Alpha 1-Antitrypsin Deficiency
Conditions
Brief summary
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-634 at various doses.
Detailed description
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.).
Interventions
DRUGVX-634
Suspension for oral administration.
DRUGPlacebo
Suspension for oral administration.
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
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 * Participants of non childbearing potential * Non smoker or ex-smoker for at least 3 months before screening Key
Exclusion criteria
* Any condition possibly affecting drug absorption Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | 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 |
Secondary
| Measure | Time frame |
|---|---|
| Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-634 | Day 1 up to Day 20 |
| Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-634 | Day 1 up to Day 20 |
| Part A and B: Urine Concentration of VX-634 | Part A Cohort A3: Days 1 and 5; Part B Cohort B3: Days 1, 10 and 11 |
Countries
United States
Outcome results
None listed