Back to resultsA Phase 1, First-in-human Study of VX-634
Primary Purpose
Alpha 1-Antitrypsin Deficiency
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
VX-634
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Alpha 1-Antitrypsin Deficiency
Eligibility Criteria
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.
Sites / Locations
- Celerion - TempeRecruiting
- University of FloridaRecruiting
- Central Florida Pulmonary Group, P.A.Recruiting
- ICON LenexaRecruiting
- ICON Salt Lake CityRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Placebo Comparator
Experimental
Placebo Comparator
Arm Label
Part A
Placebo Part A
Part B
Placebo Part B
Arm Description
Participants grouped in different cohorts will receive a single ascending dose of VX-634.
Participants will be randomized to receive placebo matched to VX-634.
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.
Participants will be randomized to receive placebo matched to VX-634.
Outcomes
Primary Outcome Measures
Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Secondary Outcome Measures
Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-634
Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-634
Part A and B: Urine Concentration of VX-634
Full Information
NCT ID
NCT05579431
First Posted
October 10, 2022
Last Updated
September 28, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
1. Study Identification
Unique Protocol Identification Number
NCT05579431
Brief Title
A Phase 1, First-in-human Study of VX-634
Official Title
A Phase 1, First-in-human Study of Safety, Tolerability, and Pharmacokinetics of VX-634
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 13, 2022 (Actual)
Primary Completion Date
May 24, 2023 (Actual)
Study Completion Date
June 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-634 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.).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alpha 1-Antitrypsin Deficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
114 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Part A
Arm Type
Experimental
Arm Description
Participants grouped in different cohorts will receive a single ascending dose of VX-634.
Arm Title
Placebo Part A
Arm Type
Placebo Comparator
Arm Description
Participants will be randomized to receive placebo matched to VX-634.
Arm Title
Part B
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo Part B
Arm Type
Placebo Comparator
Arm Description
Participants will be randomized to receive placebo matched to VX-634.
Intervention Type
Drug
Intervention Name(s)
VX-634
Intervention Description
Suspension for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Suspension for oral administration.
Primary Outcome Measure Information:
Title
Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
Day 1 up to Day 20
Secondary Outcome Measure Information:
Title
Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-634
Time Frame
Day 1 up to Day 20
Title
Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-634
Time Frame
Day 1 up to Day 20
Title
Part A and B: Urine Concentration of VX-634
Time Frame
Part A Cohort A3: Days 1 and 5; Part B Cohort B3: Days 1, 10 and 11
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
617-341-6777
Email
medicalinfo@vrtx.com
Facility Information:
Facility Name
Celerion - Tempe
City
Tempe
State/Province
Arizona
ZIP/Postal Code
85283
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Individual Site Status
Recruiting
Facility Name
Central Florida Pulmonary Group, P.A.
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Individual Site Status
Recruiting
Facility Name
ICON Lenexa
City
Lenexa
State/Province
Kansas
ZIP/Postal Code
66219
Country
United States
Individual Site Status
Recruiting
Facility Name
ICON Salt Lake City
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84124
Country
United States
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
IPD Sharing 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
Learn more about this trial
A Phase 1, First-in-human Study of VX-634
We'll reach out to this number within 24 hrs