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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-513754-29-00 | Other Identifier | EU Trial (CTIS) Number |
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The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: VX-121/TEZ/D-IVA | Experimental | Participants will receive VX-121/TEZ/D-IVA in the morning. |
|
| Part B: VX-121/TEZ/D-IVA | Experimental | Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-121/TEZ/D-IVA | Drug | Fixed-dose combination for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites | From Day 1 up to Day 22 | |
| Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to Day 50 | |
| Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 28 |
| Measure | Description | Time Frame |
|---|---|---|
| Part B: Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 24 | |
| Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites | From Day 1 up to Week 16 |
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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 |
|---|---|---|---|---|---|---|
| Children's Hospital of Orange County | Orange | California | 92868 | United States | ||
| Stanford University Clinical and Translational Research Unit |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39756425 | Derived | Hoppe JE, Kasi AS, Pittman JE, Jensen R, Thia LP, Robinson P, Tirakitsoontorn P, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Salinas DB, Zhu J, Chen YC, Rodriguez-Romero V, Sosnay PR, Davies G; VX21-121-105 Study Group. Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med. 2025 Mar;13(3):244-255. doi: 10.1016/S2213-2600(24)00407-7. Epub 2025 Jan 2. |
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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 B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale | At Day 1 and Week 24 |
| Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) | From Baseline Through Week 24 |
| Part B: Number of Pulmonary Exacerbation (PEx) | From Baseline Through Week 24 |
| Part B: Number of CF-Related Hospitalizations | From Baseline Through Week 24 |
| Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score | From Baseline Through Week 24 |
| Part B: Absolute Change in Body Mass Index (BMI) | From Baseline at Week 24 |
| Part B: Absolute Change in BMI-for-age Z-score | From Baseline at Week 24 |
| Part B: Absolute Change in Weight | From Baseline at Week 24 |
| Part B: Absolute Change in Weight-for-age Z-score | From Baseline at Week 24 |
| Part B: Absolute Change in Weight-for-length | From Baseline at Week 24 |
| Part B: Absolute Change in Weight-for-length Z-score | From Baseline at Week 24 |
| Part B: Absolute Change in Height | From Baseline at Week 24 |
| Part B: Absolute Change in Height-for-age Z-score | From Baseline at Week 24 |
| Part B: Absolute Change in Length | From Baseline at Week 24 |
| Part B: Absolute Change in Length-for-age Z-score | From Baseline at Week 24 |
| Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L) | From Baseline Through Week 24 |
| Part B: Proportion of Participants With SwCl <30 mmol/L | From Baseline Through Week 24 |
| Palo Alto |
| California |
| 94304 |
| United States |
| Children's Hospital of Colorado | Aurora | Colorado | 80045 | United States |
| The Emory Clinic / Children's Healthcare of Atlanta at Egleston | Atlanta | Georgia | 30322 | 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 |
| Washington University School of Medicine / St. Louis Children's Hospital | St Louis | Missouri | 63110 | United States |
| Cohen Children's Medical Center | Lake Success | New York | 11042 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Texas Children's Hospital - Wallace Tower | 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 and Children's Hospital | North Adelaide | Australia |
| The Royal Children's Hospital | Parkville | Australia |
| Queensland Children's Hospital | South Brisbane | Australia |
| The Hospital for Sick Children | Toronto | Canada |
| British Columbia Children's Hospital | Vancouver | Canada |
| CHU Lyon - Hopital Femme Mere-Enfant | Bron | France |
| Hopital Necker, Enfants Malades | Paris | France |
| Charité - Paediatric Pulmonology Department | Berlin | Germany |
| Universitatsklinikum Essen | Essen | Germany |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Erasmus Medical Center / Sophia Children's Hospital | Rotterdam | Netherlands |
| Starship Children's Hospital | Grafton | New Zealand |
| Sahlgrenska Universitetssjukhuset | Gothenburg | Sweden |
| Karolinska University Hospital - Pulmonology | Stockholm | Sweden |
| Inselspital - Universitaetsspital Bern | Bern | Switzerland |
| Kinderspital Zurich | Zurich | Switzerland |
| Children and Young Adults Research Unit | 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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