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Information for Parents

About Clinical Trials

Before widespread use, a drug or intervention is carefully studied for its effectiveness in treating a patient's condition. Here are some basic questions about what clinical trials are, and what is involved in the process of conducting a clinical trial:

What is a clinical trial?
What are the different types of clinical trials?
What happens during a clinical trial?
Who can participate in a clinical trial?
What are the benefits of participating in a clinical trial?
 Why is it important for children to participate in clinical trials?
What are side effects and adverse reactions?

What are the risks of participating in a clinical trial?
What is a protocol?
What is informed consent?
How is the safety of the participant protected?
Can a participant leave a clinical trial after it has begun?
How are clinical trials conducted at Kennedy Krieger Institute more thorough?

What is a clinical trial?

A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. There are two main types of clinical studies: clinical trials and observational studies. ClinicalTrials.gov includes both interventional and observational studies.

In a clinical trial (also called an interventional study), participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants' behavior, for example, diet.

In an observational study, investigators assess health outcomes in groups of participants according to a protocol or research plan. Participants may receive interventions, which can include medical products, such as drugs or devices, or procedures as part of their routine medical care, but participants are not assigned to specific interventions by the investigator (as in a clinical trial). For example, investigators may observe a group of older adults to learn more about the effects of different lifestyles on cardiac health.

Source: ClinicalTrials.gov

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What are the different types of clinical trials?

  • Natural history studies provide valuable information about how disease and health progress.
  • Prevention trials look for better ways to prevent a disease in people who have never had the disease or to prevent the disease from returning. Better approaches may include medicines, vaccines, or lifestyle changes, among other things.
  • Screening trials test the best way to detect certain diseases or health conditions.
  • Diagnostic trials determine better tests or procedures for diagnosing a particular disease or condition.
  • Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
  • Quality of life trials (or supportive care trials) explore and measure ways to improve the comfort and quality of life of people with a chronic illness.

Source: NIH.gov

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What happens during a clinical trial?

The clinical trial process depends on the kind of trial being conducted. Every research team includes physicians or researchers, nurses, social workers, and other healthcare professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed. Some clinical trials involve more tests and visits to a physician than the participant would normally have for an illness or condition. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.

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Who can participate in a clinical trial?

All clinical trials have guidelines about who can participate called inclusion/exclusion criteria. Factors that allow someone to participate in a clinical trial are "inclusion criteria." Those that exclude or not allow participation are "exclusion criteria." These criteria are based on factors such as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy volunteers.

Source: NIH.gov

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What are the benefits of participating in a clinical trial?

Well-designed and well-executed clinical trials provide the best approach for participants to:

  • Play an active role in their health care.
  • Gain access to new research treatments before they are widely available.
  • Receive regular and careful medical attention from a research team that includes doctors and other health professionals.
  • Help others by contributing to medical research.

To decide whether a clinical trial may be right for you or your child, talk to your doctor, or visit our Information for Participants page for questions to consider before participating.

Source: NIH.gov

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Why is it important for children to participate in clinical trials?

Children are not little adults, yet they are often given medicines and treatments that were only tested in adults. There is a lot of evidence that children’s developing brains and bodies can respond to medicines and treatments differently than how adults respond. The way to get the best treatments for children is through research designed specifically for them.

We have already made great strides in improving children's health outcomes through clinical research. Vaccines, treatments for children with cancer, and interventions for premature babies are just a few examples of how this targeted research can be helpful. However, there are still many questions to answer and more children waiting to benefit.

To learn more about the importance of clinical trials for children, go to the National Institute of Health’s (NIH) Children and Clinical Studies page.

Source: NIH.gov

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What are side effects and adverse reactions?

Side effects are any undesired actions or effects of the experimental drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

Source: NIH.gov

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What are the risks of participating in a clinical trial?

  • There may be unpleasant, serious, or even life-threatening side effects to experimental treatment.
  • The study may require more time and attention than standard treatment would, including visits to the study site, more blood tests, more treatments, hospital stays, or complex dosage requirements.

Source: NIH.gov

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What is a protocol?

Clinical research is conducted according to a plan known as a protocol. The protocol is carefully designed to safeguard the participants’ health and answer specific research questions. A protocol describes the following:

  • Who is eligible to participate in the trial
  • Details about tests, procedures, medications, and dosages
  • The length of the study and what information will be gathered

A clinical study is led by a principal investigator (PI), who is often a doctor. Members of the research team regularly monitor the participants’ health to determine the study’s safety and effectiveness.

Source: NIH.gov

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Informed consent is the process of learning the key facts about a clinical trial before deciding whether to participate. The process of providing information to participants continues throughout the study. To help someone decide whether to participate, members of the research team explain details of the study. The research team provides an informed consent document, which includes such details about the study as its purpose, duration, required procedures, and who to contact for various purposes. The informed consent document also explains risks and potential benefits.

If the participant decides to enroll in the trial, the informed consent document will be signed. Informed consent is not a contract. Volunteers are free to withdraw from the study at any time.

Source: NIH.gov

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How is the safety of the participant protected?

Each clinical trial in the United States must be approved and monitored by an Institutional Review Board (IRB) to ensure that the risks are minimal and are worth any potential benefits. An IRB is an independent committee that consists of physicians, statisticians, and members of the community who ensure that clinical trials are ethical and that the rights of participants are protected. Federal regulation requires all institutions in the United States that conduct or support biomedical research involving people to have an IRB initially approve and periodically review the research.

Source: NIH.gov

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Can a participant leave a clinical trial after it has begun?

Yes, a participant can leave a clinical trial, at any time. However, the participant should let the research team know when withdrawing from the study, and their reasons for leaving.

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How are clinical trials conducted at Kennedy Krieger Institute more thorough?

Kennedy Krieger’s Clinical Trials Unit provides a safe environment for testing the effectiveness of new treatments for a wide range of diseases, injuries, and disorders. Our expertise in the underlying neurobiology of a child’s developing brain means that we have unique insight into understanding the effects of interventions on children with neurodevelopmental disabilities. This allows us to plan and conduct trials that are more methodical, and also ensure the well-being of our research subjects during the trials. 

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For more detailed information about clinical trials, visit our Information for Participants page.

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