Amyotrofik Lateral Sklerozlu (ALS) Katılımcılarda Engensis'in Güvenliği

This clinical trial investigates the safety of intramuscular Engensis administration in ALSALSAmyotrofik Lateral Skleroz, motor nöronların ilerleyici dejenerasyonu ile karakterize nörodejeneratif bir hastalıktır. patients compared to placebo. The primary endpoint is the incidence of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), injection site reactions, and clinically significant laboratory value changes. Secondary exploratory endpoints include changes in muscle function (using ALSALSAmyotrofik Lateral Skleroz, motor nöronların ilerleyici dejenerasyonu ile karakterize nörodejeneratif bir hastalıktır.FRS-R subscores for Fine and Gross Motor Function, handheld dynamometry, and Accurate Test of Limb Isometric Strength), quality of life (using ALSALSAmyotrofik Lateral Skleroz, motor nöronların ilerleyici dejenerasyonu ile karakterize nörodejeneratif bir hastalıktır.AQ-40), patient and clinician global impression scales, and lung function (Slow Vital CapacitySlow vital capacityAkciğerlerden yavaş ve tam bir şekilde nefes verilebilen hava hacmi; solunum fonksiyonunu değerlendirmek için kullanılır.). Muscle biopsies will be collected for future biomarker analyses. The study focuses on safety and exploratory efficacy signals, and any observed improvements in functional measures should be interpreted cautiously pending further investigation in larger, controlled trials.

Önemli Takib Edilecek Noktalar:
- The primary goal is to evaluate the safety of intramuscular Engensis administration in ALSALSAmyotrofik Lateral Skleroz, motor nöronların ilerleyici dejenerasyonu ile karakterize nörodejeneratif bir hastalıktır. patients.
- The study includes exploratory endpoints to assess potential effects on muscle function, quality of life, and lung function, but these are secondary to the safety evaluation.
- Muscle biopsies will be collected for future biomarker research, which could provide insights into the mechanism of action or potential predictors of response.