EXPErimental medicine Route To Success in Amyotrophic Lateral Sclerosis (EXPERTS-ALS)
Amyotrophic lateral sclerosis (ALS) is the most common form of motor neuron disease. It causes muscle weakness that worsens over time, and current treatment options are very limited. The EXPERTS-ALS study is testing several well-known drugs already used for other health conditions to see if any can help people with ALS. In this study, researchers measure a nerve protein in the blood called neurofilament light chain (NFL). High levels of NFL suggest nerves are under stress. If a drug lowers these levels, it could mean it helps protect nerve cells. This is an 'adaptive' study, meaning scientists can quickly drop drugs that show little promise and test new ones. There is no placebo (dummy pill), so everyone who joins will take an active medication for up to six months while being closely monitored by doctors.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
Amyotrophic lateral sclerosis (ALS) is a serious condition that affects the nerves responsible for movement. Over time, it leads to muscle weakness, making everyday tasks like walking, talking, swallowing, and breathing harder. Right now, there is no cure, and finding new treatments has historically taken a very long time. The EXPERTS-ALS trial aims to speed up this search by screening several potential medications quickly and safely in people living with the condition.
Rather than inventing brand-new treatments from scratch, the study tests 'repurposed' drugs. These are medicines already approved and safely used for other medical conditions, such as diabetes, high blood pressure, or Parkinson's disease. Because doctors already know a lot about how these medicines work and their safety, researchers can test them much faster to see if they offer any benefit for ALS.
To find out if a drug is working, the research team measures a protein called neurofilament light chain (NFL) in your blood. When motor nerves are damaged, they leak NFL into the bloodstream. If a trial drug lowers NFL levels, this shows it may be helping to protect nerves from further damage. Drugs that show clear promise can then quickly move into larger clinical trials.
النقاط الرئيسية
- Tests repurposed, existing medicines to see if they can slow down ALS.
- Every participant receives an active medication; there are no dummy pills.
- Uses a simple blood test (NFL level) to see if drugs protect nerve cells.
- Involves taking a medication for up to six months with hospital and remote check-ups.
- Helps researchers rapidly choose the most promising treatments for larger trials.
من قد يكون مؤهلاً؟
This trial is open to adults aged 18 and older who have a confirmed diagnosis of ALS. To take part, your condition must fall within a specific disease progression range determined by your study doctor. If you are taking riluzole, you must have been on a stable dose for at least one month before starting, or have chosen not to take it.
You cannot join if you are taking part in another drug trial, or if you have other serious illnesses affecting your heart, liver, or kidneys that could make taking the study medicines unsafe. You also cannot join if you have another active condition that alters nerve protein levels in the blood.
Because the study compares different medicines, you must be medically able to take at least two of the drugs being tested. Those who need breathing machines for more than 22 hours a day, or who are pregnant or breastfeeding, are not eligible to take part.
هل يمكن أن تناسبك هذه الدراسة؟
أجب عن هذه الأسئلة السريعة لترى ما إذا كنت مؤهلاً. هذا دليل فقط - الفريق البحثي هو الذي يتخذ القرار النهائي.
- I am aged 18 or over and have a confirmed diagnosis of ALS.
- I have been on a stable dose of riluzole for at least 30 days, or I do not take it.
- I am not currently taking part in another drug trial.
- I do not use a breathing machine for more than 22 hours a day.
- I am not pregnant or breastfeeding, and I am willing to use effective contraception if required.
ماذا يتضمن المشاركة؟
If you join the study, you will take an active study drug for up to six months. There is no dummy pill (placebo), so you and your doctor will know exactly which medication you are receiving. Before you start, the team will check your medical history and take baseline measurements, including blood tests.
During the six months, you will attend hospital visits so the medical team can monitor your health and check for any side effects. To make taking part easier, appointments at week four and week eight can often be done from home by phone or video call. You will also have three fasting blood tests: one at the start, one at week 12, and one at week 24. For these visits, you will be asked not to eat from 8:30 pm the evening before and drink only water until after your morning blood test.
Travel costs for hospital visits can be reimbursed. The study team will stay in regular contact with you, and you can stop taking the medication or leave the study at any point without it affecting your usual NHS care.
المخاطر والفوائد المحتملة
المواقع (15)
- Royal Hallamshire HospitalتقريبيSheffield, England
- Oxford Radcliffe Hospital NHS Trustالمدينة فقطأكسفورد، إنجلترا
- The Anne Rowling Regenerative Neurology Clinicالمدينة فقطإدنبرة، اسكتلندا
- Royal Devon University Healthcare NHS Foundation Trustالمدينة فقطExeter, England
- مستشفى كينغز كوليدجتقريبيلندن، إنجلترا
- مستشفى سالفورد الملكيغير مؤكدSalford, England
- مستشفى جامعة ويلزغير مؤكدكارديف، ويلز
- مستشفى كلية الجامعةغير مؤكدلندن، إنجلترا
- مستشفى رويال فيكتورياغير مؤكدNewcastle upon Tyne, England
- The Walton Centre NHS Foundation Trustغير مؤكدLiverpool, England
- St George's Hospitalغير مؤكدلندن، إنجلترا
- صندوق الرعاية الصحية والاجتماعية الغربيةغير مؤكدلندنديري، أيرلندا الشمالية
أسئلة شائعة
Will I receive a dummy pill (placebo)?
No. In this trial, there is no dummy pill. Every participant receives one of the active study medications.
Can I stay on my regular ALS medication, like riluzole?
Yes, provided you have been on a stable dose of riluzole for at least 30 days before joining the study.
How long will I take the study drug?
You will take the assigned study medication for up to six months, with regular check-ups to monitor your progress.
Do I have to travel to the hospital for every visit?
No. While some visits require blood tests at the hospital, your week 4 and week 8 check-ups can usually be done by video or phone.
What happens if I experience unpleasant side effects?
You should tell your study team straight away. They can adjust your dose, pause the medication, or stop it completely if necessary.
كيفية معرفة المزيد
Jennifer Petrie
تحدث دائمًا إلى طبيبك العام أو الأخصائي قبل اتخاذ قرار بالمشاركة في دراسة.
هل أنت مهتم بالمشاركة؟
نقاش
مناقشة المجتمع
مدعوم من منتدانا في community.patient.info. يرجى التحلي بالاحترام — هذه ليست نصيحة طبية.