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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 for the treatment of Amyotrophic Lateral Sclerosis (ALS) in adult participants with SOD1-ALS. It is administered intrathecally and is currently in Phase 1 clinical development.
AMT-162 targets the SOD1 gene, which is associated with a form of Amyotrophic Lateral Sclerosis known as SOD1-ALS. By targeting this gene, the therapy aims to address the underlying genetic cause of the disease.
AMT-162 is being developed by uniQure N.V., a biopharmaceutical company listed on the stock exchange under the ticker symbol QURE. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy.
AMT-162 is currently in Phase 1 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The ongoing Phase 1 trial is designed to assess its safety, tolerability, and exploratory efficacy.
AMT-162 is being studied in a Phase 1 clinical trial with the identifier NCT06100276. This trial is titled 'Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)' and is currently active but not recruiting.
AMT-162 is a gene therapy specifically designed for SOD1-ALS, a genetic form of Amyotrophic Lateral Sclerosis. It is administered intrathecally and targets the SOD1 gene. The ongoing Phase 1 trial is enrolling participants in the United States and Sweden.