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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-503081-74-00 | Other Identifier | EU CT |
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The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VNZ/TEZ/D-IVA | Experimental | Part A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study. Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VNZ/TEZ/D-IVA | Drug | Fixed-dose combination tablets or granules for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) | From Baseline up to Week 100 | |
| Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) | From Baseline up to Week 196 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 96 | |
| Part B: Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 192 | |
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Key Inclusion Criteria:
Parts A and B:
• Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)
Part B:
-Meets at least 1 of the following criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Orange County | Orange | California | 92868 | United States | ||
| Stanford University - Palo Alto - Pulmonology |
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
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| Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) |
| From Baseline Through Week 100 |
| Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) | From Baseline Through Week 196 |
| Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx) | From Baseline Through Week 100 |
| Part B: Number of Pulmonary Exacerbation (PEx) | From Baseline Through Week 196 |
| Part A (All Cohorts): Number of CF- Related Hospitalizations | From Baseline Through Week 100 |
| Part B: Number of CF- Related Hospitalizations | From Baseline Through Week 196 |
| Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score | From Baseline Through Week 100 |
| Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score | From Baseline Through Week 196 |
| Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI) | From Baseline Through Week 100 |
| Part B: Absolute Change in Body Mass Index (BMI) | From Baseline Through Week 196 |
| Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score | From Baseline Through Week 100 |
| Part B: Absolute Change in BMI-for-age Z-score | From Baseline Through Week 196 |
| Part A (Cohort 3): Absolute Change in Weight-for-length | From Baseline Through Week 100 |
| Part B: Absolute Change in Weight-for-length | From Baseline Through Week 196 |
| Part A (Cohort 3): Absolute Change in Weight-for-length Z-score | From Baseline Through Week 100 |
| Part B: Absolute Change in Weight-for-length Z-score | From Baseline Through Week 196 |
| Part A (All Cohorts): Absolute Change in Weight | From Baseline Through Week 100 |
| Part B: Absolute Change in Weight | From Baseline Through Week 196 |
| Part A (All Cohorts): Change in Weight-for-age Z-score | From Baseline Through Week 100 |
| Part B: Change in Weight-for-age Z-score | From Baseline Through Week 196 |
| Part A (Cohorts 1 and 2): Absolute Change in Height | From Baseline Through Week 100 |
| Part B: Absolute Change in Height | From Baseline Through Week 196 |
| Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score | From Baseline Through Week 100 |
| Part B: Absolute Change in Height-for-age Z-score | From Baseline Through Week 196 |
| Part A (Cohort 3): Absolute Change in Length | From Baseline Through Week 100 |
| Part B: Absolute Change in Length | From Baseline Through Week 196 |
| Part A (Cohort 3): Absolute Change in Length-for-age Z-score | From Baseline Through Week 100 |
| Part B: Absolute Change in Length-for-age Z-score | From Baseline Through Week 196 |
| Palo Alto |
| California |
| 94304 |
| United States |
| Children's Hospital of Colorado - Pulmonology | Aurora | Colorado | 80045 | United States |
| The Emory Clinic - Clifton Road - Pulmonology | Atlanta | Georgia | 30322 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology | Chicago | Illinois | 60611 | United States |
| JW Riley Hospital for Children - Pulmonology | Indianapolis | Indiana | 46202 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| The Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| St. Louis Children's Hospital - Pulmonology | St Louis | Missouri | 63110 | United States |
| Cohen Children's Medical Center - Lakeville Road | Lake Success | New York | 11042 | United States |
| Cincinnati Children's Hospital Medical Center - Pulmonology | Cincinnati | Ohio | 45229 | United States |
| UH Cleveland Medical Center - Pulmonology | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Children's Hospital of Pittsburgh - Pulmonology | Pittsburgh | Pennsylvania | 15224 | United States |
| Texas Children's Hospital - Wallace Tower - Pulmonology | Houston | Texas | 77030 | United States |
| Vermont Lung Center | Colchester | Vermont | 05446 | United States |
| American Family Childrens Hospital | Madison | Wisconsin | 53792 | United States |
| The Kids Research Institute Australia | Nedlands | Australia |
| Women's & Children's Hospital | North Adelaide | Australia |
| The Royal Children's Hospital Melbourne | Parkville | Australia |
| Children's Health Queensland Hospital and Health Service | South Brisbane | Australia |
| Hospital for Sick Children - Pulmonology | Toronto | Canada |
| British Columbia Children's Hospital | Vancouver | Canada |
| Hopital Femme Mere-Enfant | Bron | France |
| Hopital Necker Enfants Malades - Pulmonology | Paris | France |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Universitatsklinikum Essen | Essen | Germany |
| Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre | Hanover | Germany |
| Sophia Children's Hospital | Rotterdam | Netherlands |
| Starship Child Health | Grafton | New Zealand |
| Sahlgrenska Universitetssjukhuset - Göteborg CF-center | Gothenburg | Sweden |
| Karolinska University Hospital - Pulmonology | Stockholm | Sweden |
| Inselspital Bern | Bern | Switzerland |
| Kinderspital Zurich - Abteilung Pneumologie | Zurich | Switzerland |
| Noah's Ark Children's Hospital for Wales | Cardiff | United Kingdom |
| Great Ormond Street Hospital for Children | London | United Kingdom |
| 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 |
|---|---|
| C000731138 | deutivacaftor, tezacaftor , vanzacaftor |
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