A Phase 1, First-in-human Study of VX-634

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-634 at various doses.

Detailed Description
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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.).

Official Title
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A Phase 1, First-in-human Study of Safety, Tolerability, and Pharmacokinetics of VX-634

Conditions
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Alpha 1-Antitrypsin Deficiency

Intervention / Treatment
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* Drug: VX-634
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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/Exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Part A<br>Participants grouped in different cohorts will receive a single ascending dose of VX-634. | Drug: VX-634<br>* Suspension for oral administration.<br>|
| Placebo Comparator: Placebo Part A<br>Participants will be randomized to receive placebo matched to VX-634. | Drug: Placebo<br>* Suspension for oral administration.<br>|
| Experimental: Part B<br>Participants grouped in different cohorts will receive multiple doses of VX-634.The dose levels will be determined based on the data from Part A. | Drug: VX-634<br>* Suspension for oral administration.<br>|
| Placebo Comparator: Placebo Part B<br>Participants will be randomized to receive placebo matched to VX-634. | Drug: Placebo<br>* Suspension for oral administration.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 |