FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS

Korábbi kezelés

• Has a tracheostomy or requires continuous assisted ventilation more than 22 hours per day during the preceding 3 months before the first Screening Visit
• Has a contraindication to brain MRI (e.g., pacemaker, metal implants)
• Is currently enrolled in another clinical trial or has received an investigational treatment within the past 4 weeks
• Has previously received gene therapy, stem cell therapy, or another Antisense oligonucleotide (ASO) treatment

Más betegség

• Has a contraindication to lumbar puncture or spinal injection (e.g., blood clotting disorders, certain blood thinners, signs of increased pressure in the brain)
• Has a clinically significant condition other than ALS that could interfere with study participation

Egyéb

• Carries a confirmed SOD1 or FUS gene mutation
• Has significant abnormal liver, kidney, or blood test results

• Adults aged 18 to 75 years
• Diagnosis of ALS (clinically definite, clinically probable, or clinically probable laboratory supported)
• ALS symptoms began within the past 24 months
• Able to perform breathing tests: slow vital capacity (SVC) with consistent results, with breathing capacity of at least 60% of the expected value
• Able and willing to meet all study requirements, including travel to the study site, brain magnetic resonance imaging (MRI) scans, lumbar punctures, and blood draws
• Able and willing to use wearable sensors and complete speech assessments at home
• On a stable dose of approved ALS medication for at least 4 weeks prior to screening
• Capable of providing informed consent