with PH1
Research type
Research Study
Full title
A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1
IRAS ID
227923
Contact name
Sally-Anne Hulton
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2016-003134-24
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Primary Hyperoxaluria Type 1 (PH1) is a rare genetic disease characterised by excessive production of oxalate. Oxalate crystallises in the urinary tract resulting in recurrent kidney and bladder stones, which cause urinary obstruction, infection, and progressive kidney failure.
There are currently no approved therapies for the treatment of PH1, other than liver transplantation. Disease management involves high fluid intake and vitamin B6. Patients progressing to kidney failure require frequent dialysis however this intensive dialysis up to 6 sessions per week is associated with complications. Early kidney/liver transplant is the only cure however; this is limited by organ availability. There is a high-unmet need for treatment for PH1.
Alnylam Pharmaceuticals, Inc. have developed a new drug, ALN-GO1 that has been designed to suppress an enzyme in the body called glycolate oxidase to reduce the production of oxalate in the liver.
This study will be conducted to evaluate the long-term administration of ALN-G01 in participants with PH1.
The study, which will allow approximately 2 years of treatment, will be carried out at two study centres in the United Kingdom and 20 patients with PH1 will be enrolled globally.
This is an open label, unblinded study meaning that participants will receive sub-cutaneous injections of ALN-GO1 at the same dose and regimen as they received in the study ALN-GO1-001. The study doctor and the participant will know the study medication/dose level, which is being administered.REC name
North East - York Research Ethics Committee
REC reference
17/NE/0321
Date of REC Opinion
20 Dec 2017
REC opinion
Further Information Favourable Opinion