Alpha 1-Antitrypsin DeficiencyPhase 1NCT05579431
A Phase 1, First-in-human Study of VX-634
A Phase 1 study testing VX-634 in people with Alpha 1-Antitrypsin Deficiency, run by Vertex Pharmaceuticals Incorporated at 5 sites in United States.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-634 at various doses. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency
- What is being tested?
- VX-634
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 127 participants
- Where
- 5 sitesUnited States
- Study dates
- 13 Oct 2022to 13 Nov 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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
Who cannot join
- Any condition possibly affecting drug absorption
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
University of Florida
Gainesville, Florida, 32610
ICON Lenexa
Lenexa, Kansas, 66219
Central Florida Pulmonary Group, P.A.
Orlando, Florida, 32803
ICON Salt Lake City
Salt Lake City, Utah, 84124
Celerion - Tempe
Tempe, Arizona, 85283
About this study
Official title: 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.
Read the 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.).
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Being tested
- VX-634, Placebo
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-634Drug
Suspension for oral administration.
PlaceboDrug
Suspension for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Part A | Experimental | Participants grouped in different cohorts will receive a single ascending dose of VX-634. Receives: Drug: VX-634 |
| Placebo Part A | Placebo comparator | Participants will be randomized to receive placebo matched to VX-634. Receives: 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. Receives: Drug: VX-634 |
| Placebo Part B | Placebo comparator | Participants will be randomized to receive placebo matched to VX-634. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Day 20
Secondary outcomes (3)
Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-634
Measured: Day 1 up to Day 20
Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-634
Measured: Day 1 up to Day 20
Part A and B: Urine Concentration of VX-634
Measured: Part A Cohort A3: Days 1 and 5; Part B Cohort B3: Days 1, 10 and 11
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2022
- Study start13 Oct 2022
- Primary completion24 May 2023
- Study completion13 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha 1-Antitrypsin DeficiencyStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 127 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05579431
- Sponsor's protocol ID
- VX22-634-001
- Status last verified
- Mar 2024
- First submitted
- 10 Oct 2022
- Last updated
- 12 Mar 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, First-in-human Study of Safety, Tolerability, and Pharmacokinetics of VX-634
- ClinicalTrials.gov ID
- NCT05579431
- Acronym / sponsor ID
- VX22-634-001
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 127 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 13 Oct 2022 (actual)
- Primary completion
- 24 May 2023 (actual)
- Study completion
- 13 Nov 2023 (actual)
- First posted
- 13 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part A | Experimental | Drug: VX-634Participants grouped in different cohorts will receive a single ascending dose of VX-634. |
| Placebo Part A | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive placebo matched to VX-634. |
| Part B | Experimental | Drug: VX-634Participants grouped in different cohorts will receive multiple doses of VX-634.The dose levels will be determined based on the data from Part A. |
| Placebo Part B | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive placebo matched to VX-634. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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 (2 listed)
- Any condition possibly affecting drug absorption
- Other protocol defined Inclusion/Exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VX-634Suspension for oral administration. | Drug | Part A, Part B |
| PlaceboSuspension for oral administration. | Drug | Placebo Part A, Placebo Part B |
Primary outcome
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
3 secondary outcomes
Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-634
Time frame: Day 1 up to Day 20
Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-634
Time frame: Day 1 up to Day 20
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2024.
- Individual participant dataSharing planned: No. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VX-634 in people with Alpha 1-Antitrypsin Deficiency, run by Vertex Pharmaceuticals Incorporated at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Mar 2024.