A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
- Conditions
- Cystic Fibrosis
- Interventions
- Drug: PlaceboDrug: TEZ/IVADrug: IVA
- Registration Number
- NCT03559062
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 67
- Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
- Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
- Participants with a screening LCI2.5 result ≥7.5.
- Participants who are able to swallow tablets.
Key
- Clinically significant cirrhosis with or without portal hypertension.
- Colonization with organisms associated with a more rapid decline in pulmonary status.
- Solid organ or hematological transplantation.
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks. TEZ/IVA TEZ/IVA Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks. TEZ/IVA IVA Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks. TEZ/IVA Placebo Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks. Ivacaftor IVA Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks. Ivacaftor Placebo Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
- Primary Outcome Measures
Name Time Method Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8 From baseline through Week 8 LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
- Secondary Outcome Measures
Name Time Method Absolute Change in Sweat Chloride At Week 8 From baseline at Week 8 Sweat samples were collected using an approved collection device.
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8 From baseline through Week 8 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.
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit From first dose of study drug up to safety follow-up visit (up to Week 12)
Trial Locations
- Locations (27)
Universitaetsklinikum Koeln
🇩🇪Koeln, Germany
Groupe Hospitalier Pellegrin - Hôpital des Enfants
🇫🇷Bordeaux Cedex, France
Lady Cilento Children's Hospital
🇦🇺South Brisbane, Australia
Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ
🇵🇱Dziekanow Lesny, Poland
Universitair Ziekenhuis Brussel - Campus Jette
🇧🇪Brussels, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
The Children's Hospital at Westmead
🇦🇺Westmead, Australia
Universitaetsklinikum Heidelberg
🇩🇪Heidelberg, Germany
Universitaetsklinikum Tuebingen
🇩🇪Tuebingen, Germany
Inselspital - Universitaetsspital Bern
🇨🇭Bern, Switzerland
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
🇩🇪Giessen, Germany
Klinikum der Johann Wolfgang Goethe-Universitaet
🇩🇪Frankfurt, Germany
Princess Margaret Hospital for Children
🇦🇺Perth, Australia
Leeds General Infirmary
🇬🇧Leeds, United Kingdom
Kinderspital Zuerich
🇨🇭Zuerich, Switzerland
Southampton General Hospital
🇬🇧Southampton, United Kingdom
University of Copenhagen Rigshospitalet
🇩🇰Copenhagen, Denmark
Nottingham University Hospital City Campus
🇬🇧Nottingham, United Kingdom
Hôpital Necker - Enfants Malades
🇫🇷Paris, France
Universitaetsklinikum Jena
🇩🇪Jena, Germany
Hunter Medical Research Institute (HMRI)
🇦🇺New Lambton Heights, Australia
Our Lady's Children's Hospital
🇮🇪Dublin, Ireland
Universitaetsklinikum Essen
🇩🇪Essen, Germany
Royal Hospital for Sick Children
🇬🇧Edinburgh, United Kingdom
University Hospital Limerick
🇮🇪Limerick, Ireland
Royal Brompton Hospital
🇬🇧London, United Kingdom