Clinical trials: Cystic Fibrosis
Cystic Fibrosis (CF)
Rethinking Early Airway Clearence Therapies
The REACT trial consists of two parallel, randomized studies; the Hypertonic Saline Study and the Dornase Alfa Study. Health outcomes among people with cystic fibrosis (CF) have been steadily improving, most recently with the…
- Trial name
- REACT (NCT07696338)
- Testing
- As-needed hypertonic saline (HS), Twice daily hypertonic saline (HS), As-needed dornase alfa (DA)
- Where
- 39 sites in United States, Canada
- Volunteers
- 405 people
- Sponsor
- Nicole Hamblett
Cystic Fibrosis Related Diabetes, Cystic Fibrosis (CF)
Verapamil Effect in Cystic Fibrosis-related Dysglycemia
The investigators are conducting a pilot open-label pre/post interventional trial in adolescents and adults with cystic fibrosis (CF) and abnormal glucose tolerance or early CF-related diabetes mellitus (CFRD) to assess the…
- Conditions
- Cystic Fibrosis Related Diabetes, Cystic Fibrosis (CF), Abnormal Glucose Tolerance
- Trial name
- NCT07688070
- Testing
- Verapamil Hydrochloride
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Rhode Island Hospital
Cystic Fibrosis, COPD
High Resolution Micro OCT Imaging
The purpose of this study is to learn about using the imaging to make images of the lungs and nose with the long-term goal of the research leading to potential treatments and new therapies for patients with cystic fibrosis.
- Conditions
- Cystic Fibrosis, COPD, PCD - Primary Ciliary Dyskinesia, Covid19
- Trial name
- NCT03256773
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Cystic Fibrosis
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).
- Trial name
- BEGIN (NCT04509050)
- Testing
- Ivacaftor or elexacaftor/tezacaftor/ivacaftor
- Where
- 35 sites in United States
- Volunteers
- 210 people
- Sponsor
- Sonya Heltshe
Cystic Fibrosis, Cystic Fibrosis Pulmonary Exacerbation
Standardizing Treatments for Pulmonary Exacerbations - Aminoglycoside Study
The purpose of this study is to look at pulmonary exacerbations in people with cystic fibrosis (CF) that need to be treated with antibiotics given through a tube inserted into a vein (intravenous or IV).
- Trial name
- STOP360AG (NCT05548283)
- Testing
- Beta-lactam antibiotic, Aminoglycoside
- Where
- 60 sites in United States, Canada
- Volunteers
- 730 people
- Sponsor
- Chris Goss
Cystic Fibrosis, Back Pain
Osteopathic Treatment in Adult Patients With Cystic Fibrosis
To study the contribution of osteopathy on the reduction of pain in adult patients with cystic fibrosis
- Conditions
- Cystic Fibrosis, Back Pain, Neck Pain, Chest Pain
- Trial name
- MUCOSTEO (NCT01293019)
- Testing
- Osteopathic treatment, Usual care
- Where
- France
- Volunteers
- 32 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Non-cystic Fibrosis Bronchiectasis
Long-term Evaluation of the SIMEOX Device at Home in Non-cystic Fibrosis Bronchiectasis
Bronchiectasis is a chronic lung disease of multiple aetiologies characterised by permanent dilatation of the calibre of a territory of the bronchial tree with impaired mucociliary clearance.
- Trial name
- Home-BRAC (NCT06487273)
- Testing
- SIMEOX, Remote Physiotherapy
- Where
- 57 sites in France, Germany and 4 more
- Volunteers
- 622 people
- Sponsor
- Physio-Assist
Cystic Fibrosis Lung
4D-710 in Adult Patients With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Trial name
- CF (NCT05248230)
- Testing
- 4D-710
- Where
- 20 sites in United States
- Volunteers
- 30 people
- Sponsor
- 4D Molecular Therapeutics
Cystic Fibrosis (CF)
TIDES 2.0: Prevalence and Longitudinal Course of Depression, Anxiety, and Behavior Problems in Children With Cystic Fibrosis Under 12 Years of Age
This is a longitudinal, observational epidemiological study designed to estimate the prevalence of depression, anxiety, and behavior problems in children ages 18 months through 11 years with cystic fibrosis (CF).
- Trial name
- TIDES 2 (NCT07048574)
- Where
- 13 sites in United States
- Volunteers
- 600 people
- Sponsor
- State University of New York at Buffalo
Cystic Fibrosis, Ventilatory Defect
Quantitation and Spatial Registration of Airways Dysfunction With Dynamic 19F MRI in Cystic Fibrosis
The purpose of this study is to look at lung ventilation in people with cystic fibrosis over time (1 year) using magnetic resonance imaging (MRI) with an inhaled contrast gas, and compare these measures to lung function assessed…
- Trial name
- NCT06057714
- Testing
- 19F MRI
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of North Carolina, Chapel Hill
Cystic Fibrosis in Children
19F MRI in Healthy Children and Children With Mild Cystic Fibrosis Lung Disease
This study capitalizes on the emerging technology of 19F MRI, using conventional 'thermally' polarized perfluorinated gas (perfluoropropane, or PFP) mixed with oxygen and studied with magnetic resonance imaging (MRI) to visualize…
- Trial name
- NCT06066723
- Testing
- 19F MRI
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Cystic Fibrosis (CF), Cystic Fibrosis Gastrointestinal Disease
Gastrointestinal Response of Pediatric Cystic Fibrosis Patients on Mediterranean Diet
There is no study to date that has evaluated the impact or effect of a Mediterranean diet in children with CF. The goal of this study would be to help provide better guidance around questions the investigators, as CF care…
- Trial name
- MedDietCF (NCT07223255)
- Testing
- Mediterranean diet
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Dartmouth-Hitchcock Medical Center
Cystic Fibrosis (CF), New Diagnostics
Study to Enable New Diagnostics for Pulmonary Microbes in People With CF
Sputum culture has been the best approach to detect harmful bacteria in the lungs of people with cystic fibrosis (CF). With the widespread use of new CF therapies (like Trikafta and Alyftrak), it is more difficult for people with…
- Trial name
- SEND-CF (NCT07312734)
- Where
- 9 sites in United States
- Volunteers
- 300 people
- Sponsor
- Chris Goss
Cystic Fibrosis (CF), Pancreatic Insufficiency
Safety of Stopping Pancreatic Enzyme Replacement Therapy in Children With Improved Pancreatic Function After Highly Effective Modulator Therapy
The goal of this clinical trial is to evaluate the safety of stopping pancreatic enzyme replacement therapy (PERT) in children with cystic fibrosis (CF) receiving CFTR modulator therapy (CFTRm) who have regained pancreatic…
- Conditions
- Cystic Fibrosis (CF), Pancreatic Insufficiency, Pancreas Disease, Exocrine Pancreatic Insufficiency
- Trial name
- NCT07632768
- Testing
- Discontinuation of Pancreatic Enzyme Replacement Therapy
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Indiana University
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Cystic Fibrosis
Volatilomic Approaches for the Study of CFTR Modulators (VOLATIL-CF)
This study relies on the hypotheses that (1) exhaled breath is intimately correlated to the patient's lung condition and that (2)the composition of exhaled breath , i.e.
- Trial name
- VOLATIL-CF (NCT05726994)
- Testing
- exhaled breath collection, Data collection
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Severe Asthma, COPD
Evaluation of Ventilation Defects Downstream of Mucus Plugs in Patients With Muco-Obstructive Lung Disease
In this study, xenon MRI will be used to evaluate regional functional consequences of mucus plugs in the lungs of patients with muco-obstructive pulmonary disease.
- Conditions
- Severe Asthma, COPD, Non-CF Bronchiectasis, Cystic Fibrosis (CF)
- Trial name
- NCT06985225
- Testing
- Hyperpolarized 129 Xenon
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Kansas Medical Center
Cystic Fibrosis
Non-invasive Prenatal Diagnosis of Monogenic Disorders by Linked-reads Technology
Description of the presence of cell-free fetal DNA in maternal plasma allowed the possibility of non-invasive prenatal diagnosis.
- Trial name
- NID (NCT03622892)
- Where
- 6 sites in France
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- University Hospital, Brest
Cystic Fibrosis Lung, Respiratory Infections, Recurrent, Chronic
A Double-Blind, Active-Controlled, Multiple-Ascending Dose Study of Aerosolized RSP-1502 in Subjects With CF and Chronic PA Lung Infection
A double-blind, active-controlled, multiple-ascending dose, safety study of aerosolized RSP-1502 in subjects with cystic fibrosis Pseudomonas aeruginosa lung infection.
- Conditions
- Cystic Fibrosis Lung, Respiratory Infections, Recurrent, Chronic, Pseudomonas Aeruginosa
- Trial name
- NCT06016088
- Testing
- RSP-1502, Tobramycin inhalation solution
- Where
- 22 sites in United States, Australia
- Volunteers
- 71 people
- Sponsor
- Respirion Pharmaceuticals Pty Ltd
Cystic Fibrosis (CF)
Home Air Pollution in Children With Cystic Fibrosis Study
Cystic Fibrosis (CF) is a devastating chronic pulmonary disease that continues to cause significant morbidity and mortality despite great advances in therapies.
- Trial name
- HEROIC-CF (NCT07665203)
- Volunteers
- 200 people
- Sponsor
- Seattle Children's Hospital