Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04167345 |
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Recruitment Status :
Terminated
(at Sponsor's discretion)
First Posted : November 18, 2019
Results First Posted : February 2, 2022
Last Update Posted : February 2, 2022
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Sponsor:
Vertex Pharmaceuticals Incorporated
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
- Study Details
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Brief Summary:
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Alpha 1-Antitrypsin Deficiency | Drug: VX-814 Drug: Placebo | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 48 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects |
| Actual Study Start Date : | January 13, 2020 |
| Actual Primary Completion Date : | November 14, 2020 |
| Actual Study Completion Date : | November 14, 2020 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Alpha-1 antitrypsin deficiency
MedlinePlus related topics:
Alpha-1 Antitrypsin Deficiency
| Arm | Intervention/treatment |
|---|---|
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Placebo Comparator: Parts A1, A2 and B Combined: Placebo
Participants received placebo matched to VX-814 in the treatment period for 28 days.
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Drug: Placebo
Placebo matched to VX-814 for oral administration. |
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Experimental: Part A1: VX-814 100 milligrams (mg)
Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days.
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Drug: VX-814
Tablet for oral administration. |
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Experimental: Part A1: VX-814 200 mg
Participants received VX-814 200 mg q12h in the treatment period for 28 days.
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Drug: VX-814
Tablet for oral administration. |
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Experimental: Parts A1 and A2 Combined: VX-814 400 mg
Participants received VX-814 400 mg q12h in the treatment period for 28 days.
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Drug: VX-814
Tablet for oral administration. |
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Experimental: Part B: VX-814 600 mg
Participants received VX-814 600 mg q12h in the treatment period for 28 days.
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Drug: VX-814
Tablet for oral administration. |
Primary Outcome Measures :
- 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 ]
Secondary Outcome Measures :
- 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 ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years to 80 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- Subjects must have a PiZZ genotype confirmed at screening
- Plasma AAT levels indicating severe deficiency at screening
Key Exclusion Criteria:
- 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04167345
Locations
Show 28 study locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Study Documents (Full-Text)
Documents provided by Vertex Pharmaceuticals Incorporated:
Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol [PDF] May 21, 2020
Statistical Analysis Plan [PDF] November 11, 2020
| Responsible Party: | Vertex Pharmaceuticals Incorporated |
| ClinicalTrials.gov Identifier: | NCT04167345 |
| Other Study ID Numbers: |
VX19-814-101 |
| First Posted: | November 18, 2019 Key Record Dates |
| Results First Posted: | February 2, 2022 |
| Last Update Posted: | February 2, 2022 |
| Last Verified: | January 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| 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 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency Liver Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases |
Genetic Diseases, Inborn Subcutaneous Emphysema Emphysema Pathologic Processes |