Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VTx-002 · 1 trial · 1 indication
Assessed by reviewing the nature, incidence, severity, relatedness, seriousness and outcome of treatment emergent adverse events.
| Arm | Type | Description |
|---|---|---|
| Gene Therapy Group 1: Dose 1 (Low Dose) | EXPERIMENTAL | Gene Therapy: VTx-002 6 participants will receive dose 1 administered intra cisterna magna. Dosing of the first 3 participants will be staggered at specific timepoints apart and with a safety monitoring committee review of health-related information in between each participant being dosed. VTx-002 is a single dose therapy Drug: Preventative (Prophylactic) Medication: Methylprednisolone |
| Gene Therapy Group 2: Dose 2 (High Dose) | EXPERIMENTAL | Gene Therapy: VTx-002 6 participants will receive dose 2 administered intra cisterna magna. The participant dosing in group 2 will be staggered as it was in group 1. VTx-002 is a single dose therapy. Drug: Preventative (Prophylactic) Medication - Methylprednisolone |
| Name | Type | Description |
|---|---|---|
| VTx-002 | GENETIC | An investigational gene therapy targeting a specific protein. |
| Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone | DRUG | To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance. |
Key Inclusion Criteria: 1. Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit requirements 2. Male or female ≥ 18 years of age 3. Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 20...