Cystic Fibrosis Clinical Trial
Official title:
A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age
Verified date | June 2023 |
Source | Vertex Pharmaceuticals Incorporated |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 210 |
Est. completion date | June 2030 |
Est. primary completion date | June 2030 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 11 Years |
Eligibility | Key Inclusion Criteria: - Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene Key Exclusion Criteria: - History of solid organ, hematological transplantation, or cancer - Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15) - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply. |
Country | Name | City | State |
---|---|---|---|
Australia | Telethon Kids Institute | Nedlands | |
Australia | Women & Children's Hospital | North Adelaide | |
Australia | The Royal Children's Hospital | Parkville | |
Australia | Queensland Children's Hospital | South Brisbane | |
France | CHU Lyon - Hopital Femme Mere-Enfant | Bron Cedex | |
France | Hopital Necker, Enfants Malades | Paris Cedex 15 | |
Germany | Charite Paediatric Pulmonology Department | Berlin | |
Germany | Kinderklinik III, Abt. fur Pneumologie | Essen | |
Germany | Medizinische Hochschule Hannover | Hannover | |
Netherlands | Erasmus Medical Center / Sophia Children's Hospital | Rotterdam | |
Sweden | Sahlgrenska Universitetssjukhuset | Göteborg | |
Switzerland | Inselspital - Universitaetsspital Bern | Bern | |
Switzerland | Kinderspital Zuerich | Zürich | |
United Kingdom | Children and Young Adults Research Unit | Cardiff | |
United Kingdom | Great Ormond Street Hospital for Children | London | |
United States | The Emory Clinic / Children's Healthcare of Atlanta at Egleston | Atlanta | Georgia |
United States | Children's Hospital of Colorado | Aurora | Colorado |
United States | Boston Children's Hospital | Boston | Massachusetts |
United States | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio |
United States | Vermont Lung Center | Colchester | Vermont |
United States | Nationwide Children's Hospital | Columbus | Ohio |
United States | Texas Children's Hospital - Baylor College of Medicine | Houston | Texas |
United States | Riley Hospital for Children at Indiana University Health | Indianapolis | Indiana |
United States | The Children's Mercy Hospital | Kansas City | Missouri |
United States | University of Wisconsin Hospital and Clinics | Madison | Wisconsin |
United States | Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota |
United States | Cohen Children's Medical Center | New York | New York |
United States | Children's Hospital of Orange County | Orange | California |
United States | Stanford University | Palo Alto | California |
United States | UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Oregon Health & Science University | Portland | Oregon |
United States | Washington University School of Medicine / St. Louis Children's Hospital | Saint Louis | Missouri |
Lead Sponsor | Collaborator |
---|---|
Vertex Pharmaceuticals Incorporated |
United States, Australia, France, Germany, Netherlands, Sweden, Switzerland, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites | From Day 1 up to Day 22 | ||
Primary | 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 | ||
Primary | 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 | ||
Secondary | Part B: Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 24 | ||
Secondary | Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites | From Day 1 up to Week 16 | ||
Secondary | Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale | At Day 1 and Week 24 | ||
Secondary | Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) | From Baseline Through Week 24 | ||
Secondary | Part B: Number of Pulmonary Exacerbation (PEx) | From Baseline Through Week 24 | ||
Secondary | Part B: Number of CF-Related Hospitalizations | From Baseline Through Week 24 | ||
Secondary | Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score | From Baseline Through Week 24 | ||
Secondary | Part B: Absolute Change in Body Mass Index (BMI) | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in BMI-for-age Z-score | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Weight | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Weight-for-age Z-score | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Weight-for-length | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Weight-for-length Z-score | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Height | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Height-for-age Z-score | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Length | From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Length-for-age Z-score | From Baseline at Week 24 | ||
Secondary | Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L) | From Baseline Through Week 24 | ||
Secondary | Part B: Proportion of Participants With SwCl <30 mmol/L | From Baseline Through Week 24 |
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