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Completed

NCT Number: NCT03691779

Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 year–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Queensland Children's Hospital, South Brisbane, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

ELX/TEZ/IVA

Drug

Fixed-dose combination tablet orally once daily in the morning.

Other names: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor

IVA

Drug

IVA tablet orally once daily in the evening.

Other names: VX-770, ivacaftor

Primary outcomes

  1. Part A: Maximum Observed Plasma Concentration (Cmax) of ELX, TEZ, and IVA

    Time frame: Part A: Day 15

  2. Part A: Observed Pre-dose Plasma Concentration (Ctrough) of ELX, TEZ, and IVA

    Time frame: Part A: Day 15

  3. Part A: Area Under the Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24h) of ELX, TEZ, and IVA

    Time frame: Part A: Day 15

  4. Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Time frame: Part B: Day 1 Through Safety Follow-up Visit (up to Week 28)

Secondary outcomes

  1. Part A: Cmax of ELX Metabolite (M23-ELX), TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)

    Time frame: Part A: Day 15

  2. Part A: Ctrough of ELX Metabolite (M23-ELX), TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)

    Time frame: Part A: Day 15

  3. Part A: AUC0-24h of ELX Metabolite (M23-ELX) and TEZ Metabolite (M1-TEZ)

    Time frame: Part A: Day 15

  4. Part A: Area Under the Concentration Versus Time Curve From 0 to 6 Hours (AUC0-6h) of IVA Metabolite (M1-IVA)

    Time frame: Part A: Day 15

    The AUC data was analyzed for up to 6 hours for IVA metabolite (M1-IVA). Therefore, AUC0-6h is reported for M1-IVA metabolite.

  5. Part A: Safety and Tolerability as Assessed by Number of Participants With TEAEs and SAEs

    Time frame: Part A: Day 1 Through Safety Follow-up Visit (up to Day 43)

  6. Part B: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

    Time frame: Part B: From Baseline Through Week 24

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  7. Part B: Absolute Change in Sweat Chloride (SwCl)

    Time frame: Part B: From Baseline Through Week 24

    Sweat samples were collected using an approved collection device.

  8. Part B: Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score

    Time frame: Part B: From Baseline Through Week 24

    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.

  9. Part B: Absolute Change in Body Mass Index (BMI)

    Time frame: Part B: From Baseline at Week 24

    BMI was defined as weight in kg divided by squared height in meters (m^2).

  10. Part B: Absolute Change in BMI For-Age Z-Score

    Time frame: Part B: From Baseline at Week 24

    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.

  11. Part B: Absolute Change in Weight

    Time frame: Part B: From Baseline at Week 24

  12. Part B: Absolute Change in Weight-for-age Z-Score

    Time frame: Part B: From Baseline at Week 24

    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.

  13. Part B: Absolute Change in Height

    Time frame: Part B: From Baseline at Week 24

  14. Part B: Absolute Change in Height-for-Age Z-Score

    Time frame: Part B: From Baseline at Week 24

    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.

  15. Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale

    Time frame: Part B: At Week 24

    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.

  16. Part B: Number of Pulmonary Exacerbations Events

    Time frame: Part B: From Baseline Through Week 24

    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.

  17. Part B: Number of CF Related Hospitalizations

    Time frame: Part B: From Baseline Through Week 24

    The total number of CF related hospitalization events across all participants were reported.

  18. Part B: Ctrough of ELX, ELX Metabolite (M23-ELX), TEZ, TEZ Metabolite (M1-TEZ), IVA and IVA Metabolite (M1-IVA)

    Time frame: Part B: At Week 4

  19. Part B: Absolute Change in Lung Clearance Index 2.5 (LCI2.5)

    Time frame: Part B: From Baseline Through Week 24

    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.

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study 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

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Oct 2, 2018
Registry last updated
Oct 22, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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