Phase I study to compare uridine PK in healthy volunteers (QCL117618)
Research type
Research Study
Full title
A Phase-1, Single-Part, Six Period, Sequential, Open-Label Study to Compare Uridine Pharmacokinetics of Prototype Formulations of Uridine Triacetate with Reference to Uridine Triacetate Oral Granules in Healthy Male and Female Subjects
IRAS ID
212363
Contact name
Joan Helton
Contact email
Sponsor organisation
Wellstat Therapeutics Corporation
Eudract number
2016-002967-33
Duration of Study in the UK
0 years, 2 months, 8 days
Research summary
The Sponsor is developing a prototype formulation of the study drug uridine triacetate, for the potential treatment of mitochondrial disease. Mitochondria are present within every cell in the body and are responsible for producing energy to support cell function. In mitochondrial disease, mitochondria do not produce enough energy causing cells to die. When multiple cells in the same organ die, this results in organ failure. \n\nThe purpose of this study is to look at how two new forms of uridine triacetate are taken up by the body compared to reference uridine triacetate oral granules, an already available form of the study drug. \n\nThe study will consist of up to 6 sequential study periods involving up to 14 healthy male and female subjects. In regimen A, subjects will receive the oral granule reference product pre-mixed with chocolate pudding. In regimen B, subjects will receive the study drug, uridine triacetate, in particle form mixed with chocolate pudding. In regimen C subjects will receive uridine triacetate as a liquid (prototype 1) mixed with syrup. In regimen D subjects will receive uridine triacetate liquid (prototype 1) mixed with chocolate pudding. Regimens E and F are optional and their implementation will be determined after regimens D and E. The treatment to be given in these optional cohorts will be uridine triacetate oral liquid prototype 2, prototype 3 or particles either mixed with a syrup or with chocolate pudding.\n\nSubjects will remain in the clinical unit from the evening prior to regimen A until 24 hours post-dose of regimen D, which is a total of 8 days. For each of the optional two cohorts (E & F) subjects will be re-admitted for 24 hours. Blood samples will be taken at regular intervals after every dose of the study drug for pharmacokinetic analysis.\n
REC name
Wales REC 2
REC reference
16/WA/0234
Date of REC Opinion
7 Sep 2016
REC opinion
Further Information Favourable Opinion