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AMT-162 · 1 trial · 1 indication
Occurrence of TEAEs upon administration of ascending doses of AMT-162
| Arm | Type | Description |
|---|---|---|
| 3 single Ascending Dose Levels | EXPERIMENTAL | Experimental: 3 single Ascending Dose Levels The study will be open-label with an initial plan to explore 3 dose levels of AMT-162 in approximately 6 to 12 Participants in total. Each Participant will receive a single dose of AMT-162 delivered via an intrathecal (IT) infusion and will be followed for up to 5 years after AMT-162 administration. |
| EXPANSION COHORT | EXPERIMENTAL | Expansion cohort: To further test selected dose from the SAD part in approximately 6 to 8 participants The study will be open-label. Each Participant will receive a single dose of AMT-162 delivered via an intrathecal (IT) infusion and will be followed for up to 5 years after AMT-162 administration. |
| Name | Type | Description |
|---|---|---|
| AMT-162 | DRUG | AMT-162, the investigational product (IP), is a nonreplicating, rep/cap-deleted, self-complementary Recombinant adeno-associated virus (rAAV) vector based on adeno-associated virus (AAV) serotype rh10 and contains complementary deoxyribonucleic acid (cDNA) encoding an artificial miRNA targeting the SOD1 gene. |
Inclusion Criteria: * Confirmed clinical and genetic diagnosis of SOD1-mediated ALS (SOD1-ALS) experiencing signs and/or symptoms of lower motor neuron dysfunction (weakness, atrophy, cramps, fasciculations), with or without upper motor neuron symptoms (weakness, bring reflexes, spasticity). * ALSF...
AMT-162 is an investigational gene therapy being developed by uniQure N.V. for the treatment of amyotrophic lateral sclerosis (ALS). It is designed to target SOD1 and is administered intrathecally. The therapy is currently in Phase 1 clinical development.
AMT-162 is being developed for the treatment of amyotrophic lateral sclerosis (ALS), specifically in patients with SOD1 mutations. It is not yet approved for any indication and remains an investigational therapy in clinical development.
AMT-162 targets SOD1, the gene encoding superoxide dismutase 1. By targeting SOD1, the therapy aims to address the underlying genetic cause of SOD1-ALS. The approach is designed to reduce toxic SOD1 protein levels.
AMT-162 is being developed by uniQure N.V., a gene therapy company. uniQure trades on the Nasdaq under the ticker symbol QURE. The company is responsible for the clinical development of AMT-162.
AMT-162 is in Phase 1 clinical development. The ongoing trial is evaluating its safety, tolerability, and exploratory efficacy in adults with SOD1-ALS. It has not been approved by the FDA or any regulatory agency.
AMT-162 is being studied in a Phase 1 trial, NCT06100276, titled "Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)." The trial is active but not recruiting, with an estimated enrollment of 20 participants in the United States and Sweden.