Learn about cystic fibrosis, a genetic disorder that affects the lungs, pancreas, and other organs, and how to treat and live with this chronic disease.
CF is a rare genetic disease found in about 30,000 people in the U.S. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make informed decisions about your health care.
If you or your child has just been diagnosed with cystic fibrosis, or your doctor has recommended testing for CF, you may have many questions.
Diagnosing CF is a multistep process. A complete diagnostic evaluation should include a newborn screening, a sweat chloride test, a genetic or carrier test, and a clinical evaluation at a CF Foundation-accredited care center.
Raising a child with cystic fibrosis can bring up many questions because CF affects many aspects of your child’s life. Here you’ll find resources to help you manage your child’s daily needs and find the best possible CF care.
Living with cystic fibrosis comes with many challenges, including medical, social, and financial. By learning more about how you can manage your disease every day, you can ultimately help find a balance between your busy lifestyle and your CF care.
People with CF are living longer, healthier lives than ever before. As an adult with CF, you may reach key milestones you might not have considered. Planning for these life events requires careful thought as you make decisions that may impact your life.
People with cystic fibrosis are living longer and more fulfilling lives, thanks in part to specialized CF care and a range of treatment options.
Cystic Fibrosis Foundation-accredited care centers provide expert care and specialized disease management to people living with cystic fibrosis.
We provide funding for and accredit more than 120 care centers and 53 affiliate programs nationwide. The high quality of specialized care available throughout the care center network has led to the improved length and quality of life for people with CF.
The Cystic Fibrosis Foundation provides standard care guidelines based on the latest research, medical evidence, and consultation with experts on best practices.
As a clinician, you’re critical in helping people with CF maintain their quality of life. We’re committed to helping you partner with patients and their families by providing resources you can use to improve and continue to provide high-quality care.
As part of the Cystic Fibrosis Foundation's mission to help improve the lives of people living with cystic fibrosis, the PSDC initiative taps the CF community to inform key efforts to support the management of daily care.
Your cystic fibrosis care team includes a group of CF health care professionals who partner with you to provide specialized, comprehensive CF care.
Many cystic fibrosis patients and families face complicated issues related to getting the care they need. But CF Foundation Compass makes sure that no one has to do it alone.
For many people with cystic fibrosis, dealing with insurance is as much a part of living with the disease as nebulizers and vests. Many people with CF and their families face issues related to getting the care they need, but no one has to do it alone.
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for CF and supports a broad range of research initiatives to tackle the disease from all angles.
The CF Foundation offers a number of resources for learning about clinical trials and treatments that are being developed to improve the treatment of cystic fibrosis.
Our understanding of CF continues to evolve as scientists study what causes the disease and how it affects the body. These insights drive the development of new and better treatments and bring us one step closer to a cure.
Researchers, supported by the CF Foundation, have made tremendous advances to improve the health and quality of life of people with CF. We are committed to providing the tools and resources you need to continuously build upon this work.
Each clinical trial has a study sponsor and a protocol. Potential drugs must move through four well-defined phases of testing in patients.
The trial sponsor, often the pharmaceutical company that develops the therapy or medication, prepares the protocol for the clinical trial. A protocol is a plan that explains
how the trial will work, what will be done during the trial, and why. Each medical center that conducts the trial uses the same protocol.
Key information in a protocol includes:
Study-related medical care is often provided to the patient at no cost.
Clinical researchers work to ensure that they avoid bias in clinical trials. Bias refers to human choices or other factors (unrelated to the protocol) that might affect the trial's results. For example, if doctors could choose which patients to assign
to comparison groups in a study, some might assign sicker patients to the treatment group and healthier patients to the control group (not receiving treatment). The doctors might not even realize they are doing this, and it could affect trial results.
Randomization helps ensure that researchers don't introduce bias into the trial. In many clinical trials that test the effectiveness of a medication, half of the participants receive the medication in question. The other half receive a placebo, which
contains no medication. Randomization involves assigning patients to these comparison groups by chance, rather than choice.
Researchers also may use “blinding” to help avoid bias. In a blinded trial, researchers will not know which patients are receiving treatment and which ones are receiving a placebo.
For any new drug to receive approval by the U.S. Food and Drug Administration (FDA) and become available to the public, it must pass through three phases
of interventional clinical trials to show that it is safe and effective in treating the disease. If the FDA approves the drug, it will continue to be monitored for safety and effectiveness in what is known as a Phase 4 study.
The infographic below shows a breakdown of the questions that researchers try to answer, the number of participants needed, and the time per participant for each phase of research.
Please note that the length of time refers to the time it takes to participate in a trial, not the entire length of the phase. It takes time to enroll participants and to process
and analyze the results of each phase. All told, it typically takes 10 to 14 years from the time a drug is discovered in a laboratory to its possible approval by the FDA for people with CF.
All clinical trials have guidelines about who can join. Some enroll healthy people. Others enroll only people with certain conditions, such as CF.
A CF clinical trial may have other guidelines in addition to requiring that the study volunteer has CF. These guidelines are known as inclusion criteria and exclusion criteria, and they ensure that the research results are reliable and help to reduce
the risk for those enrolling in the trial.
Inclusion criteria and exclusion criteria refer to the standards used to decide whether a person may or may not enroll in a clinical trial. Criteria are based on such factors as age, gender, disease, previous treatment history, and other medical conditions.
The criteria depend on the type of trial. For example, the age that the person with CF must be to participate varies according to each trial because drugs work differently in young children than they do in adults. Before a drug can be tested in young
children, it must be shown to be safe and effective in adults with CF.
If researchers are testing how well a particular antibiotic works in fighting Pseudomonas aeruginosa, then the trial would have inclusion criteria
specifying that only people with CF who are infected with that bacterium can join the trial. Those who do not have that bacterial infection would be excluded from the trial.
call people with CF who participate in clinical trials, and all those who
support them, trailblazers. Help us
blaze a trail to better treatments and a cure for CF.
Clinical research can be sponsored (i.e. paid for) in part or entirely by any number of organizations or individuals. For example, medical institutions, universities, foundations, voluntary groups, drug companies, and federal agencies, such as the
National Institutes of Health (NIH), all sponsor research.
The sponsor chooses principal investigators to run the trials. Study-related medical care is often provided to the participant at no cost.
The Cystic Fibrosis Foundation is the primary supporter of CF research in the United States. Nearly all approved CF therapies available today were made possible because of research funded by the CF Foundation.
The Foundation facilitates and financially supports clinical trials through its
Therapeutics Development Network (TDN), which is made up of CF care centers with an expertise
in clinical research.
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