Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder. Patients experience problems such as muscle weakness, difficulty in walking, chewing, swallowing, talking, shortness of breath, and occasionally behavioral changes. ALS is one of the most common neuromuscular diseases in the world and affects 1-3 people per 100,000 a year globally.
Current treatments for patients with ALS aim to treat the individual symptoms. The ORARIALS-01 study is investigating whether a new investigational treatment, arimoclomol, is effective in slowing down the progression of ALS in patients by addressing the underlying biology of the disease.
If you qualify and are selected for this study, your involvement would be up to 18 months. During this time, you will be expected to take this study drug as directed by the study doctor and assist the study doctor and their team by participating in clinical assessment measures and answering questionnaires. If you are invited to the clinic to discuss the study in more detail by your selected study site, you will be provided with more information and have the opportunity to ask questions.
In the study, doctors will compare the study drug to a placebo. A placebo is a treatment that looks identical to the study drug but doesn’t contain any active drug. At the start of the study, all patients are randomly assigned to either receive the study drug or a placebo. Neither you nor your study doctor will know which treatment you are receiving. This method allows doctors to evaluate what benefits the study drug might have on patients who receive it.
Clinical research studies are conducted to determine whether the drug(s) being used in the study are safe and effective in treating a particular disease.
All drugs must go through several phases of clinical research before they can be made available to the public. The earliest clinical study phases that investigational drugs go through look at whether a drug is safe and explore any side effects it causes. A later phase clinical study aims to test whether a new treatment is better than existing treatments. This is a phase III clinical study, which is investigating whether the study drug, arimoclomol, is safe and effective in treating ALS.
Arimoclomol has been previously investigated in phase II and III studies where no significant safety concerns were observed and there were indications of it's effectiveness.
Every person who takes part in a clinical research study is supported by a dedicated team that includes doctors, nurses, and other healthcare professionals. The commitment of each participant and the entire study team is important to help meet the objectives of the study. All clinical research studies are performed according to strict ethical and governmental guidelines to ensure that participants’ rights are protected while the information is being collected.
Your participation in this study may last up to 82 weeks. You are free to withdraw at any time. If you wish to withdraw from the study, please contact your study doctor.
You are free to leave the study at any time and this will not affect your future care in any way. Your doctor will continue to look after you and advise you about alternative treatment options.
All medications, both those that are approved and those undergoing clinical trials, may potentially cause unwanted side effects – some of these are known, but there could be others that have not previously been observed. Study procedures may also involve risks. You will be informed about all of the known potential side effects of the study drug and study procedures before you decide to take part in this clinical study. If any further risks are discovered while the study is ongoing, you will be informed immediately.
If you do experience any symptoms or discomfort during the study, you can contact the study doctor at any time to discuss the best course of action for you. The study treatment can be stopped if you or the study doctor have any concerns.
ALS is a degenerative disease, so it is expected that your symptoms will change significantly over the course of the study. Your study doctor will be closely monitoring your health at all times and will discuss additional or alternative treatment options with you, if necessary. These options may include: continuing with the study treatment, withdrawing from the study, switching to a different type of treatment, or taking medication to relieve your symptoms.
Your health and wellbeing are of utmost importance during the study and any decisions regarding the study treatment or any other treatments will be made with that in mind.
Your data will be collected, handled, and processed in compliance with applicable regulation guidelines. It will not be shared with anyone other than in circumstances that will be explained in your consent form.
Your identity will not be known to the company sponsoring this clinical trial or any of their research partners. Your identity will be replaced with a Subject Identification Number.