Amyotrophic Lateral Sclerosis
Conditions
Brief summary
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)
Detailed description
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
Interventions
DRUGVTx-002
DRUGVTx-002 Diluent
Sponsors
VectorY Therapeutics B.V.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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) | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 | — |
Outcome results
None listed