Cystic Fibrosis Clinical Trial
Official title:
Real World Clinical Outcomes With Novel Modulator Therapy Combinations in People With CF (RECOVER)
RECOVER is a prospective, multicenter observational study designed to measure the real world clinical effectiveness of elexacaftor, tezacaftor and ivacaftor triple combination therapy (Kaftrio) in people with cystic fibrosis over a two year period. Measured outcomes include measures of lung function, lung inflammation, lung imaging, abdominal symptoms, gut inflammation, liver function, pancreatic exocrine function, nasal inflammation, quality of life and adherence to therapy. The study will examine outcomes in children aged six years and above over a period of two years. The first phase of the study will commence in 2020, recruiting children 12 years and older who have started on clinical treatment with Kaftrio.
Our aim with RECOVER is to examine the clinical impact of Kaftrio on key clinical outcomes in people with CF in a real-world setting. For this study, in addition to some of the more traditional ways of monitoring clinical outcomes in people with CF such a standard lung function, nutrition, exacerbations and liver disease, we are proposing to include some novel outcome measures not typically used in clinical trials such as lung clearance index (LCI) and spirometry controlled chest CT. By implementing an extensive study protocol that will include important outcomes in a number of areas of health in people with CF, and matching this to a comprehensive biosample collection plan, we will have the power to gain important insight into how Kaftrio works, and what impact it has on rescue of CFTR function in this group of people. Data on the following outcomes will be collected during the study: Lung Clearance Index Ultra-low dose, spirometry-controlled CT scanning Sweat Chloride Nasal Lavage (inflammatory markers and microbiome) Fraction of Exhaled Nitric Oxide (FeNO) Liver Ultrasound Liver examination (signs of liver disease) Sputum Collection (inflammatory markers and microbiome) Stool Collection (inflammation, microbiome, fecal elastase) Abdominal symptom questionnaire CFQ-R (quality of life) Adherence to treatment Height, weight, BMI Forced Expiratory volume in 1 second (FEV1) Microbiological culture of airway specimens (clinical laboratories at sites) The Lead Investigator is Paul McNally, with Prof. Jane Davies as Co-Lead Investigator. The study will operate in collaboration with our academic and clinical partners and the CF registries in Ireland and the UK. The study is supported by the European CF Society Clinical Trials Network (ECFS-CTN). The study is being run as a CTIMP in the UK clinical sites, as determined by the MHRA. In the Irish sites, the HPRA has determined this study to be an observational research study. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT04696198 -
Thoracic Mobility in Cystic Fibrosis Care
|
N/A | |
Completed |
NCT00803205 -
Study of Ataluren (PTC124™) in Cystic Fibrosis
|
Phase 3 | |
Terminated |
NCT04921332 -
Bright Light Therapy for Depression Symptoms in Adults With Cystic Fibrosis (CF) and COPD
|
N/A | |
Completed |
NCT03601637 -
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
|
Phase 3 | |
Terminated |
NCT02769637 -
Effect of Acid Blockade on Microbiota and Inflammation in Cystic Fibrosis (CF)
|
||
Recruiting |
NCT06032273 -
Lung Transplant READY CF 2: CARING CF Ancillary RCT
|
N/A | |
Recruiting |
NCT06030206 -
Lung Transplant READY CF 2: A Multi-site RCT
|
N/A | |
Recruiting |
NCT06012084 -
The Development and Evaluation of iCF-PWR for Healthy Siblings of Individuals With Cystic Fibrosis
|
N/A | |
Recruiting |
NCT06088485 -
The Effect of Bone Mineral Density in Patients With Adult Cystic Fibrosis
|
||
Recruiting |
NCT05392855 -
Symptom Based Performance of Airway Clearance After Starting Highly Effective Modulators for Cystic Fibrosis (SPACE-CF)
|
N/A | |
Recruiting |
NCT04056702 -
Impact of Triple Combination CFTR Therapy on Sinus Disease.
|
||
Recruiting |
NCT04039087 -
Sildenafil Exercise: Role of PDE5 Inhibition
|
Phase 2/Phase 3 | |
Completed |
NCT04038710 -
Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.
|
||
Completed |
NCT04058548 -
Clinical Utility of the 1-minute Sit to Stand Test as a Measure of Submaximal Exercise Tolerance in Patients With Cystic Fibrosis During Acute Pulmonary Exacerbation
|
N/A | |
Completed |
NCT03637504 -
Feasibility of a Mobile Medication Plan Application in CF Patient Care
|
N/A | |
Recruiting |
NCT03506061 -
Trikafta in Cystic Fibrosis Patients
|
Phase 2 | |
Completed |
NCT03566550 -
Gut Imaging for Function & Transit in Cystic Fibrosis Study 1
|
||
Recruiting |
NCT04828382 -
Prospective Study of Pregnancy in Women With Cystic Fibrosis
|
||
Completed |
NCT04568980 -
Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis
|
||
Recruiting |
NCT04010253 -
Impact of Bronchial Drainage Technique by the Medical Device Simeox® on Respiratory Function and Symptoms in Adult Patients With Cystic Fibrosis
|
N/A |