Early Phase 1 Motor Neurone Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You are willing to participate and have signed a consent form showing you understand the study's purpose and risks
• You are 18 years old or older (any gender)
• You have been diagnosed with ALS (Lou Gehrig's disease) using standard international diagnostic guidelines, and genetic testing has confirmed you do not carry certain gene mutations (in the SOD1 or FUS genes)
• Your ALS symptoms first appeared less than 2 years ago
• Your lung breathing capacity (measured while sitting) is at least 50% of what would be expected for someone of your age, gender, and height
• You have either stopped taking riluzole (a common ALS medication) long enough before the study, or you have been on a stable dose of it for at least 30 days and will continue that dose throughout the study
• You have either stopped taking edaravone (another ALS medication) long enough before the study, or you have been on a stable dose for at least 60 days and will continue that dose throughout the study

Who may not be able to join:

• You have another condition affecting motor nerve function (such as primary lateral sclerosis or cervical spine disease) that could be confused with ALS
• You have a higher-than-normal risk of bleeding that is not well controlled, including problems with blood clotting, platelet disorders (such as haemophilia), or abnormalities near the area where a spinal injection would be given
• You have serious liver, kidney, or heart disease, uncontrolled immune system disorders, poorly controlled diabetes (confirm with trial site), high blood pressure that is not well managed, or a history of serious stomach ulcers or gastrointestinal bleeding
• You currently have an active infection that requires treatment
• You have had cancer within the past 5 years
• You have significant memory or thinking problems, dementia, or unstable mental health conditions such as psychosis, thoughts of suicide, or untreated severe depression in the past 90 days
• You are currently taking certain medications, herbal remedies, or over-the-counter products that strongly affect how the body processes drugs (such as ketoconazole, erythromycin, or grapefruit juice) (confirm specific medications with trial site)
• You have taken certain other ALS treatments or experimental drugs within one month before your first dose (confirm with trial site which medications apply)
• You are expected to need blood-thinning or anti-clotting medications around the time of spinal injections
• You have received immune-suppressing treatment within the past 3 months
• You have a surgically implanted drain or tube in your brain or spinal fluid system
• You have previously received gene therapy or stem cell therapy at any point
• You have tested positive for HIV, syphilis, active hepatitis B or C, or active tuberculosis
• You have significant abnormalities in blood test results that the doctor feels would make participation unsafe
• You have a specific type of heart rhythm abnormality detected on an ECG (heart tracing) (confirm with trial site)
• You have previously had a serious allergic reaction to the study drug or similar medications
• You cannot take corticosteroids (steroid medications) or sirolimus for any reason
• You have had drug or alcohol misuse issues within the past 6 months that could affect your ability to participate
• You drink more than 14 units of alcohol per week or smoke more than 5 cigarettes per day on average
• You are currently pregnant or breastfeeding
• The trial doctor or sponsor believes, for any other reason, that participation would not be appropriate for you

Important: Always verify eligibility with the trial site directly before applying.