FELL-HD-s
Research type
Research Study
Full title
FELL-HD-s: A trial to assess markers of autophagy in patients with Huntington’s disease who have been treated with felodipine
IRAS ID
297596
Contact name
Roger Barker
Contact email
Sponsor organisation
Cambridge University Hospitals NHS Foundation Trust & University of Cambridge
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Huntington's disease (HD) is an inherited condition that causes damage to cells in the brain over time due to the production of an abnormal protein called mutant huntingtin (mHTT). Currently there is no cure for HD and its progress cannot be reverse or slowed down.
One way to try and stop HD progressing is to increase (upregulate) its clearance from cells. One normal process that cells use to clear proteins (including mHTT) is called autophagy.
Felodipine is a drug which has been shown in animal models to upregulate the autophagy process. The purpose of this trial is to investigate whether felodipine is able to increase autophagy in people with HD by looking at markers in the blood and/or cerebrospinal fluid (CSF).
We plan to recruit up to 18 participants in this trial. Participants are early-stage HD patients who will be treated with felodipine through the FELL-HD trial. The participant’s trial duration will be up to 66 weeks, consisting of a 4 week screening period and a 62 week sample collection period.Lay summary of study results: In the FELL-HD samples (FELL-HD-s) study we collected blood samples as well as samples of CSF- the fluid that surrounds the brain and spinal cord and which is collected by a lumbar puncture. These were collected over the duration of the trial including the time the treatment was given to see whether there were changes in the levels of proteins that are thought to reflect the ongoing disease process. These included markers of nerve cell health (NF-L), the level of the protein linked to the disease (mHtt) as well as a measure of the non-nerve cells in the brain (GFAP). None of these changed significantly over the course of the trial which means that felodipine did not make things worse, nor did it significantly slow down how the disease was progressing using these measures. However, this having been said we only looked over one year and these markers change very little over such times in people with HD normally. So that while we saw no major changes this does not mean that the drug might not be able to have some more positive effects over longer time frames.
REC name
East Midlands - Nottingham 1 Research Ethics Committee
REC reference
22/EM/0007
Date of REC Opinion
2 Mar 2022
REC opinion
Further Information Favourable Opinion