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

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

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

Study Overview

• Status: Completed
• Conditions: Alpha 1-Antitrypsin Deficiency
• Intervention / Treatment: Drug: Placebo; Drug: VX-634

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.).

• Study Type: Interventional
• Enrollment (Actual): 127
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Medical Information; Phone Number: 617-341-6777; Email: medicalinfo@vrtx.com

Study Locations

• United States
  • Arizona
    • Tempe, Arizona, United States, 85283
      • Celerion - Tempe
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
    • Orlando, Florida, United States, 32803
      • Central Florida Pulmonary Group, P.A.
  • Kansas
    • Lenexa, Kansas, United States, 66219
      • ICON Lenexa
  • Utah
    • Salt Lake City, Utah, United States, 84124
      • ICON Salt Lake City

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part A; Participants grouped in different cohorts will receive a single ascending dose of VX-634.	Drug: VX-634; Suspension for oral administration.
Placebo Comparator: Placebo Part A; Participants will be randomized to receive placebo matched to VX-634.	Drug: Placebo; Suspension for oral administration.
Experimental: Part B; 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; Suspension for oral administration.
Placebo Comparator: Placebo Part B; Participants will be randomized to receive placebo matched to VX-634.	Drug: Placebo; Suspension for oral administration.

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome 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

Collaborators and Investigators

• Sponsor: Vertex Pharmaceuticals Incorporated

Study record dates

Study Major Dates

• Study Start (Actual): October 13, 2022
• Primary Completion (Actual): May 24, 2023
• Study Completion (Actual): November 13, 2023

Study Registration Dates

• First Submitted: October 10, 2022
• First Submitted That Met QC Criteria: October 10, 2022
• First Posted (Actual): October 13, 2022

Study Record Updates

• Last Update Posted (Actual): March 12, 2024
• Last Update Submitted That Met QC Criteria: March 7, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Liver Diseases; Genetic Diseases, Inborn; Subcutaneous Emphysema; Emphysema; Alpha 1-Antitrypsin Deficiency
• Other Study ID Numbers: VX22-634-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: 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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No