Trial ID: ISRCTN15525855
Other - Phase 2 - Recruiting
Background and study aims Amyotrophic lateral sclerosis (ALS), the most common form of motor neuron disease (MND), is a fatal disease affecting 1 in 300 people. ALS causes muscle wastage that gets worse over time, leading to loss of the ability to walk, talk, eat and eventually breathe. There is no cure or highly effective disease-slowing therapy for ALS. Historically, drug trials for ALS have been unsuccessful. A process that can quickly screen drugs in people with ALS, rather than in cells or animal models, is believed will be better at selecting treatments for the large Phase III trials that are needed. EXPERTS-ALS is a UK multicentre study that will screen drugs for their potential to slow the progression of ALS, by looking at changes in blood test markers. Higher blood levels of a marker known as neurofilament light chain (NFL), a building block of nerve cells, are associated with faster rates of ALS disability. A drug will be recommended to go forward into Phase III trials if a large and important reduction in blood NFL levels is seen. Who can participate? Patients with a diagnosis of ALS aged at least 18 years old and over at the time of consent What does the study involve? EXPERTS-ALS is an adaptive trial, meaning that drugs can be added, assessed and removed as the trial is ongoing. Drugs will be selected for the study based on explicit criteria agreed upon by a group of experts. The data generated throughout the study will be analysed, and if a drug does not meet the team's pre-agreed criteria, it will be removed from the study. There will be no placebo/dummy drug. Participants will know what drug they are receiving and will take it for a maximum of 6 months. The study aims to test between 9-12 drugs over 5 years. What are the possible benefits and risks of participating? The results of this study will help to prioritise drugs for testing in larger clinical trials to inform the treatment of future patients with ALS. By taking part in the study, participants are directly helping to do this. Taking part in EXPERTS-ALS will require participants to attend additional hospital appointments. Travel costs to EXPERTS-ALS appointments will be reimbursed if required. To try and minimise the burden, it will be possible for participants to attend the week 4 and week 8 study appointments remotely (via telephone or video call) unless there are drug-specific safety assessments required. The study drugs are all repurposed medications which are used widely in other settings however participants may experience side effects from taking the study drugs. Possible side effects will be listed in the drug-specific PISs, and information on who to contact if side effects are experienced will be included. Participants will also be asked about any side effects experienced at their routine study visits. Depending on the drug, it may be possible to reduce the dose taken if a participant is struggling with side effects. Local study teams will review participants’ treatment regularly and may decide to pause or stop the medication if needed. Fasting blood samples will be required at three time points, one before randomisation (at either the screening or baseline visit), one at week 12 and one at week 24. Participants will be required not to eat anything from 8.30 pm the night before their appointment and to drink only water. These appointments will be scheduled to take place in the morning to minimise inconvenience for participants. Where is the study run from? Royal Hallamshire Hospital, UK When is the study starting and how long is it expected to run for? February 2024 to September 2028 Who is funding the study? EXPERTS-ALS is funded by the Efficacy and Mechanism Evaluation (EME) Programme, a Medical Research Council (MRC) and National Institute for Health and Care Research (NIHR) partnership. Who is the main contact? experts-als@sheffield.ac.uk
Sheffield Teaching Hospitals NHS Foundation Trust
Source: https://www.isrctn.com/