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VX-668 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Part A | EXPERIMENTAL | Participants grouped in different cohorts will receive a single ascending dose of VX-668. |
| Placebo Part A | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-668. |
| Part B | EXPERIMENTAL | Participants grouped in different cohorts will receive multiple doses of VX-668.The dose levels will be determined based on the data from Part A. |
| Placebo Part B | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-668. |
| Name | Type | Description |
|---|---|---|
| VX-668 | DRUG | Suspension for oral administration. |
| Placebo | DRUG | Suspension for oral administration. |
Key Inclusion Criteria: * Participants of age between 18 to 55 years (inclusive) * Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m\^2) * A total body weight of more than (\>)50 kg * Nonsmoker or ex-smoker for at least 3 months before screening Key Exclusion Criteria: * Any c...
VX-668 is an investigational small molecule developed by Vertex Pharmaceuticals for alpha-1 antitrypsin deficiency, a rare genetic disorder. It is designed to target the AAT protein. VX-668 is in Phase 1 clinical development and has been studied in a completed first-in-human trial.
VX-668 is being developed as a potential treatment for alpha-1 antitrypsin deficiency, a rare inherited condition. It is a small molecule that targets the AAT protein. The drug is investigational and has not been approved for any indication; it has completed a Phase 1 trial.
VX-668 targets the AAT protein, also known as alpha-1 antitrypsin. It is a small molecule designed to interact with this protein. By targeting AAT, the drug aims to address the underlying protein deficiency in alpha-1 antitrypsin deficiency.
VX-668 is being developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is a biopharmaceutical company focused on rare diseases and other serious conditions.
VX-668 is in Phase 1 clinical development. It has completed one Phase 1 trial and is not currently listed as active in further trials. The drug remains investigational and has not been approved by the FDA for any use.
VX-668 has been evaluated in a Phase 1 first-in-human study, NCT05727800, titled "A Phase 1, First-in-human Study of VX-668." This completed trial enrolled 114 participants with alpha-1 antitrypsin deficiency in the United States and included healthy volunteers.