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

Alpha 1-Antitrypsin Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05579431
• Other study ID #: VX22-634-001
• Status: Completed
• Phase: Phase 1
• Start date: October 13, 2022
• Est. completion date: November 13, 2023

Study information

• Verified date: March 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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 information / eligibility

• Status: Completed
• Enrollment: 127
• Est. completion date: November 13, 2023
• Est. primary completion date: May 24, 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency

Intervention

Drug:
• VX-634
Suspension for oral administration.
• Placebo
Suspension for oral administration.

Locations

Country	Name	City	State
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	ICON Salt Lake City	Salt Lake City	Utah
United States	Celerion - Tempe	Tempe	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-634		Day 1 up to Day 20
Secondary	Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-634		Day 1 up to Day 20
Secondary	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