Cystic Fibrosis Clinical Trial
Official title:
A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-445/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age
Verified date | August 2021 |
Source | Vertex Pharmaceuticals Incorporated |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-445, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects 6 through 11 years of age with F/F and F/MF genotypes.
Status | Completed |
Enrollment | 71 |
Est. completion date | August 7, 2020 |
Est. primary completion date | August 7, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Years to 11 Years |
Eligibility | Key Inclusion Criteria: - Homozygous or heterozygous for F508del mutation (F/F or F/MF genotypes) - Forced expiratory volume in 1 second (FEV1) value =40% of predicted mean for age, sex, and height. Key Exclusion Criteria: - Clinically significant cirrhosis with or without portal hypertension - Lung infection with organisms associated with a more rapid decline in pulmonary status. - Solid organ or hematological transplantation. Other protocol defined Inclusion/Exclusion criteria may apply. |
Country | Name | City | State |
---|---|---|---|
Australia | Queensland Children's Hospital | South Brisbane | |
Australia | The Children's Hospital at Westmead | Westmead | |
Canada | The Hospital for Sick Children | Toronto | |
Canada | British Columbia's Children's Hospital | Vancouver | |
Ireland | Children's Health Ireland at Crumlin | Dublin | |
Ireland | Children's Health Ireland at Temple Street | Dublin | |
United Kingdom | Birmingham Children's Hospital | Birmingham | |
United Kingdom | Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital | London | |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Boston Children's Hospital | Boston | Massachusetts |
United States | Clinical Research of Charlotte | Charlotte | North Carolina |
United States | Ann & Robert Lurie Children's Hospital of Chicago | Chicago | Illinois |
United States | Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio |
United States | Nationwide Children's Hospital | Columbus | Ohio |
United States | Texas Children's Hospital | Houston | Texas |
United States | The Children's Mercy Hospital | Kansas City | Missouri |
United States | Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota |
United States | Northwell Health- Long Island Jewish Medical Center | New Hyde Park | New York |
United States | Children's Hospital of Orange County | Orange | California |
United States | Oregon Health & Science University | Portland | Oregon |
United States | Seattle Children's Hospital | Seattle | Washington |
Lead Sponsor | Collaborator |
---|---|
Vertex Pharmaceuticals Incorporated |
United States, Australia, Canada, Ireland, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Part A: Maximum Observed Plasma Concentration (Cmax) of ELX, TEZ, and IVA | Part A: Day 15 | ||
Primary | Part A: Observed Pre-dose Plasma Concentration (Ctrough) of ELX, TEZ, and IVA | Part A: Day 15 | ||
Primary | Part A: Area Under the Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24h) of ELX, TEZ, and IVA | Part A: Day 15 | ||
Primary | Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Part B: Day 1 Through Safety Follow-up Visit (up to Week 28) | ||
Secondary | Part A: Cmax of ELX Metabolite (M23-ELX), TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA) | Part A: Day 15 | ||
Secondary | Part A: Ctrough of ELX Metabolite (M23-ELX), TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA) | Part A: Day 15 | ||
Secondary | Part A: AUC0-24h of ELX Metabolite (M23-ELX) and TEZ Metabolite (M1-TEZ) | Part A: Day 15 | ||
Secondary | Part A: Area Under the Concentration Versus Time Curve From 0 to 6 Hours (AUC0-6h) of IVA Metabolite (M1-IVA) | The AUC data was analyzed for up to 6 hours for IVA metabolite (M1-IVA). Therefore, AUC0-6h is reported for M1-IVA metabolite. | Part A: Day 15 | |
Secondary | Part A: Safety and Tolerability as Assessed by Number of Participants With TEAEs and SAEs | Part A: Day 1 Through Safety Follow-up Visit (up to Day 43) | ||
Secondary | Part B: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Part B: From Baseline Through Week 24 | |
Secondary | Part B: Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | Part B: From Baseline Through Week 24 | |
Secondary | Part B: Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | Part B: From Baseline Through Week 24 | |
Secondary | Part B: Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kg divided by squared height in meters (m^2). | Part B: From Baseline at Week 24 | |
Secondary | Part B: Absolute Change in BMI For-Age Z-Score | BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | Part B: From Baseline at Week 24 | |
Secondary | Part B: Absolute Change in Weight | Part B: From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Weight-for-age Z-Score | The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | Part B: From Baseline at Week 24 | |
Secondary | Part B: Absolute Change in Height | Part B: From Baseline at Week 24 | ||
Secondary | Part B: Absolute Change in Height-for-Age Z-Score | The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | Part B: From Baseline at Week 24 | |
Secondary | Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale | The study drug acceptability (participant reaction) was assessed by a visual analog scale that incorporates a 5 point facial hedonic scale (Liked it Very Much, Liked it a Little, Not sure, Disliked it a Little, Disliked it Very Much). Number of participants with the indicated categorical response in the drug acceptability assessment were reported. | Part B: At Week 24 | |
Secondary | Part B: Number of Pulmonary Exacerbations Events | Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol defined criteria. The total number of pulmonary exacerbations events across all participants were reported. | Part B: From Baseline Through Week 24 | |
Secondary | Part B: Number of CF Related Hospitalizations | The total number of CF related hospitalization events across all participants were reported. | Part B: From Baseline Through Week 24 | |
Secondary | Part B: Ctrough of ELX, ELX Metabolite (M23-ELX), TEZ, TEZ Metabolite (M1-TEZ), IVA and IVA Metabolite (M1-IVA) | Part B: At Week 4 | ||
Secondary | Part B: Absolute Change in Lung Clearance Index 2.5 (LCI2.5) | LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value. | Part B: From Baseline Through Week 24 |
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