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The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-668 at various doses.
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-668 | Drug | Suspension for oral administration. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Day 20 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-668 | Day 1 up to Day 20 | |
| Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-668 | Day 1 up to Day 20 | |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Celerion - Tempe | Tempe | Arizona | 85283 | United States | ||
| University of Florida |
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
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| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| Placebo |
| Drug |
Suspension for oral administration. |
|
| Part A and B: Urine Concentration of VX-668 |
| Part A Cohort A3: Days 1 and 5; Part B Cohort B3: Days 1, 10, and 11 |
| Gainesville |
| Florida |
| 32610 |
| United States |
| Central Florida Pulmonary Group, P.A. | Orlando | Florida | 32803 | United States |
| ICON Lenexa | Lenexa | Kansas | 66219 | United States |
| ICON Salt Lake City | Salt Lake City | Utah | 84124 | United States |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |