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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-002746-40 | EudraCT Number |
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Study drug dosing was permanently discontinued at the Sponsor's discretion based on frequency and nature of skin and subcutaneous tissue disorders.
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The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group A | Experimental | Participants received VX-864 every 12 hours (q12h) for 48 weeks or until study drug dosing was terminated. |
|
| Group B | Experimental | Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated, and undergo a second liver biopsy at either Week 24 or Week 48. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-864 | Drug | Tablets for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels | From Baseline at Week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Blood Functional AAT Levels | From Baseline up to Week 48 | |
| Change in Blood Antigenic AAT Levels | From Baseline up to Week 48 | |
| Change in Blood Z-polymer Levels |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32610 | United States | ||
| Central Florida Pulmonary Group, P.A. |
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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A total of 14 participants were enrolled from 23 February 2023 to 03 November 2023 in this study. Study drug dosing and efficacy assessments were terminated early due to Sponsor decision, therefore evaluation of efficacy outcome measure was not completed.
This study had 2 Groups: Group A participants did not have a liver biopsy and Group B participants have 2 liver biopsies performed over the course of the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Group A VX-864 500 mg | Participants received VX-864 every 12 hours (q12h) for 48 weeks or until study drug dosing was terminated. |
| FG001 | Group B VX-864 500 mg | Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 2, 2023 | Aug 11, 2025 |
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| From Baseline up to Week 48 |
| Group B: Change in Z-polymer Accumulation in the Liver | From Baseline up to Week 48 |
| Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 52 |
| Orlando |
| Florida |
| 32803 |
| United States |
| The University of Iowa Hospitals and Clinics: Adult Pulmonary Clinic | Iowa City | Iowa | 52242 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Hannibal Regional Healthcare System | Hannibal | Missouri | 63401 | United States |
| Columbia University Irving Medical Center | New York | New York | 10032 | United States |
| Marsico Clinical Research Center at UNC Pulmonary Clinic | Chapel Hill | North Carolina | 27517 | United States |
| Renovatio Clinical | Houston | Texas | 77380 | United States |
| University of Utah Health | Salt Lake City | Utah | 84108 | United States |
| Inova Fairfax Medical Campus | Falls Church | Virginia | 22042 | United States |
| University Hospital RWTH Aachen | Aachen | Germany |
| Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital | Beaumont | Ireland |
| King's College Hospital | London | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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The Full Analysis Set (FAS) is defined as all enrolled participants who have received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Group A VX-864 500 mg | Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated. |
| BG001 | Group B VX-864 500 mg | Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants | No |
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| Race/Ethnicity, Customized | Count of Participants | Participants | No |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels | Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48. | Posted | From Baseline at Week 48 |
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| Secondary | Change in Blood Functional AAT Levels | Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48. | Posted | From Baseline up to Week 48 |
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| Secondary | Change in Blood Antigenic AAT Levels | Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48. | Posted | From Baseline up to Week 48 |
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| |||||||||||||||||||||||
| Secondary | Change in Blood Z-polymer Levels | Data was not collected for this Outcome Measure as the study drug dosing was terminated prior to any participant reaching Week 48. | Posted | From Baseline up to Week 48 |
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| |||||||||||||||||||||||
| Secondary | Group B: Change in Z-polymer Accumulation in the Liver | No participants in Group B underwent a second liver biopsy at week 24 or week 48 as the study drug dosing was terminated prior to any participant reaching week 24 or week 48. Therefore, data was not collected for this Outcome Measure. | Posted | From Baseline up to Week 48 |
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| Secondary | Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Safety set included all participants who had received at least 1 dose of study drug in this study. | Posted | Count of Participants | Participants | Day 1 up to Week 52 |
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Day 1 up to Week 52
Safety set included all participants who had received at least 1 dose of study drug in this study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Group A VX-864 500 mg | Participants received VX-864 q12h for 48 weeks or until study drug dosing was terminated. | 0 | 10 | 1 | 10 | 10 | 10 |
| EG001 | Group B VX-864 500 mg | Participants undergo a liver biopsy before receiving VX-864 q12h for 48 weeks or until study drug dosing was terminated and undergo a second liver biopsy at either Week 24 or Week 48. | 0 | 4 | 0 | 4 | 4 | 4 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diverticulitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Porphyria non-acute | Congenital, familial and genetic disorders | MedDRA 27.0 | Systematic Assessment |
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| Abdominal distension | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Mouth ulceration | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Paraesthesia oral | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Diverticulitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
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| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Dysgeusia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Hyperaesthesia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Irritability | Psychiatric disorders | MedDRA 27.0 | Systematic Assessment |
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| Benign prostatic hyperplasia | Reproductive system and breast disorders | MedDRA 27.0 | Systematic Assessment |
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| Blister | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Dyshidrotic eczema | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Photosensitivity reaction | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Post inflammatory pigmentation change | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Pseudoporphyria | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 30, 2023 | Aug 11, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| Male |
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