PHYOX8
Research type
Research Study
Full title
A Phase 2 Open-Label Multicenter Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Nedosiran in Pediatric Patients from Birth to 11 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function.
IRAS ID
299680
Contact name
Faidra Veligratli
Contact email
Sponsor organisation
Dicerna Pharmaceuticals, Inc
Eudract number
2021-001083-16
Clinicaltrials.gov Identifier
n/a, n/a
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Primary hyperoxularia (PH) 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. There are 3 types of PH (PH1, PH2, and PH3) and each is attributed to a specific gene mutation. Symptoms of this disease start early in life from early infancy in some patients.
Patients with PH are predisposed to the development of multiple and recurrent urinary tract and kidney stones. At present, one treatment for PH1 is approved in the US and Europe. There is an unmet need for treatment in all subtypes of PH globally particularly for PH2 and PH3 as no therapies are presently approved. Current supportive therapies used to mitigate effects of the disease are burdensome to both the patient and their caregivers and are not highly effective at preventing disease progression.
Nedosiran is a drug which is intended to reduce and prevent the production of oxalate in the liver in all genetic forms of PH (PH1, PH2, and PH3).
The purpose of this study is to test the safety and efficacy of nedosiran in male and female children from birth to 5 years of age. This study will take place in clinical sites in America, Australia, Asia and Europe and will include 15 patients.
The study will consist of 3 period:
Screening: This will last up to 35 days and will check patients eligibility to participate.
Treatment: Patients will need to visit the study clinic once monthly for 6 months and will receive an injection of nedosiran under the skin.
Follow up visit: One further visit at the study site approximately 30 days following completion of study treatment.
Patients who complete this study may be eligible for long term treatment with nedosiran in a further follow up study.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
21/SC/0222
Date of REC Opinion
23 Aug 2021
REC opinion
Further Information Favourable Opinion