Alpha 1-Antitrypsin DeficiencyPhase 2NCT04167345
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
A Phase 2 study testing VX-814 in people with Alpha 1-Antitrypsin Deficiency, run by Vertex Pharmaceuticals Incorporated at 28 sites in 4 countries.
Trial summary
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency
- What is being tested?
- VX-814
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- 28 sitesin 4 countries
- Study dates
- 13 Jan 2020to 14 Nov 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects must have a PiZZ genotype confirmed at screening
- Plasma AAT levels indicating severe deficiency at screening
Who cannot join
- History of a medical condition that could negatively impact the ability to complete the study
- Solid organ, or hematological transplantation or is currently on a transplant list
- History of use of gene therapy or RNAi therapy at any time previously
- Other protocol defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
28 sites in 4 countries.
28 sites in 4 countries
United States22 sites
University of Alabama at Birmingham
Birmingham, Alabama, 35233
University of North Carolina Medical Center
Chapel Hill, North Carolina, 27514
Medical University of South Carolina
Charleston, South Carolina, 29425
University of Cincinnati
Cincinnati, Ohio, 45267
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
National Jewish Health
Denver, Colorado, 80206
Inova Fairfax Medical Campus
Falls Church, Virginia, 22042
University of Florida, Shands Hospital
Gainesville, Florida, 32610
Blessing Corporate Services, Inc., dba Blessing Health System
Hannibal, Missouri, 63401
Renovatio Clinical
Houston, Texas, 77380
The University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Miami Miller School of Medicine
Miami, Florida, 33136
University of Minnesota
Minneapolis, Minnesota, 55455
Columbia University Medical Center
New York, New York, 10032
Central Florida Pulmonary Group, PA
Orlando, Florida, 32803
Temple University Hospital
Philadelphia, Pennsylvania, 19140
Oregon Health & Science University
Portland, Oregon, 97239
University of California Davis Medical Center
Sacramento, California, 95817
University of Utah Health
Salt Lake City, Utah, 84108
The University of Texas Health Science Center at Tyler
Tyler, Texas, 75708
Wake Forest University Baptist Medical Center
Winston-Salem, North Carolina, 27157
Germany3 sites
University Hospital RWTH Aachen
Aachen
Universitätsklinikum Essen
Essen
Medizinische Hochschule Hannover
Hanover
Canada2 sites
Queen Elizabeth II Health Sciences Center
Halifax
Inspiration Research Ltd
Toronto
Ireland1 site
Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital
Beaumont
About this study
Official title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Being tested
- VX-814, 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-814Drug
Tablet for oral administration.
PlaceboDrug
Placebo matched to VX-814 for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Parts A1, A2 and B Combined: Placebo | Placebo comparator | Participants received placebo matched to VX-814 in the treatment period for 28 days. Receives: Drug: Placebo |
| Part A1: VX-814 100 milligrams (mg) | Experimental | Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days. Receives: Drug: VX-814 |
| Part A1: VX-814 200 mg | Experimental | Participants received VX-814 200 mg q12h in the treatment period for 28 days. Receives: Drug: VX-814 |
| Parts A1 and A2 Combined: VX-814 400 mg | Experimental | Participants received VX-814 400 mg q12h in the treatment period for 28 days. Receives: Drug: VX-814 |
| Part B: VX-814 600 mg | Experimental | Participants received VX-814 600 mg q12h in the treatment period for 28 days. Receives: Drug: VX-814 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
Measured: From Baseline at Day 28
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Week 8
Secondary outcomes (2)
Change in Plasma Antigenic AAT Levels
Measured: From Baseline at Day 28
Observed Pre-dose Plasma Concentration of VX-814
Measured: Pre-dose at Day 7, Day 14, Day 21, and Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2019
- Study start13 Jan 2020
- Primary completion14 Nov 2020
- Study completion14 Nov 2020
Why it stopped: at Sponsor's discretion
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 48 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
- NCT04167345
- Sponsor's protocol ID
- VX19-814-101
- Status last verified
- Jan 2022
- First submitted
- 15 Nov 2019
- Last updated
- 2 Feb 2022
- Results posted
- Yes, on ClinicalTrials.gov
- 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 2 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects
- ClinicalTrials.gov ID
- NCT04167345
- Acronym / sponsor ID
- VX19-814-101
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: at Sponsor's discretion
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 13 Jan 2020 (actual)
- Primary completion
- 14 Nov 2020 (actual)
- Study completion
- 14 Nov 2020 (actual)
- First posted
- 18 Nov 2019 (actual)
- Results
- Posted 2 Feb 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Parts A1, A2 and B Combined: Placebo | Placebo comparator | Drug: PlaceboParticipants received placebo matched to VX-814 in the treatment period for 28 days. |
| Part A1: VX-814 100 milligrams (mg) | Experimental | Drug: VX-814Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days. |
| Part A1: VX-814 200 mg | Experimental | Drug: VX-814Participants received VX-814 200 mg q12h in the treatment period for 28 days. |
| Parts A1 and A2 Combined: VX-814 400 mg | Experimental | Drug: VX-814Participants received VX-814 400 mg q12h in the treatment period for 28 days. |
| Part B: VX-814 600 mg | Experimental | Drug: VX-814Participants received VX-814 600 mg q12h in the treatment period for 28 days. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Subjects must have a PiZZ genotype confirmed at screening
- Plasma AAT levels indicating severe deficiency at screening
Key exclusion criteria (4 listed)
- History of a medical condition that could negatively impact the ability to complete the study
- Solid organ, or hematological transplantation or is currently on a transplant list
- History of use of gene therapy or RNAi therapy at any time previously
- 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-814Tablet for oral administration. | Drug | Part A1: VX-814 100 milligrams (mg), Part A1: VX-814 200 mg, Part B: VX-814 600 mg, Parts A1 and A2 Combined: VX-814 400 mg |
| PlaceboPlacebo matched to VX-814 for oral administration. | Drug | Parts A1, A2 and B Combined: Placebo |
Primary outcome
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
Time frame: From Baseline at Day 28
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 8
2 secondary outcomes
Change in Plasma Antigenic AAT Levels
Time frame: From Baseline at Day 28
Observed Pre-dose Plasma Concentration of VX-814
Time frame: Pre-dose at Day 7, Day 14, Day 21, and Day 28
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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
Related trials for Alpha 1-Antitrypsin Deficiency
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 1/2
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
Alpha-1 Antitrypsin Deficiency (AATD)
RecruitingPhase 4
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
Alpha 1-Antitrypsin, COPD
Enrolling by invitationObservational study
AATD-Liver Scan Study
Alpha-1 Antitrypsin Deficiency, AATD
RecruitingPhase 3
A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)
Alpha 1 Antitrypsin Deficiency
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.
28 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VX-814 in people with Alpha 1-Antitrypsin Deficiency, run by Vertex Pharmaceuticals Incorporated at 28 sites in 4 countries.
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 2 inclusion and 4 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: stopped early, as of the sponsor's last update on 2 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Germany, Canada, Ireland. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels (time frame: From Baseline at Day 28). The study lists 2 primary outcomes in all. 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 2 Feb 2022.