Natural History of Patients with PH3 and History of Stone Events
Research type
Research Study
Full title
A Natural History Study of Patients with Genetically Confirmed Primary Hyperoxaluria Type 3 with a History of Stone Events
IRAS ID
289642
Contact name
Kerry Russell
Contact email
Sponsor organisation
Dicerna Pharmaceuticals Inc
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
n/a, n/a
Duration of Study in the UK
2 years, 8 months, 2 days
Research summary
Participants in this study have Primary Hyperoxaluria Type 3 (PH3). This is an inherited disease where lack of function of a particular liver enzyme causes the body to accumulate excess amounts of a substance called oxalate. Oxalate leads to a build-up of insoluble calcium salts in the kidneys. People with PH3 are at increased risk for developing kidney stones. Symptoms can develop at any time from infancy to adulthood. Most individuals are diagnosed with PH3 in childhood or early adulthood. Affected individuals may develop kidney stones at any age, and sometimes by the age of 5 years. Kidney function can be impacted by frequent kidney stones. Over time kidney function may become compromised from frequent stones and/or too much calcium deposited in the kidneys, resulting in chronic kidney disease.
Currently there is no approved therapy for the treatment of patients with PH3. Supportive therapies, such as drinking plenty of water and dietary restrictions of foods high in oxalate, are used to help lower oxalate levels and inhibit the formation of kidney stones.No study drug or other experimental treatment/procedures will be administered. This is an observational study requiring urine collection and kidney ultrasound in order to collect data on the rate of new stone formation and the degree of excess calcium in the kidneys to explore the potential relationship between levels of oxalate in the kidneys and new stone formation. This will be beneficial for increasing understanding of the course of PH3 and potentially help to create new therapies for this disease.
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
20/SW/0191
Date of REC Opinion
18 Feb 2021
REC opinion
Further Information Favourable Opinion