PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1/2 and ESRD
Research type
Research Study
Full title
A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients with Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, with or without Dialysis
IRAS ID
287608
Contact name
Kerry Russell
Contact email
Sponsor organisation
Dicerna Pharmaceuticals Inc
Eudract number
2020-002826-97
Clinicaltrials.gov Identifier
n/a, n/a
Duration of Study in the UK
4 years, 3 months, 1 days
Research summary
Primary Hyperoxaluria Types 1 (PH1) and 2 (PH2) are rare inherited conditions where lack of function of a particular liver enzyme that causes the body to build up excess amounts of a substance called oxalate, leading to recurrent kidney and bladder stones. How the kidneys work can be impacted by these stones.
Over time kidney function may be reduced by frequent stones and/or buildup of calcium resulting in chronic kidney disease and possibly end stage kidney disease (ESRD), which is a life-threatening condition that prevents the kidneys from filtering fluids and waste products from the body. People with PH1 and PH2 who develop ESRD require haemodialysis (the use of a machine and filter to remove waste products and water from the blood).
Currently there is no approved therapy for the treatment of PH patients. Supportive therapies, such as drinking plenty of water and restricting foods high in oxalate, are used to help lower oxalate levels and decrease formation of kidney stones. If the kidney is damaged, a kidney transplant may be needed for PH1 or PH2. People with PH1 and PH2 may need more than one kidney transplant during their lifetime as oxalate can build up again after transplant. A liver transplant may be the only option for a cure in patients with PH1.
In this study, the drug DCR-PHXC is being assessed to determine the safety of this medication and how effective it is in reducing excess levels of kidney-damaging oxalate in patients with PH1 or PH2 and severe renal impairment. DCR-PHXC reduces oxalate by reducing the enzyme in the final step of the oxalate producing process. DCR-PHXC will be taken as a monthly injection during this study.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
21/SC/0011
Date of REC Opinion
24 Feb 2021
REC opinion
Further Information Favourable Opinion