Phase 2 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 have been diagnosed with ALS (also known as Lou Gehrig's disease), confirmed using established medical criteria that look at nerve and muscle function, how symptoms have spread, and ruling out other conditions.
• People aged 18 to 75 years old (male or female).
• People who are willing and able to sign a consent form agreeing to take part in the study.
• People who, in the trial doctor's judgment, are able to complete all study visits, tests, and procedures.
• People whose ALS motor weakness symptoms began no more than 24 months (2 years) ago.
• People whose overall ALS function score (ALSFRS-R) is 38 or higher at the screening visit.
• People whose ALS breathing-related function score is 9 or higher at the screening visit.
• People whose ALS swallowing and speech-related function score is 9 or higher at the screening visit.
• People whose lung capacity (forced vital capacity) is greater than 70% of what would be expected for someone of their age and size.
• People whose peak breathing-in flow rate is at least 100 litres per minute.
• People who have been taking the standard ALS medication Riluzole at a stable dose for at least 4 weeks before signing the consent form.
• Women who could become pregnant must agree to use highly effective contraception during the study and for 3 months after the last dose, and must agree not to donate eggs during that time. Accepted options include hormonal contraceptives, intrauterine devices, confirmed post-menopause (no period for at least 1 year), or surgical sterilisation.
• Men who are sexually active with a woman who could become pregnant must agree to use highly effective contraception, or a combination of two acceptable methods, during the study and for 3 months after the last dose, and must agree not to donate sperm during that time.

Who may not be able to join:

• People whose ALS function score drops by 2.5 points or more between the screening visit and the first day of the study.
• People whose ALS started with swallowing or speech problems (bulbar onset), with a bulbar subscore below 9.
• People who are currently using any form of breathing support, such as a CPAP machine, non-invasive ventilation, a breathing machine through a tracheostomy, or supplemental oxygen at any time of day.
• People who have another significant neurological condition that could affect study assessments, such as serious memory or thinking problems or clinical dementia.
• People with serious psychiatric conditions such as schizophrenia or bipolar disorder. People with depression may be considered, but only if their depression has been stable with no major depressive episode in the past year.
• People with serious heart conditions, including certain abnormal heart rhythms, significant heart failure, a heart attack, or unstable chest pain within the 6 months before screening.
• People with moderate to severe lung disease, or who have difficulty using inhaled medications.
• People who are unable to use a dry powder inhaler device to take inhaled medication.
• People who have taken another experimental drug or treatment within 30 days or 5 half-lives of that drug (whichever is longer) before starting the study.
• People who regularly use inhaled protein-based medications (such as inhaled insulin or inhaled parathyroid hormone).
• People who weigh 32 kg or less, or whose body mass index (BMI) is below 17.5 or above 35.0 at screening.
• People with moderate to severe liver disease, including cirrhosis, liver cancer, or active hepatitis, or with certain abnormal liver blood test results.
• People with moderate to severe kidney disease, based on a specific blood test measuring kidney filtering ability.
• People with any other significant medical condition or abnormal test result that the trial doctor believes could affect safety or the study results.
• Women who are pregnant or breastfeeding.

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