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51 Cystic Fibrosis Trials

Power is an online platform that helps thousands of Cystic Fibrosis patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

This trial is testing a combination of three medications to help people with cystic fibrosis. The goal is to see if these drugs are safe and effective over time. These medications work by improving the function of a faulty protein in cystic fibrosis patients, which can enhance their lung function and overall health. Trikafta is a combination of three drugs (elexacaftor, tezacaftor, and ivacaftor) that target the F508del mutation of the CFTR gene.
No Placebo Group
Prior Safety Data
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 3
Age:1+
Sex:All
180 Participants Needed
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.
No Placebo Group
Prior Safety Data
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:12+
Sex:All
822 Participants Needed
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.
No Placebo Group
Prior Safety Data
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:1 - 11
Sex:All
210 Participants Needed
The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF). It will also learn if the drug works to treat works to treat CF with a specific mutation. The purpose of this research study is to: * test the safety and effectiveness of multiple doses of the study drug, SPL84 * test how multiple doses of the drug are processed by the body Researchers will compare drug SPL84 to a placebo (a look-alike substance that contains no drug) to see if drug SPL84 is safe and if it works to treat CF. Participants will: Take drug SPL84 or a placebo by inhalation every week for 9 weeks months Visit the clinic approximately 14 times over 17.5 weeks for checkups and tests
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
24 Participants Needed
This trial is testing VX-522, a new treatment for adults with cystic fibrosis who don't respond to current treatments. The treatment aims to fix or improve the faulty gene causing the disease, potentially reducing symptoms and improving health.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 64
Sex:All
33 Participants Needed
This trial is testing whether using one antibiotic or two different antibiotics together is better for treating lung infections in people with cystic fibrosis. It targets patients with worsening lung symptoms needing IV antibiotics. The antibiotics work by killing or stopping bacteria to improve breathing and reduce symptoms.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:6+
Sex:All
730 Participants Needed
This trial is testing a new gene therapy called 4D-710 in adults with cystic fibrosis who can't use standard treatments. The goal is to see if it is safe and effective. 4D-710 is part of a new generation of AAV vectors being developed for cystic fibrosis gene therapy.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
40 Participants Needed
CMTX-101 is a bacterial biofilm disrupting monoclonal antibody being developed as an adjunctive therapy to standard of care antibiotics. The goal of this clinical trial is to assess the safety and tolerability of CMTX-101 in people with cystic fibrosis (pwCF). The main questions the study aims to answer are: * Are single doses of CMTX-101 IV infusion safe and tolerated * What is the pharmacokinetic (PK) profile of single doses of CMTX-101 * Do single doses of CMTX-101 induce development of anti-drug antibodies (ADA) and neutralizing antibodies (Nabs)
Stay on current meds
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
41 Participants Needed
The purpose of this study is to assess the safety and tolerability of two 5-day infusion cycles of IV gallium in adult patients with CF who are infected with NTM. Funding Source - FDA Office of Orphan Products Development (OOPD)
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
40 Participants Needed
A double-blind, active-controlled, multiple-ascending dose, safety study of aerosolized RSP-1502 in subjects with cystic fibrosis Pseudomonas aeruginosa lung infection.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:12+
Sex:All
72 Participants Needed
Cystic fibrosis (CF) results in the thickening of mucus in the lungs and other organs due to dysfunction of a transmembrane conductance protein. This allows buildup of bacteria that results in inflammation, leading to tissue breakdown and loss of function. In the lungs, this process causes loss of air exchange structures progressing to diminished lung function. The exchange of oxygen in the lungs depends on both the integrity of air conduits and vasculature. Most clinical assessments, however, focus on ventilatory function, with the assumption that any vascular compromise is secondary. Nevertheless, there is evidence, some from the investigator's lab, to suggest that perfusion anomalies in the lung occur before signs of ventilatory dysfunction. Thus, the inflammatory processes of CF may impact pulmonary microvasculature specifically and concurrently or prior to damage to ventilatory structures. This study aims to apply a new MRI method to serially measure regional lung perfusion, without the use of contrast agent, in children with CF and to associate it with regional assessments of ventilation and to serum cytokines or proteomic markers of angiogenesis and inflammatory processes. The investigator's lab has recently developed a noninvasive, non-contrast, method of labeling blood flowing into the lungs and generating a map of perfusion. The investigator aims to couple this technique to existing methods using hyperpolarized Xenon to map ventilation. The investigator will apply these methods over time in CF patients, monitoring the relationship between regional perfusion and ventilation defects. This pilot work will provide the foundation for larger studies to establish the essential etiological role of perfusion deficits in CF.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:6 - 21
Sex:All
26 Participants Needed
The introduction of triple combination CFTR modulator therapy for patients with Cystic Fibrosis (CF) with at least one copy of the deltaF508 mutation is expected to provide major health benefits, but will also require novel outcome measures that can detect CF lung disease at an early stage, capture the efficacy of new therapies when disease manifestations are limited, as well as determine whether stopping existing chronic maintenance therapies does not have negative effects. In the past decade, research has focused on the multiple breath washout (MBW) test, as a sensitive outcome measure, especially if highly-effective modulator therapies are initiated in early childhood. Even LCI, however, may not adequately capture early lung function changes, thus warranting investigation of even more sensitive outcome measures. Magnetic resonance imaging (MRI) has the advantage of being a radiation-free modality, making it more suitable for assessing response to therapy in a shorter time frame with repeated imaging. Inhalation of a hyperpolarized gas enables the visualization and quantification of regional ventilation in the lung and can be combined with structural MRI to assess both structure and function in parallel. The main Investigator and others have recently formed an international consortium (the 129Xe MRI Clinical Trial Consortium), comprised of both imaging experts and pulmonary clinicians to standardize imaging procedures, thus facilitating multi-site implementations. Data from this proposed study (HyPOINT; Hyperpolarized Imaging for New Treatments) will inform the future utility of MRI for both longitudinal studies to track disease progression over time as well as for future interventional trials. Further, the current study could inform the design of future trials of interventions of patients for whom currently no effective CFTR modulator therapy is available and for patients with rare genotypes thus laying the groundwork for a more personalized medicine approach in the near-term future.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:6 - 18
Sex:All
64 Participants Needed
To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:6 - 8
Sex:All
44 Participants Needed
Lung transplant is an option for treating end-stage lung disease in cystic fibrosis (CF). In the United States, more people with CF and low lung function die each year than undergo lung transplant. More than half of people with CF who die without a lung transplant were never referred for consideration. Patient preference not to undergo lung transplant may account for 25-40% of decisions to defer referral. Rates of death without transplant are higher for people with CF who are members of marginalized communities, including those with Black race, Hispanic ethnicity, or low socioeconomic status. Increasing awareness of lung transplant among people with CF, and promoting understanding of the risks and benefits of transplant, can potentially reduce the number of people with CF who die without a lung transplant. The CF Foundation (CFF) lung transplant referral guidelines were developed to optimize the timing of referral for lung transplant. These guidelines recommend annual conversations with people with CF once their forced expiratory volume in one second (FEV1) is \<50% predicted. Considering lung transplant as a treatment option before it is medically needed will allow more time to learn about lung transplant and address any barriers to lung transplant that may exist. Investigators are interested in understanding how people with CF use lung transplant educational resources and how one prepares for having discussions and/or making decisions about lung transplant as a treatment option for advanced CF. The purpose of this study is to test whether a research website improves patient preparedness for discussions about lung transplant. Investigators also aim to understand whether there are unique factors that affect people with CF from communities with decreased access to transplant ("communities of concern"). Study involvement will span 6 months and study activities will involve the following: * Four Zoom research sessions (15-30 minutes each) * Survey assessments * Access to a research website that contains educational resources about lung transplant * Audio recording of a routine CF clinic visit to determine if and how lung transplant is discussed between a participant and his/her/their CF doctor
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
132 Participants Needed
This is an open-label, single center, prospective study of patients with a diagnosis of cystic fibrosis (CF) and a history of methicillin-resistant Staphylococcus aureus (MRSA), also known as oxacillin-resistant Staphylococcus aureus (ORSA), being treated with intravenous ceftaroline. Patients will have intravenous ceftaroline dosed according to current recommendations based upon its approved usage in pediatric patients for community acquired pneumonia. The investigators will analyze pharmacokinetic (PK) and pharmacodynamic (PD) data using serum concentrations of ceftaroline measured at various time-points following infusion of ceftaroline to develop PK/PD guided dosing strategies of ceftaroline in pediatric CF patients that would be tailored to account for bacterial susceptibility and disease progression.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:2 - 21
Sex:All
24 Participants Needed
The STOP PEDS RCT is a multicenter, parallel, open label randomized controlled trial evaluating the long-term (one year) and short-term safety and efficacy of two antibiotic treatment strategies for the management of outpatient pulmonary exacerbations (PEx) in the pediatric CF population.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:3 - 18
Sex:All
430 Participants Needed
The purpose of this study is to validate and utilize a personalized medicine approach to identify potential treatments with current FDA approved CFTR modifiers for non-approved CF gene mutations. The study will perform ex vivo testing of CFTR function and current marketed CFTR modulating drugs on expanded nasal cells at Cincinnati Children's Human Nasal Epithelium (HNE) Core Laboratory. The results will be confirmed and translated into bedside care through an N of 1 trial to determine effectiveness of treatment.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:6+
Sex:All
50 Participants Needed
This multi-center randomized controlled trial (RCT) will compare efficacy and safety endpoints using the insulin-only configuration of the iLet Bionic Pancreas System (BP) versus a control group using their usual care insulin delivery method and continuous glucose monitoring (CGM) during a 13-week study period in individuals ≥14 years old with cystic fibrosis-related diabetes (CFRD). After 13 weeks, participants will continue in a 13-week Extension Phase in which the BP group will continue to use the BP system and the Usual Care group will initiate use of the BP system.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:14+
Sex:All
150 Participants Needed
Implementation of a Clinical Tool to Improve Waitlist Mortality in Patients With Cystic Fibrosis
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
10 Participants Needed
This study is testing a non invasive way to measure airway pH in individuals with Asthma and Cystic Fibrosis using a new inhaled drug. The airway pH will help health care providers in creating tailored treatment plans for individuals suffering from these specific conditions.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18 - 50
Sex:All
74 Participants Needed
This is a phase 1b/2 study of a single dose of intravenous (IV) bacteriophage in males and non-pregnant females, at least 18 years old, diagnosed with Cystic Fibrosis (CF). This clinical trial is designed to assess the safety and microbiological activity of bacteriophage product Walter Reed Army Institute of Research- PAM-Cystic Fibrosis1 (WRAIR-PAM-CF1), directed at Pseudomonas aeruginosa in clinically stable CF individuals chronically colonized with P. aeruginosa. WRAIR-PAM-CF1 is a 4 component anti-pseudomonal bacteriophage mixture containing between 4 x 10\^7 and 4 x 10\^9 Plaque Forming Units (PFU) of bacteriophage. Enrollment will occur at up to 20 clinical sites in the United States. In stage 1, two eligible subjects will be assigned to each of the three dosing arms receiving a single dosage of the IV bacteriophage therapy (4 x 10\^7 PFU, 4 x 10\^8 PFU, and 4 x 10\^9 PFU; total of 6 sentinel subjects), followed by 30 plus or minus 7 days observation period. If no Serious Adverse Events (SAEs)(related to the study product) are identified during the 96 hours after bacteriophage administration for all Sentinel Subjects in Stage 1, the study will proceed to Stage 2. In Stage 2a, 32 subjects will be enrolled into one of 4 arms (placebo IV, 4 x 10\^7 PFU, 4 x 10\^8 PFU, and 4 x 10\^9 PFU) in a 1:1:1:1 allocation. An interim analysis will be performed after all subjects have completed follow up visit 5 on Day 8+3 to select the IV bacteriophage dose with the most favorable safety and microbiological activity profile. During Stage 2b, subjects will be randomized into the bacteriophage (dose selected based on Interim Analysis following Stage 2a) or placebo arm. The final sample size is expected to be up to 72 subjects total with up to 25 subjects in the placebo arm and up to 25 subjects in the Stage 2b bacteriophage dose.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18 - 99
Sex:All
73 Participants Needed
Image of trial facility.

ELX/TEZ/IVA for Cystic Fibrosis

Pittsburgh, Pennsylvania
This trial is testing a combination of three medications to help people with cystic fibrosis. The medications work together to fix a broken protein in the body, which can improve lung function and overall health.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2+
Sex:All
71 Participants Needed
Image of trial facility.

RCT2100 for Cystic Fibrosis

Pittsburgh, Pennsylvania
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 65
Sex:All
158 Participants Needed
The study will evaluate the feasibility of using smartphone speakers and microphones to evaluate the caliber of the airways, detect airway obstruction, aid in airway disease diagnosis, and identify disease exacerbations.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:8 - 65
Sex:All
800 Participants Needed
The aim of this study is to assess the utility of CGMs to determine the optimal method to dose meal-time insulin. The investigators will examine glucose excursions in patients with CF who will dose meal-time rapid-acting insulin by carbohydrate counting versus fixed-dose rapid-acting insulin. The carbohydrate ratio and fixed doses will be determined by existing doses, total daily insulin doses, body weight, and insulin sensitivity along with predisposition to hypoglycemia. Bolus insulin dosing is an important part of CFRD management due to the high nutritional demands of these patients. If dosed incorrectly, this could lead to marked hyperglycemia and could worsen nutritional status due to urinary glucose losses. In this project, the investigators will perform a within-subjects' comparison of the 2 standard methods of meal-time rapid-acting insulin dosing.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:18 - 80
Sex:All
13 Participants Needed
Image of trial facility.

Volara System for Cystic Fibrosis

Pittsburgh, Pennsylvania
This is a small pilot study with the goal of identifying a superior sputum collection method in Cystic Fibrosis patients unable to produce a sputum. Participants will use the Volara System during clinic visit in an attempt to produce sputum.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
20 Participants Needed
This study is a randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of ensifentrine inhalation suspension (3 mg) delivered twice daily via standard jet nebulizer over at least 24 weeks, compared to placebo, in subjects with non-cystic fibrosis bronchiectasis (NCFBE).
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18 - 85
Sex:All
180 Participants Needed
To evaluate the micro-biologic efficacy and safety of a streamlined treatment for early onset methicillin-resistant staphylococcus aureus (MRSA) in patients with cystic fibrosis.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 45
Sex:All
42 Participants Needed
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
12 Participants Needed
Image of trial facility.

ARCT-032 for Cystic Fibrosis

Northfield, Illinois
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
12 Participants Needed
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Frequently Asked Questions

How much do Cystic Fibrosis clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Cystic Fibrosis clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Cystic Fibrosis trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Cystic Fibrosis is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Cystic Fibrosis medical study ?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Cystic Fibrosis clinical trials ?

Most recently, we added Rifaximin for Bloating in Cystic Fibrosis, ARCT-032 for Cystic Fibrosis and Antibiotic Treatments for Cystic Fibrosis to the Power online platform.