DefINe
Research type
Research Study
Full title
A randomised, double-blind, placebo-controlled trial examining the safety and efficacy of deferiprone in patients with neuroferritinopathy
IRAS ID
1006406
Contact name
Patrick Chinnery
Contact email
Sponsor organisation
Cambridge University Hospitals NHS Foundation Trust and University of Cambridge
Eudract number
2022-000216-58
Research summary
Neuroferritinopathy is a disorder caused by a mutation (fault) in a gene which leads to iron gradually building up in the brain. The build-up of iron eventually leads to the death of brain cells (called neurons), and certain brain regions that help control movement are particularly affected.
There are no treatments known to reduce or slow the progression of neuroferritinopathy. Some treatments are available to help with the symptoms of neuroferritinopathy, but these may only be effective for a limited amount of time and have significant side effects.
Deferiprone is a medication that is used to remove excess iron from the body and is licensed for use in the UK for a condition called thalassaemia major. It has been shown in a related disorder that deferiprone is able to reduce brain iron and slow clinical progression, and we now want to test this in people with neuroferritinopathy. The purpose of the trial is to test whether deferiprone can reduce the iron build-up in the brain of people with neuroferritinopathy.
Participants in this trial will take tablets twice a day for 12 months. There is a 50% chance (much like flipping a coin) that the tablets will contain deferiprone. The other half of participants will receive a ‘dummy drug’ called a placebo. Neither the participant nor the trial doctor will know which treatment is being taken, although the trial doctor can find out if necessary.
The trial is being conducted in Cambridge, although participants may be recruited from elsewhere. Participants will need to attend four in-person visits in Cambridge over a 12-month period, as well as have monthly phone calls and regular blood tests between the in-person visits. A final phone visit will take place at 13 months.
To be eligible, participants must have a genetic diagnosis of neuroferritinopathy, be aged 16 or over, and fulfil additional eligibility criteria.
REC name
Wales REC 3
REC reference
23/WA/0145
Date of REC Opinion
5 Sep 2023
REC opinion
Further Information Favourable Opinion