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

NCT05579431 | Completed Phase 1 FDA Drug

• 1 other identifier: VX22-634-001
• Study Type: interventional
• Target: 127
• Locations: 1 country
• Sites: 5

Brief Summary

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

Conditions

Alpha 1-Antitrypsin Deficiency

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• 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

Study Arms (4)

Part A

EXPERIMENTAL

Participants grouped in different cohorts will receive a single ascending dose of VX-634.

Drug: VX-634

Placebo Part A

PLACEBO COMPARATOR

Participants will be randomized to receive placebo matched to VX-634.

Drug: Placebo

Part B

EXPERIMENTAL

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

Placebo Part B

PLACEBO COMPARATOR

Participants will be randomized to receive placebo matched to VX-634.

Drug: Placebo

Interventions

VX-634 DRUG

Suspension for oral administration.

Part A; Part B

Placebo DRUG

Suspension for oral administration.

Placebo Part A; Placebo Part B

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• Any condition possibly affecting drug absorption

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (5)

Celerion - Tempe

Tempe, Arizona, 85283, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Central Florida Pulmonary Group, P.A.

Orlando, Florida, 32803, United States

Location

ICON Lenexa

Lenexa, Kansas, 66219, United States

Location

ICON Salt Lake City

Salt Lake City, Utah, 84124, United States

Location

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2022
• First Posted: October 13, 2022
• Study Start: October 13, 2022
• Primary Completion: May 24, 2023
• Study Completion: November 13, 2023
• Last Updated: March 12, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)