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AMT-191 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Dose Ranging Cohort 1 | EXPERIMENTAL | - |
| Dose Ranging Cohort 2 | EXPERIMENTAL | - |
| Dose Ranging Cohort 3 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| AMT-191 | DRUG | A recombinant serotype 5 based adeno-associated viral vector (AMT-191) for one-time intravenous (IV) administration will be investigated in this study. This recombinant AAV5-based vector contains a coding deoxyribonucleic acid (DNA) sequence for human α-galactosidase A. Delivery of AMT-191 to the systemic circulation is expected to result in a therapeutic effect by promoting the liver expression of the lysosomal enzyme GLA in plasma levels in patients with Fabry disease. |
Key Inclusion Criteria: * Male of age ≥ 18 years and ≤50 years * Confirmed clinical diagnosis of classic Fabry disease (FD) defined as: 1. Absent or minimal αGAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR 2. α-galactosidase A (GLA) pathogenic or ...
AMT-191 is an investigational small molecule being developed for the treatment of Fabry Disease, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is designed to address the underlying enzyme deficiency associated with the condition.
AMT-191 targets α-Gal A, an enzyme that is deficient or non-functional in patients with Fabry Disease. By targeting this enzyme, the drug aims to address the metabolic pathway affected by the disease. This mechanism is central to its potential therapeutic effect in treating the condition.
AMT-191 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 drug for Fabry Disease.
AMT-191 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities such as the FDA. The ongoing Phase 1 trial is designed to assess the drug's safety, pharmacokinetics, pharmacodynamics, and exploratory efficacy in patients.
AMT-191 is being evaluated in a Phase 1 clinical trial with the identifier NCT06270316. This study, titled 'Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease,' is currently recruiting participants in the United States. The trial is enrolling male patients aged 18 years and older, with a target enrollment of 12 participants.
AMT-191 is the primary name for this investigational drug. No alternative names have been reported for this asset. It is being studied specifically for its potential role in treating Fabry Disease and is distinct from other therapies in development for the same condition.