Phase 2 Motor Neurone Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- People who are willing and able to give written consent and follow all study requirements, including attending study site visits
- People aged 18 or older, of any gender
- People who have been diagnosed with ALS (also called Motor Neurone Disease) according to a specific set of medical criteria called the El Escorial criteria, at the probable or definite level
- People whose ALS has been confirmed through laboratory testing to not be caused by changes in the FUS or SOD1 genes
- People whose first signs of weakness (such as weakness in the arms or legs, difficulty speaking, difficulty swallowing, or shortness of breath) appeared no more than 18 months ago
- People whose lung breathing capacity, measured while seated upright, is at least 80% of the expected level at the time of screening
- People whose disease progression falls within a specific range on a scoring system called the TRICALS risk score (between -2 and -6) at the time of screening
- People who have a reliable caregiver, partner, or legal representative who is willing and able to support them throughout the study, and who can provide consent on their behalf if the disease progresses to a point where the person cannot do so themselves
- People already taking riluzole and/or edaravone may be eligible, provided the dose has been stable for at least 2 weeks (riluzole) or one full treatment cycle (edaravone) before the screening visit
- Women who could become pregnant, and men whose female partners could become pregnant, must agree to use highly effective contraception during and after the study; women who could become pregnant must not currently be pregnant or breastfeeding
- Women who cannot become pregnant must either be post-menopausal or have had a surgical procedure that prevents pregnancy (such as removal of the uterus, removal of both ovaries, or tubal ligation)
Who may not be able to join:
- People who have been diagnosed with another significant brain or nervous system condition, separate from ALS, that could explain their symptoms or affect study results
- People whose spinal, neck, or brain scans (MRI or MRA) show a significant abnormality
- People who already have a tracheostomy (a breathing tube in the throat) or a feeding tube at the time of screening
- People who have medical reasons why they cannot take corticosteroids (steroid medications), such as osteoporosis, uncontrolled blood pressure, diabetes, or high cholesterol
- People who have had a serious illness or medical condition within the 6 months before screening that could pose a safety risk or interfere with the study, including heart disease, uncontrolled diabetes, liver disease, autoimmune conditions requiring strong immune-suppressing treatment, cancer, or a current psychiatric diagnosis
- People whose blood test results at screening show significant problems, such as poor liver or kidney function, abnormal blood clotting, or infections such as Hepatitis or HIV
- People who are taking blood-thinning medications (such as warfarin, heparin, or similar drugs) and are unable to safely stop them before certain study procedures
- People who cannot have MRI, MRA, or CT scans due to severe claustrophobia or an intolerance to contrast agents used in imaging
- People who have medical reasons why general anaesthesia or deep sedation would not be safe for them
- People who test positive for illegal drugs (note: prescribed medications and responsibly used medicinal or recreational marijuana where permitted may be exceptions — confirm with trial site)
- People who are generally considered frail, or where the treating doctor considers that joining the study would not be in the person's best interest, or that the person is unlikely to be able to continue participating throughout the study
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Phone: patients@vectorytx.com
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs); The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs); The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs); The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs); The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs); The number of participants with treatment related adverse events (AEs)...
Can't join this trial?
Data last synced from ClinicalTrials.gov: 23 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.