ALN-GO1 in Healthy Adult Subjects and Patients with PH1
Research type
Research Study
Full title
A Phase 1/2, Single-Blind, Placebo-Controlled, Single- and Multiple-Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1
IRAS ID
197517
Contact name
Sally-Anne Hulton
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2015-004407-23
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 11 months, 28 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.\nThere are currently no approved therapies for the treatment of PH1. Disease management involves high fluid intake and vitamin B6. Patients progressing to kidney failure require intense kidney dialysis however this is intrusive increasing 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.\nAlnylam 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.\nThis study will be conducted in two parts: Part A with healthy volunteers and Part B with patients with PH1. The study is being done to evaluate how well different doses of ALN-GO1 are tolerated in healthy volunteers and in PH1 patients, and their safety.\nPart A will be carried out at one study centre in the United Kingdom and will enroll up to 40 healthy adults (18-64 years old). Part B will take place across Europe, United States and Israel and will enroll up to 20 patients with PH1 (6-64 years old). \nThis is a randomised, single-blind, placebo-controlled study meaning that participants will be randomly assigned to receive either ALN-GO1 or placebo (looks like the study drug but with no active ingredient). Participants will be randomly assigned to different dose levels of the study drug. Participants in Part A will receive study drug once. Participants in Part B will receive three doses of study drug 28 days apart. The study doctor will know which study drug/dose level is being given however the patient will not.
REC name
North East - York Research Ethics Committee
REC reference
16/NE/0010
Date of REC Opinion
10 Feb 2016
REC opinion
Further Information Favourable Opinion