Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
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ClinicalTrials.gov Identifier: NCT04474197 |
Recruitment Status :
Completed
First Posted : July 16, 2020
Results First Posted : May 26, 2022
Last Update Posted : May 26, 2022
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Sponsor:
Vertex Pharmaceuticals Incorporated
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
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Study Type | Interventional |
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Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment |
Condition |
Alpha1-Antitrypsin Deficiency |
Interventions |
Drug: VX-864 Drug: Placebo |
Enrollment | 44 |
Participant Flow
Recruitment Details | |
Pre-assignment Details | This study was conducted in participants 18 through 80 of years of age, inclusive with the PiZZ genotype. |
Arm/Group Title | Placebo | VX-864 100 mg | VX-864 300 mg | VX-864 500 mg |
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Participants received placebo matched to VX-864 in the treatment period for 28 days. | Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days. | Participants received VX-864 300 mg q12h in the treatment period for 28 days. | Participants received VX-864 500 mg q12h in the treatment period for 28 days. |
Period Title: Overall Study | ||||
Started | 7 | 10 | 9 | 18 |
Completed | 7 | 9 | 9 | 18 |
Not Completed | 0 | 1 | 0 | 0 |
Reason Not Completed | ||||
Withdrawal of consent (not due to adverse event) | 0 | 1 | 0 | 0 |
Baseline Characteristics
Arm/Group Title | Placebo | VX-864 100 mg | VX-864 300 mg | VX-864 500 mg | Total | |
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Participants received placebo matched to VX-864 in the treatment period for 28 days. | Participants received VX-864 100 mg q12h in the treatment period for 28 days. | Participants received VX-864 300 mg q12h in the treatment period for 28 days. | Participants received VX-864 500 mg q12h in the treatment period for 28 days. | Total of all reporting groups | |
Overall Number of Baseline Participants | 7 | 10 | 9 | 18 | 44 | |
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[Not Specified]
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 7 participants | 10 participants | 9 participants | 18 participants | 44 participants | |
63.4 (10.5) | 55.1 (5.3) | 53.2 (16.2) | 57.4 (9.9) | 57.0 (10.9) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 7 participants | 10 participants | 9 participants | 18 participants | 44 participants | |
Female |
5 71.4%
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6 60.0%
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6 66.7%
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14 77.8%
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31 70.5%
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Male |
2 28.6%
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4 40.0%
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3 33.3%
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4 22.2%
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13 29.5%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 7 participants | 10 participants | 9 participants | 18 participants | 44 participants | |
Hispanic or Latino |
0 0.0%
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0 0.0%
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0 0.0%
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1 5.6%
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1 2.3%
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Not Hispanic or Latino |
7 100.0%
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8 80.0%
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8 88.9%
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15 83.3%
|
38 86.4%
|
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Unknown or Not Reported |
0 0.0%
|
2 20.0%
|
1 11.1%
|
2 11.1%
|
5 11.4%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 7 participants | 10 participants | 9 participants | 18 participants | 44 participants | |
American Indian or Alaska Native |
0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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Asian |
0 0.0%
|
0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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Black or African American |
0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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White |
7 100.0%
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10 100.0%
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9 100.0%
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18 100.0%
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44 100.0%
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More than one race |
0 0.0%
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0 0.0%
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0 0.0%
|
0 0.0%
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0 0.0%
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Unknown or Not Reported |
0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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Plasma Functional Alpha-1 Antitrypsin (AAT) Levels
Mean (Standard Deviation) Unit of measure: Micromole per liter |
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Number Analyzed | 7 participants | 10 participants | 9 participants | 18 participants | 44 participants | |
4.7 (1.3) | 4.0 (0.7) | 3.8 (0.9) | 4.1 (0.6) | 4.1 (0.9) |
Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Results Point of Contact
Name/Title: | Medical Monitor |
Organization: | Vertex Pharmaceuticals Incorporated |
Phone: | 617-341-6777 |
EMail: | medicalinfo@vrtx.com |
Responsible Party: | Vertex Pharmaceuticals Incorporated |
ClinicalTrials.gov Identifier: | NCT04474197 |
Other Study ID Numbers: |
VX19-864-101 2019-004881-16 ( EudraCT Number ) |
First Submitted: | July 13, 2020 |
First Posted: | July 16, 2020 |
Results First Submitted: | May 3, 2022 |
Results First Posted: | May 26, 2022 |
Last Update Posted: | May 26, 2022 |