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This is a Phase 3, multicenter, open-label and roll-over study in participants who are 12 to <24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LUM/IVA | Experimental | Participants weighing 7 to less than (<) 9 kilograms (kg) received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing greater than or equal to (>=)14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LUM/IVA | Drug | LUM/IVA granules for oral administration |
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| 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 120 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | From Baseline at Week 96 |
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Key Inclusion Criteria:
Participants From Study VX16-809-122 Part B (Study 122)
Participants Not From Study 122
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| Arkansas Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37983082 | Derived | Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4. | |
| 33331662 | Derived |
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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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The study was conducted in participants with cystic fibrosis (CF) aged 12 months through less than (<) 24 months of age at treatment initiation who were homozygous for F508del and participants who completed the 24-week treatment period along with the safety follow-up in study VX16-809-122 (NCT03601637) Part B.
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| ID | Title | Description |
|---|---|---|
| FG000 | LUM/IVA | Participants weighing 7 to less than (<) 9 kilograms (kg) received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing greater than or equal to (>=)14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 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 12, 2021 | Jul 25, 2024 |
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| Little Rock |
| Arkansas |
| 72202 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Yale New Haven Hospital | New Haven | Connecticut | 06510 | United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Riley Hospital for Children at Indiana University Health | Indianapolis | Indiana | 46202 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| The Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Cardinal Glennon Children's Hospital - St. Louis University | St Louis | Missouri | 63104 | United States |
| University of Rochester Medical Center | Rochester | New York | 14642 | United States |
| NC TraCS Institute - CTRC University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Wake Forest University Baptist Medical Center | Winston-Salem | North Carolina | 27157 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Children's Medical Center of Dallas | Dallas | Texas | 75235 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| University of Utah / Primary Children's Medical Center | Salt Lake City | Utah | 84132 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| University of Wisconsin Hospital and Clinics | Madison | Wisconsin | 53792 | United States |
| McGill University Health Centre, Glen Site, Montreal Children's Hospital | Montreal | Canada |
| The Hospital for Sick Children | Toronto | Canada |
| British Columbia's Children's Hospital | Vancouver | Canada |
| Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3. |
| Rollover Participants |
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| Lumacaftor (LUM)/ Ivacaftor (IVA)-Naïve Participants |
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| COMPLETED |
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| NOT COMPLETED |
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Baseline value was defined as the most recent non-missing measurement (scheduled or unscheduled) collected on or before the first dose of LUM/IVA in this study.
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| ID | Title | Description |
|---|---|---|
| BG000 | LUM/IVA | Participants weighing 7 to <9 kg received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants >=14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 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 | months |
| ||||||||||||||||||||||
| 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 | No |
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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 all participants who are exposed to any amount of study drug in this study. | Posted | Count of Participants | Participants | No | Day 1 up to Week 120 |
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| Secondary | Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | The Full Analysis Set (FAS) included all participants who were enrolled and dosed in Study 124. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | Mean | Standard Deviation | millimole per liter (mmol/L) | From Baseline at Week 96 |
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|
Day 1 to Week 120
Safety set included all participants who are exposed to any amount 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 | LUM/IVA | Participants weighing 7 to < 9 kg at Day 1 received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to <14 kg at Day 1 received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing >=14 kg at Day 1 received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks. | 0 | 52 | 12 | 52 | 50 | 52 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Anaphylactic reaction | Immune system disorders | MedDRA 26.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Cellulitis orbital | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Lower respiratory tract infection viral | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Oral herpes | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Parainfluenzae virus infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Near drowning | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
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| Procedural pain | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
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| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 26.0 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Croup infectious | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Molluscum contagiosum | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Otitis media | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Otitis media acute | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Respiratory syncytial virus infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Viral upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Skin laceration | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 26.0 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA 26.0 | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA 26.0 | Systematic Assessment |
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| Pseudomonas test positive | Investigations | MedDRA 26.0 | Systematic Assessment |
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| SARS-CoV-2 test positive | Investigations | MedDRA 26.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 26.0 | 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 | Aug 3, 2023 | Jul 25, 2024 | 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 |
|---|---|
| C000599212 | lumacaftor, ivacaftor drug combination |
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| Not collected per local regulations |
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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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| Not collected per local regulations |
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| Other |
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| More than one race |
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