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.

Condition studied

Amyotrophic Lateral Sclerosis

Investigational medicinal product(s)

Eligibility

Primary endpoint

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)

Endpoint detail

Change in revised ALS Functional Rating Scale (ALSFRS-R) over 6 and 12 months, Time to permanent assisted ventilation, or death over 52 weeks, Change in slow vital capacity (SVC) over 6 and 12 months, Survival, Immunogenicity against adeno-associated virus (AAV) capsid