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FAQs About Clinical Trials

Why participate in a clinical trial?

About-Clinical-Trials
Click here for a printer-friendly guide to CF clinical trials. It is also available in Spanish.

People with CF choose to participate in clinical trials for a number of different reasons. Some possible benefits include:

  • Taking an active role in managing your own CF care.
  • Gaining access to new treatments before they are more widely available.
  • Getting expert CF care at CF Foundation-accredited health care centers.
  • Helping advance our knowledge of CF.
  • Receiving a treatment that works for you or your child.


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Who can participate in a clinical trial?

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 help ensure that the research results are reliable.

For example, the age that the person with CF must be to participate varies according to each trial and depends on a number of factors, with safety always first in mind. 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 older people with CF.

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What are the different phases of clinical trials?

It typically takes 10 to 14 years from the time a drug is discovered in a laboratory to its possible approval by the U.S. Food and Drug Administration (FDA) for people with CF. This lengthy process occurs in phases, which have different purposes and help scientists answer important questions.

In Phase 1 trials, researchers test an experimental drug or treatment in a small group of people to 1) learn if it is safe, 2) find a safe and tolerable dosage range, 3) decide how to administer the drug (for example, orally, intravenously, by inhaling) and 4) learn the side effects.

In Phase 2 trials, the experimental drug or treatment is given to a larger group of people to see how well it works (also called efficacy) and to keep testing its safety. At this stage, the trials do not provide enough information to know whether or not a drug works to treat an illness, but people receiving the drug may report some benefits. Phase 2 trials can take up to two years.

In Phase 3 trials, the experimental drug or treatment is given to large groups of people to 1) continue testing how well it works, 2) determine dosage amounts, 3) watch for side effects, 4) compare it with commonly used treatments and 5) collect information regarding its safety. After a Phase 3 study is completed, the sponsor of the clinical trial reviews the results and decides whether to submit a new drug application to the FDA for approval. The FDA may take several months or longer to decide whether to approve the new drug.

After the FDA approves a drug and it becomes available for people to begin to use, Phase 4 trials may be conducted to gather more information on the drug’s effect in various patient populations and any side effects associated with long-term use.

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What are some of the risks of participating in clinical trials?

Each phase of a clinical trial carries a risk that the drug might not work or may have unwanted side effects. Possible risks include:

  • Side effects of the medications or treatments being studied.
  • Unwanted events during the trial that may or may not be related to the study drug.
  • Failure of a treatment to work.

The research team will continuously monitor your or your child’s health and safety throughout the trial, whether you are receiving the drug being studied or a placebo.

If the research team notes any worsening of your health during the trial, they will notify your regular CF care team and the study sponsor to determine if it is related to the study drug. You should contact the study team if you notice any worsening of your or your child’s health or have any concerns during the clinical trial.

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How is my safety protected?

Nothing is more important than safety in developing new CF treatments. The U.S. government has strict guidelines and safeguards to keep risks as low as possible and ensure that the risks are worth any potential benefits. There are additional layers of protection in every CF clinical trial that help determine if the risks are safe and appropriate for people who participate.

  • The U.S. Food and Drug Administration (FDA) is the federal agency that must approve all clinical trial protocols and make sure all procedures are being followed as the trial goes on. When the trial is completed, the FDA determines whether the new drug or therapy will be approved for use by other people with CF.

  • The Institutional Review Board (IRB) is usually made up of doctors, other health care providers and the general public. They look at the trial’s protocol to make sure that participants’ rights are protected and the trial does not cause them unnecessary risk. IRBs can be within a hospital or university or work as independent groups.

  • The Data Safety Monitoring Board (DSMB) is an independent committee of experts in CF care that checks information on ongoing trials, watching for possible problems or unwanted side effects. The CF Foundation is the only voluntary health organization to organize a DSMB whose members are experts in CF and completely independent and not involved in any way with the trial, the sponsor or its participants. In this way, the CF Foundation does its best to keep participants safe throughout the clinical trial.

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How can I learn about CF clinical trials in my area?

Talk with your CF physician or a member of the care team, such as the CF research coordinator, about clinical trials. CF clinical trials are generally offered at CF Foundation-accredited care centers.

You also may want to check the Find A Clinical Trial section on this website, or call our toll-free Clinical Trials Hotline at 1-877-8CF-JOIN (1-877-823-5646). The National Institutes of Health also publishes a list of clinical trials at www.clinicaltrials.gov.

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What questions should I ask about participating in a clinical trial?

When you speak with your CF physician or the research coordinator at your care center, consider asking the following questions:

  • What is the purpose of the study?
  • What will be asked of me?
  • What will be my role in the study: healthy volunteer or participant?
  • Who will be in charge of my care?
  • Will the study benefit me?
  • Will the study benefit others?
  • Why do researchers think that this particular drug or treatment might work?
  • What kinds of tests and experimental treatments are involved?
  • How do the possible risks, side effects and benefits compare with my current treatment?
  • How might this study affect my daily life?
  • How long will the study last?
  • Will hospitalization be necessary?
  • Who will pay for my participation in the study?
  • Will I be reimbursed for any expenses?
  • How will I know if the experimental drug is working?
  • Will results of the studies be given to me?
  • What type of long-term follow-up care will be required?

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If I’m compensated for participating in a clinical trial, will that affect my eligibility for Supplemental Security Income (SSI) or Medicaid?

In some clinical trials, volunteers receive compensation for participating. This is a decision made by the trial sponsor. Some sponsors may offer compensation for your extra time and travel expenses needed to take part in the study.

Under the Improving Access to Clinical Trials Act (IACT) of 2011, clinical trial participants who receive Supplemental Security Income (SSI) or Medicaid benefits may accept up to $2,000 in research compensation without losing government medical benefits. To qualify for the exemption, SSI recipients must report any compensation received for participating in a qualifying clinical trial to the Social Security Administration.

If you are considering participating in a clinical trial, you will receive information about compensation during the informed consent process before the study begins.

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Additional Resources

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