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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-004885-21 | EudraCT Number |
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The purpose of this study is to evaluate the safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in participants with CF who are 12 years of age or older.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Experimental | Participants received ELX 200 mg once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELX/TEZ/IVA | Drug | Fixed-dose combination tablets for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 36 |
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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 |
|---|---|---|---|---|---|---|
| The Prince Charles Hospital | Chermside | Australia | ||||
| Alfred Hospital |
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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Participants from Parent Studies VX19-445-117 (NCT04599465) and VX20-445-126 (NCT04969224) were enrolled in this study. A total of 86 participants were enrolled in this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Participants received elexacaftor (ELX) 200 mg once daily (qd)/tezacaftor (TEZ) 100 mg qd/ivacaftor (IVA) 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks. |
| 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 | Feb 14, 2022 | Jun 19, 2023 |
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| IVA | Drug | Tablets for oral administration. |
|
|
| Melbourne |
| Australia |
| Institute for Respiratory Health | Nedlands | Australia |
| Telethon Kids Institute | Nedlands | Australia |
| The Royal Children's Hospital | Parkville | Australia |
| Mater Adult Hospital | South Brisbane | Australia |
| Queensland Children's Hospital | South Brisbane | Australia |
| Cliniques Universitaires de Bruxelles Hopital Erasme | Brussels | Belgium |
| Universitair Ziekenhuis Brussel - Campus Jette | Brussels | Belgium |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| University of Calgary Medical Clinic of the Foothills Medical Centre | Calgary | Canada |
| Stollery Children's Hospital | Edmonton | Canada |
| Queen Elizabeth II Health Sciences Center | Halifax | Canada |
| Klinika Nemoci Plicnich a Tuberkulozy | Brno | Czechia |
| Fakultni nemocnice v Motole | Prague | Czechia |
| Hospital Saint Joan de Deu | Barcelona | Spain |
| Hospital Universitari Vall d Hebron | Barcelona | Spain |
| Hospital Universitari Vall d´Hebron Servicio de Broncoscopia | Barcelona | Spain |
| Hospital Universitario 12 de Octubre | Madrid | Spain |
| Hospital Universitario Ramon y Cajal | Madrid | Spain |
| Hospital Virgen de la Arrixaca | Murcia | Spain |
| Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari | Sabadell | Spain |
| Hospital Universitario Virgen del Rocio | Seville | Spain |
| COMPLETED |
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| NOT COMPLETED |
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Baseline data is based on the parent study baseline, which is defined as the most recent non-missing measurement collected before the first dose of study drug in the treatment period of parent studies. Baseline data is presented for participants who received at least 1 dose of study drug in this study
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 36 weeks. |
| 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 |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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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 | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Safety set included for all participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Day 1 up to Week 36 |
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|
|
Day 1 up to Week 36
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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 | ELX/TEZ/IVA | Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 36 weeks. | 0 | 86 | 4 | 86 | 33 | 86 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastroenteritis viral | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Malnutrition | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | 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 | Dec 12, 2022 | Jun 19, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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| ID | Term |
|---|---|
| C000706587 | elexacaftor, ivacaftor, tezacaftor drug combination |
| C545203 | ivacaftor |
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| Asian |
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| American Indian or Alaska Native |
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| Native Hawaiian or other Pacific Islander |
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| Other |
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| Not collected per local regulations |
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| Not collected per local regulations |
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