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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-003554-86 | EudraCT Number |
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This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks. |
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| ELX/TEZ/IVA | Experimental | Participants weighing less than (<) 30 kilograms (kg) at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) and participants weighing greater than equals to (>=) 30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELX/TEZ/IVA | Drug | Fixed-dose combination tablet for oral administration qd in the morning. |
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| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Lung Clearance Index 2.5 (LCI2.5) | The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry. | From Baseline Through Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | From Baseline Through Week 24 |
| Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
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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 |
|---|---|---|---|---|---|---|
| Telethon Kids Institute | Nedlands | Australia | ||||
| Queensland Children's 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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This study was conducted in cystic fibrosis (CF) participants aged 6 through 11 years of age.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks. |
| FG001 | ELX/TEZ/IVA | Participants weighing less than (<) 30 kilograms (kg) at screening received ELX 100 mg once daily (qd)/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) and participants weighing greater than equals to (>=) 30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 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 | Jul 29, 2020 | May 16, 2022 |
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| IVA | Drug | Tablet for oral administration qd in the evening. |
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| Placebo (matched to ELX/TEZ/IVA) | Other | Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning. |
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| Placebo (matched to IVA) | Other | Placebo matched to IVA for oral administration qd in the evening. |
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| Day 1 up to Week 28 |
| South Brisbane |
| Australia |
| The Children's Hospital at Westmead | Westmead | Australia |
| McGill University Health Centre, Glen Site, Montreal Children's Hospital | Montreal | Canada |
| The Hospital for Sick Children | Toronto | Canada |
| British Columbia Children's Hospital | Vancouver | Canada |
| Juliane Marie Center, Rigshospitalet | Copenhagen | Denmark |
| Groupe Hospitaler Pellegrin, CHU De Bordeaux | Bordeaux | France |
| CHU Lyon - Hopital Femme Mere-Enfant | Bron | France |
| Hopital Necker, Enfants Malades | Paris | France |
| Hopital Robert Debre | Paris | France |
| Centre de Perharidy | Roscoff | France |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Universitaetsklinkum Koeln, CF-Studienzentrum | Cologne | Germany |
| Universitätsklinikum Essen | Essen | Germany |
| Johann Wolfgang Goethe University | Frankfurt | Germany |
| Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin | Giessen | Germany |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin | Heidelberg | Germany |
| Hadassah University Hospital Mount Scopus | Jerusalem | Israel |
| Schneider Children's Medical Center | Petah Tikva | Israel |
| Universitair Medisch Centrum Groningen | Groningen | Netherlands |
| Erasmus Medical Center / Sophia Children's Hospital | Rotterdam | Netherlands |
| Hospital Universitari Vall d Hebron | Barcelona | Spain |
| Hospital Virgen de la Arrixaca | Murcia | Spain |
| Inselspital - Universitaetsspital Bern | Bern | Switzerland |
| Kinderspital Zuerich | Zurich | Switzerland |
| University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital | Bristol | United Kingdom |
| Children's Hospital of Wales | Cardiff | United Kingdom |
| Royal Hospital for Sick Children | Edinburgh | United Kingdom |
| Alder Hey Children's NHS Foundation Trust | Liverpool | United Kingdom |
| Great Ormond Street Hospital for Sick Children | London | United Kingdom |
| Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital | London | United Kingdom |
| Southampton General Hospital | Southampton | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks. |
| BG001 | ELX/TEZ/IVA | Participants weighing <30 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
| 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 |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Lung Clearance Index2.5 (LCI2.5) | The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry. | Mean | Standard Deviation | index |
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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 | Absolute Change in Lung Clearance Index 2.5 (LCI2.5) | The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry. | Full analysis set (FAS) included all randomized participants who carry the intended CFTR allele mutation and receive at least 1 dose of study drug. | Posted | Least Squares Mean | Standard Error | index | From Baseline Through Week 24 |
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| Secondary | Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | FAS. | Posted | Least Squares Mean | Standard Error | millimole per liter (mmol/L) | From Baseline Through Week 24 |
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| Secondary | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Safety set included all participants who received at least 1 dose of study drug in the treatment period. | Posted | Number | participants | Day 1 up to Week 28 |
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Day 1 up to Week 28
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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 | Placebo | Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks. | 0 | 61 | 9 | 61 | 53 | 61 |
| EG001 | ELX/TEZ/IVA | Participants weighing <30 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. | 0 | 60 | 4 | 60 | 46 | 60 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
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| Phimosis | Congenital, familial and genetic disorders | MedDRA 24.0 | Systematic Assessment |
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| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Intussusception | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| General physical health deterioration | General disorders | MedDRA 24.0 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Pneumonia pseudomonal | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Varicella zoster virus infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Bacterial test positive | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Steatorrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 24.0 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Bacterial test positive | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Forced expiratory volume decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Staphylococcus test positive | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0 | 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 | May 23, 2021 | May 16, 2022 | SAP_001.pdf |
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| 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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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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