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:

• People who are able to understand the purpose and risks of the study and are willing to give their formal consent to participate, in line with local laws.
• People who are 18 years of age or older.
• People who have been diagnosed with ALS (also known as motor neurone disease) — either confirmed or probable — by a doctor experienced in managing ALS.
• People whose first ALS symptom (such as weakness in the limbs, throat/mouth area, or trunk) began less than 24 months before entering the study.
• People who are taking riluzole and/or edaravone must have been on a stable dose of that medication for at least 30 days before starting the study, and must continue that stable dose throughout the trial.
• Women who could become pregnant must agree to use an acceptable form of contraception for the entire trial period and for 60 days after the last dose of the study drug, or must be confirmed as unable to become pregnant (e.g., post-menopausal for at least one year or surgically sterile).
• Female participants, or female partners of male participants, must not be pregnant or planning to become pregnant during the trial or for up to 90 days after the last dose of the study drug.
• Male participants must agree not to donate sperm and to use contraception with a female partner during the trial and for at least 90 days after the last dose of the study drug.

Who may not be able to join:

• People who have had a tracheostomy (a surgical opening in the throat for breathing) or who rely on permanent assisted ventilation.
• People whose lung capacity (measured by a specific breathing test called SVC) is below 65%.
• People with abnormal liver function shown in recent blood tests (within the past 4 weeks), unless the result is due to a harmless inherited condition called Gilbert syndrome (confirm with trial site).
• People with reduced kidney function, as measured by a specific blood test (eGFR below 60 mL/min/1.73m²).
• People with certain other abnormal blood test results, including those related to blood clotting or platelet levels (confirm with trial site).
• Women who are pregnant (confirmed by a test within 7 days before the first dose) or who are currently breastfeeding.
• People with another serious or unstable medical condition (aside from ALS) that, in the investigating doctor's opinion, could affect their safety or ability to take part.
• People with significant abnormalities found during a physical or neurological examination, in vital signs, or on a heart tracing (ECG), that in the doctor's opinion could affect safety.
• People with unstable psychiatric illness, significant cognitive difficulties, dementia, or substance misuse that could affect their ability to consent or follow study procedures.
• People who are currently enrolled in, or who have recently participated in, another clinical trial using an investigational treatment — in most cases within 30 days of the last dose of that study drug.
• People who have previously received certain gene-related or cell-based treatments, including small interfering RNA therapy, stem cell therapy, antisense oligonucleotide (ASO) therapy, or gene therapy.
• People for whom a lumbar puncture (spinal injection) would not be medically appropriate, or who have a condition that could be made worse by repeated spinal injections.
• People who have had a severe allergic reaction to any component of the study drug in the past, or who are known to be sensitive to it.

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