TIRCON
Research type
Research Study
Full title
A randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN)
IRAS ID
102715
Contact name
Patrick Chinnery
Contact email
Sponsor organisation
ApoPharma Inc
Eudract number
2012-000845-11
ISRCTN Number
n/a
Research summary
Neurodegeneration with Brain Iron Accumulation (NBIA) are a group of 7 diseases which cause increased levels of iron to become stored within the brain. The most common of these 7 diseases is called Pantothenate Kinase Associated Neurodegeneration or ‘PKAN’. The excess iron in PKAN, like the other NBIA diseases, interferes with the normal functioning of parts of the brain, in particular affecting learning, communication and movement of the arms and legs. PKAN is very progressive, and many patients sadly die in childhood or early adulthood. At present we have no proven treatment.
Many doctors and scientists believe that the best way to treat the condition will be to try and prevent the build up of iron within the brain. For years there have been drugs available which are able to ‘bind’ or stick to iron in the blood and prevent it from entering cells. However,they have not been able to get out of the blood and into the brain and therefore not been effective for problems with brain iron. Recently, the drug ‘deferiprone’ has however been developed and is already used safely in humans to treat other conditions such as the blood disorder thalassaemia. Crucially, it is much better at getting into the brain, and recently a very small number of patients with PKAN showed some improvement in a small study of the drug.
Therefore, the aim of this study is to determine whether deferiprone, over an 18 month period, is able to improve the symptoms in patients with PKAN and also reduce the levels of iron in the brain seen with MRI scans. It is hoped that this would provide the first real treatment for the condition, and may also help people with the other types of NBIA.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
13/YH/0171
Date of REC Opinion
17 Jul 2013
REC opinion
Further Information Favourable Opinion