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A Phase 1, First-in-human Study of VX-668

Phase 1
Completed
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
Drug: Placebo
Registration Number
NCT05727800
Lead Sponsor
Vertex Pharmaceuticals Incorporated
Brief Summary

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-668 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.).

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
114
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 condition possibly affecting drug absorption
  • Females of childbearing potential

Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Part AVX-668Participants grouped in different cohorts will receive a single ascending dose of VX-668.
Placebo Part APlaceboParticipants will be randomized to receive placebo matched to VX-668.
Part BVX-668Participants 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 BPlaceboParticipants will be randomized to receive placebo matched to VX-668.
Primary Outcome Measures
NameTimeMethod
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
NameTimeMethod
Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-668Day 1 up to Day 20
Part A and B: Urine Concentration of VX-668Part A Cohort A3: Days 1 and 5; Part B Cohort B3: Days 1, 10, and 11
Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-668Day 1 up to Day 20

Trial Locations

Locations (5)

ICON Salt Lake City

🇺🇸

Salt Lake City, Utah, United States

Celerion - Tempe

🇺🇸

Tempe, Arizona, United States

University of Florida

🇺🇸

Gainesville, Florida, United States

ICON Lenexa

🇺🇸

Lenexa, Kansas, United States

Central Florida Pulmonary Group, P.A.

🇺🇸

Orlando, Florida, United States

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