Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002.

Trial status: Authorised Change in revised ALS Functional Rating Scale (ALSFRS-R) over 6 and 12 months, Time to permanent assisted ventilation, or death over 12 months, Change in slow vital capacity (SVC) over 6 and 12 months, Survival, Immunogenicity against adeno-associated virus (AAV) capsid Nature, incidence, severity, relatedness, seriousness, and outcome of treatment-emergent adverse events (TEAEs) including: o Laboratory values o Magnetic resonance imaging (MRI) findings o Treatment-induced Peripheral Neuropathy Assessment Scale (TNAS) o Assessment of cellular responses to both the vector and the transgene encoded protein o Columbia Suicide Severity Rating Scale (C-SSRS)

Change in revised ALS Functional Rating Scale (ALSFRS-R) over 6 and 12 months, Time to permanent assisted ventilation, or death over 12 months, Change in slow vital capacity (SVC) over 6 and 12 months, Survival, Immunogenicity against adeno-associated virus (AAV) capsid

Age group: 65+ years, 18-64 years Gender: Female, Male Target enrollment: 4 participants