TIRCON2012V1-EXT
Research type
Research Study
Full title
Long-term Safety and Efficacy Study of Deferiprone in Patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN)
IRAS ID
190506
Contact name
Patrick Chinnery
Contact email
Sponsor organisation
ApoPharma Inc
Eudract number
2014-001427-79
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 1 months, 31 days
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 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 sadly many patients die in childhood or early adulthood. At present there is no proven treatment.
Study TIRCON2012V1-EXT is an 18-month, multi-center, single-arm, open-label extension of study TIRCON2012V1, which began in 2012 and is currently in progress. In the initial study, patients with a diagnosis of PKAN were randomised in a 2:1 ratio to receive 18 months of treatment with either deferiprone or placebo, respectively. All participants who complete TIRCON2012V1 will be offered the opportunity to continue in this extension study, with the final visit of the initial study being considered Visit 1 of the extension study.
Patients who had been randomised to receive deferiprone in the initial study (TIRCON2012V1) will continue to receive deferiprone in the extension study, while those who had been randomised to receive placebo will also receive deferiprone. Since the initial study will still be in progress at the time that the first patients enter the extension study, patients and study staff will remain blinded as to which product was received for the previous 18 months.
Patients will have their hematology blood counts monitored weekly, and will return to the study site at 6 months (Visit 2), 12 months (Visit 3), and 18 months (Visit 4) for assessments of safety and efficacy.REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
15/YH/0492
Date of REC Opinion
16 Dec 2015
REC opinion
Further Information Favourable Opinion