CFTR Modulator
Lung Perfusion Mapping for Cystic Fibrosis
This trial will use a new MRI method to measure regional lung perfusion in children with CF and relate it to regional assessments of ventilation and to serum cytokines or proteomic markers of angiogenesis and inflammatory processes.
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Trials for CF Patients
Monoclonal Antibodies
Denosumab for Osteoporosis in Cystic Fibrosis
This trial is testing a new way to treat CF patients with bone disease. Up to 100 subjects with CF will participate in a single study visit that will include a DEXA scan, micro CT, and blood collection. If results indicate bone disease, subjects will receive treatment with Denosumab for up to 5 years.
Cephalosporin Antibiotic
Ceftaroline for Cystic Fibrosis
This trial is testing a new way to give the antibiotic ceftaroline to kids with cystic fibrosis who also have a history of methicillin-resistant Staphylococcus aureus (MRSA), also known as oxacillin-resistant Staphylococcus aureus (ORSA).
Procedure
Glycine Buffer for Airway pH Measurement in Asthma
This trial is testing a new way to measure airway pH in people with asthma and cystic fibrosis, using a new inhaled drug. The airway pH will help doctors tailor treatment plans for these conditions.
Trials for CFTR Positive Patients
Procedure
Glycine Buffer for Airway pH Measurement in Asthma
This trial is testing a new way to measure airway pH in people with asthma and cystic fibrosis, using a new inhaled drug. The airway pH will help doctors tailor treatment plans for these conditions.
Angiotensin II Receptor Blocker
Losartan for Cystic Fibrosis
This trial is testing losartan, a blood pressure medication, to see if it can help clear mucus from the lungs of cystic fibrosis patients who can't use current treatments. These patients need alternative treatments because they don't respond to existing options. Losartan may reduce lung inflammation, making it easier for them to breathe.
Anti-bacterial
Antibiotics for Cystic Fibrosis
This trial tests if the antibiotic Cephalexin can help children with cystic fibrosis and a specific bacterial infection (MSSA) by improving their lung function. Cephalexin has been used in previous studies for antistaphylococcal prophylaxis in infants and young children with cystic fibrosis.
Phase 3 Trials
Anti-bacterial
Antibiotics for Cystic Fibrosis
This trial tests if the antibiotic Cephalexin can help children with cystic fibrosis and a specific bacterial infection (MSSA) by improving their lung function. Cephalexin has been used in previous studies for antistaphylococcal prophylaxis in infants and young children with cystic fibrosis.
CFTR Modulator
VX-121 + Tezacaftor + Deutivacaftor for Cystic Fibrosis
This trial tests a combination of three drugs for cystic fibrosis patients with a specific genetic mutation. The drugs work together to fix the faulty protein in their cells, helping their lungs and other organs function better. Trikafta is a combination of three drugs (elexacaftor, tezacaftor, and ivacaftor) that target the F508del mutation in the CFTR gene.
Antibiotic
Rifaximin for Bloating in Cystic Fibrosis
This trial is testing rifaximin, an antibiotic, to help people with cystic fibrosis who have gut symptoms like bloating. The antibiotic works by balancing gut bacteria to reduce discomfort. Rifaximin has been used to treat travelers' diarrhea and other conditions, and it has shown effectiveness in reducing symptoms of irritable bowel syndrome (IBS).
Trials With No Placebo
Monoclonal Antibodies
Denosumab for Osteoporosis in Cystic Fibrosis
This trial is testing a new way to treat CF patients with bone disease. Up to 100 subjects with CF will participate in a single study visit that will include a DEXA scan, micro CT, and blood collection. If results indicate bone disease, subjects will receive treatment with Denosumab for up to 5 years.
Cephalosporin Antibiotic
Ceftaroline for Cystic Fibrosis
This trial is testing a new way to give the antibiotic ceftaroline to kids with cystic fibrosis who also have a history of methicillin-resistant Staphylococcus aureus (MRSA), also known as oxacillin-resistant Staphylococcus aureus (ORSA).
Procedure
Glycine Buffer for Airway pH Measurement in Asthma
This trial is testing a new way to measure airway pH in people with asthma and cystic fibrosis, using a new inhaled drug. The airway pH will help doctors tailor treatment plans for these conditions.
Angiotensin II Receptor Blocker
Losartan for Cystic Fibrosis
This trial is testing losartan, a blood pressure medication, to see if it can help clear mucus from the lungs of cystic fibrosis patients who can't use current treatments. These patients need alternative treatments because they don't respond to existing options. Losartan may reduce lung inflammation, making it easier for them to breathe.
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Frequently Asked Questions
Introduction to cystic fibrosis
What are the top hospitals conducting cystic fibrosis research?
In the ever-evolving landscape of cystic fibrosis research, several top-notch hospitals are leading the way with their groundbreaking clinical trials. Boston Children's Hospital in Boston is at the forefront, currently conducting an impressive 13 active trials for cystic fibrosis. Their commitment to this cause spans over time and includes a remarkable 57 all-time trials since their first recorded investigation in 2008. Similarly, Nationwide Children's Hospital in Columbus has established itself as a powerhouse in cystic fibrosis research with equal enthusiasm, boasting 13 ongoing trials and an expansive history of 66 completed studies dating back to their inaugural trial in 2005.
Meanwhile, down south at the University of Alabama at Birmingham, researchers are diligently working on finding new treatments for cystic fibrosis through their involvement in 12 active clinical trials. With a strong track record of dedication to this field, they have conducted a total of 56 previous investigations since embarking upon their first trial in 2004. In Kansas City, the University of Kansas Medical Center joins these efforts by currently running ten ongoing cystic fibrosis trials while having accomplished valuable insights from prior experimentation spanning across thirty-five studies starting from its pioneering research commenced fifteen years agoin2005.
Furthermore adding itself proudly into this group is National Jewish Health located amidst Denver’s beautiful surroundings; here they showcase ten vibrant current initiatives examining novel approaches towards CF management meanwhile keeping up pace with seventy-six earlier trails from venturing into such investigative endeavors since officially commencing it around fifteen years ago until sustaining present times eleven years after inception; not only do these promising developments provide hope but also serve as motivation driving us closer towards better care prospects for individuals affected by CF nationwide.
These esteemed institutions embody optimism and progress when it comes to combating cystic fibrosis—a complex genetic disorder affecting multiple organs including lungs and digestive system—ultimately striving towards improved quality-of-life options available for patients worldwide.
Which are the best cities for cystic fibrosis clinical trials?
Pittsburgh, Pennsylvania; Boston, Massachusetts; Indianapolis, Indiana; Birmingham, Alabama; and Minneapolis, Minnesota are among the best cities for cystic fibrosis clinical trials. These cities have a significant number of active trials focused on studying various treatments like VX-121/TEZ/D-IVA, Stage 1/2a Arm 3, sodium nitrite, β-lactam Only (Non-AG), Imipenem/cilastatin/relebactam, Symdeko, Ivacaftor, and more. With their commitment to research and development in cystic fibrosis treatment options, these cities offer individuals with this condition access to cutting-edge clinical studies that pave the way for advancements in care and improved outcomes.
Which are the top treatments for cystic fibrosis being explored in clinical trials?
Clinical trials for cystic fibrosis have brought forth a range of promising treatments. One standout contender is VX-121/TEZ/D-IVA, currently being explored in four active trials and showing potential since its first listing in 2021. Additionally, ELX/TEZ/IVA has caught the attention of researchers with three ongoing trials and an impressive track record of 19 all-time cystic fibrosis studies since its introduction in 2018. Another avenue being explored is the initiation of CFTR modulator therapy, with two active trials aiming to uncover new possibilities for treating this condition that affect many individuals worldwide. Trikafta and exercise interventions also hold promise as they undergo evaluation through clinical investigation for their efficacy in managing cystic fibrosis symptoms and improving patient outcomes.
What are the most recent clinical trials for cystic fibrosis?
Exciting developments are underway in the realm of cystic fibrosis treatment, with recent clinical trials offering hope for improved outcomes. One such trial involves Rifaximin 550 MG Oral Tablet [XIFAXAN]. This medication has progressed to Phase 2 and Phase 3 studies, demonstrating potential benefits for individuals with cystic fibrosis. Another promising avenue is the use of VNZ/TEZ/D-IVA, currently in Phase 3 testing, which shows promise as a therapeutic option. Additionally, KB407 (Nebulization) has entered Phase 1, highlighting its potential role in managing this condition. These advancements signal progress toward better treatment options for those affected by cystic fibrosis.
What cystic fibrosis clinical trials were recently completed?
Several recent clinical trials have made significant strides in the pursuit of better treatments for cystic fibrosis. In September 2022, AbbVie successfully completed a trial evaluating Galicaftor's potential effectiveness. Vertex Pharmaceuticals Incorporated also achieved milestones with two trials: one investigating VX-121/TEZ/D-IVA in July 2022 and another exploring ELX/TEZ/IVA in May 2022. Nabriva Therapeutics AG completed a trial on Lefamulin in March 2022, while Insmed Incorporated concluded their Brensocatib study in November 2021. These advancements highlight ongoing efforts to improve outcomes and quality of life for individuals living with cystic fibrosis.