A Phase 1 Study of DCR-PH1 in Patients with PH 1
Research type
Research Study
Full title
A Phase 1 Study of DCR-PH1 in Patients with Primary Hyperoxaluria Type 1 (PH1)
IRAS ID
189854
Contact name
Sally-Anne Hulton
Contact email
Sponsor organisation
Dicerna Pharmaceuticals, Inc.
Eudract number
2015-003142-51
Duration of Study in the UK
1 years, 10 months, 1 days
Research summary
This purpose of this research study is to determine a safe and tolerable dose of DCR-PH1, an experimental new drug to treat 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, heart, eyes and other organs. Currently there are no approved treatments for PH1.
The study is sponsored by Dicerna Pharmaceuticals, Inc.
Subjects with PH1 participating in Study DCR-PH1-101 will be the first humans to receive DCR PH1. The study is designed in two parts; - in Part A of the study subjects will be given a single dose of DCR-PH1 by infusion and then have a series of tests over a 4 week observational period to see if DCR-PH1 has side effects and also if DCR-PH1 is changing oxalate and other metabolites in the blood and urine. Subjects will attend the clinic 8 times for planned visits.
Once the Part A maximum tolerated dose has been established, all available clinical and nonclinical data will be summarized and submitted to regulatory authorities and ethics committees, to support dosing in Part B of the study. Part B of the study will provide additional safety and tolerability information as well as better characterization of the pharmacokinetic (PK) and PD effects of multiple DCR PH1 doses.
In part A of the study, about 20 PH1 subjects from around the world will be included over a period of approximately a year.
Part B of the study will consist of a screening period, a dosing period during which subjects will receive a dose of DCR-PH1 once every 4 weeks, and a 4-week safety observational period following the last dose of DCR-PH1 administration.
The entire study including part B is expected to complete in 2017.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
16/WM/0019
Date of REC Opinion
11 Apr 2016
REC opinion
Further Information Favourable Opinion