DCR-PH1-501 An Obs Study of Patients with Primary Hyperoxaluria Type 1
Research type
Research Study
Full title
PHYOS: An Observational Study of Patients with Primary Hyperoxaluria Type 1
IRAS ID
183383
Contact name
Sally-Anne Hulton
Contact email
Sponsor organisation
Dicerna Pharmaceuticals, Inc
Duration of Study in the UK
1 years, 0 months, 29 days
Research summary
The purpose of this observational study is to gather information about Primary Hyperoxaluria type 1 (PH1). PH1 is a rare, inherited disorder of the liver in which excess oxalate production often results in severe damage to the kidneys, skin, bone, blood and eyes. The clinical course of PH1 is highly variable and biochemical markers for the disease and its progression are not well established and the assays used have not been standardised.
Currently there are no treatments for PH1.
This study is sponsored by Dicerna Pharmaceuticals, Inc.
In this study no experimental drug or procedures will be administered- thus the risk to subjects is minimal.Approximately 50 subjects with PH1 will be included in this study worldwide. The duration of participation for individual subjects is expected to be approximately 6 months.
Enrolled subjects will be seen in clinic at screening (up to 2 months before starting), day 1, month 4 and month 6. Study procedures include physical examinations, blood tests (including pregnancy tests if applicable) and at months 2, 4 and 6 subjects will be asked to provide 24 hour urine collections. Subjects will also be asked to record their fluid intake and Vitamin B6 use in a dairy and will be given quality of life questionnaires to complete.
Initially the study will enrol subjects of 12 years and older (Cohort A) as subjects of this age group will be considered for the phase 1 study of DCR-PH1. Enrollment to Cohort B (age 6-11.9 years) is expected to be initiated in mid-2016 when it has been determined that patients of this age group may be enrolled in clinical trials of DCR-PH1.
Patients participating in this observational study may be offered an opportunity to participate in upcoming clinical trials of the trial medication if they fulfil the specific study criteria.
REC name
Wales REC 4
REC reference
15/WA/0318
Date of REC Opinion
15 Oct 2015
REC opinion
Further Information Favourable Opinion