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What is a clinical trial?
Please see our clinical trial brochure.
What are some types of research?
Pre-clinical Research and Development (done in a laboratory, does not involve human subjects)
Basic, in vivo, in vitro
Clinical Research and Development
Studies in human subjects looking at new drugs or devices or new ways to use existing drugs or devices in human subjects,
Post Marketing Surveillance
Case Studies, Historical, Statistical, Behavioral, Registries
Who puts together a trial?
Clinical trials are sponsored by government agencies such as the National Institutes of Health; pharmaceutical and biotechnology companies; individual physician-investigators; health care institutions such as health maintenance organizations (HMOs); and organizations that develop medical devices or equipment.
Drug trials are often organized and paid for by pharmaceutical (drug) companies or device (products) companies. Doctors and their professional staff will carry out the clinical trial in a variety of locations such as hospitals, universities, doctors’ offices or community clinics.
Doctors may also receive funding through private grants, donations, or government agencies to do “Investigator Initiated Clinical Trials.” These types of research are written by the doctor.
Are they safe?
If your child takes part in a clinical trial, some kind of intervention which your child would not otherwise have will be done. This may make your child feel better, have no effect, or it could possibly cause side effects. Most of the time these side effects are likely to be very mild, but in rare cases they could be serious.
If your child is asked to take part in a clinical trial involving an experimental drug or device,, make sure you are told what phase of trial it is (see below). This will give you a better idea of what is involved and the level of risk your child is taking. The researcher must tell you everything they currently know about possible side effects and then the decision whether to take part will be yours and your child’s.
Types of Pharmaceutical clinical trials
Phase 1 trials
In Phase 1 clinical trials, researchers test a new drug in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. This research is often done first with healthy adult volunteers.
Phase 2 trials
If a new treatment can be shown to be safe after a phase 1 trial, it will continue to phase 2. In Phase 2 clinical trials, the study drug is given to a larger group of people (40-100) to see if it might be effective and to further evaluate its safety.
Taking part in a phase 2 study has fewer risks than phase 1. By this point researchers will know a lot more about the effects of the drug on humans and will be ready to test it on a larger group of patients or healthy volunteers.
During a phase 2 study researcher will be looking at:
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Minor side effects of the drug.
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How well the drug works.
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And possibly, alternative ways of giving the drug ( eg injection versus tablets)
Phase 3 trials
In Phase 3 studies, the study drug is given to large groups of people (more than 200) to further determine its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug to be used safely. Much more is now known about the new drug. For this reason these trials involve a larger number of subjects, often 100's or even 1000's, from many different hospitals.
The main purpose of phase 3 studies is to compare the new drug with the most commonly used existing treatment.
Researchers may want to find out:
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Whether the experimental drug is more effective than the existing treatment.
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Whether the experimental drug has more or fewer side effects than the existing treatment.
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The long term benefit of the experimental drug to a subject.
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Whether a subject’s quality of life is changed by the experimental drug.
Phase 3 studies are always randomized. This means splitting patients into two or more groups, each of which will receive a different intervention. The purpose is to compare the new drug with existing treatments for the same condition.
Phase 4 trial
Phase 4 studies are done after the drug has been approved to be marketed by the Food and Drug Administration (FDA). These studies continue testing the study drug to collect information about its effect in various populations and any side effects associated with long-term use.
Once the three phases of clinical trial are complete, the FDA reviews information from all the studies to see if the drug can be approved. If the FDA approves it the new medication will go on sale. At this point the pharmaceutical company might fund a phase 4 trial. The main purpose of this type of trial is to promote the drug and bring it to the attention of more doctors. There are few risks involved in a phase 4 trial because much is already known about the treatment. This kind of trial is of less scientific importance than earlier phases.
What is a protocol?
All clinical trials are based on a set of rules called a protocol. A protocol is a scientific paper that describes what types of people may participate in the trial, the schedule of tests, procedures, medications, dosages, and the length of the study.
What is informed parental permission and child assent?
Informing parents and children of the necessary facts about a clinical trial before deciding whether or not to participate is the most important part of a study. A form will be given to you to explain things like:
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Why the research is being done
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What the researchers want to accomplish
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What will be done during the trial and for how long
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What risks are involved in the trial
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What benefits can be expected from the trial
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What other treatments are available
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The fact that you have the right to leave the trial at any time
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What identifiable information will be used and who will have access to it
If you are considering having your child join a clinical trial, the research staff will give you and your child, if he/she is old enough, an informed permission/assent document that includes the details about the study. Because joining a clinical trial is an important decision, you and your child should ask the research team any questions you may have about the study and the forms before a decision is made. They need to answer the questions to your satisfaction.
It is also a good idea to take the permission/assent documents home and discuss them with family members or friends. Talking about the options can help you and your child to feel comfortable with the decision. If you decide for your child to join the clinical trial, be sure the staff provides you with a copy of the signed form you sign so you and your child may use it as a reference during the trial.
Informed parental permission and child assent is more than signing a form. It is a process that continues through the study. You and your child are free to ask the research team questions before, during, and after the study.
What is a Placebo?
A placebo is an inactive substance that has no treatment value. In drug studies they are known as “sugar pills.” In clinical trials, experimental drugs are often compared with placebos to assess the effectiveness of the experimental drug. In some studies, the participants in the control group will receive a placebo instead of an active drug.
What is a control or control group?
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of subjects will be given an experimental drug and the control group is given either a standard treatment for the illness or a placebo.
What is a blinded, or masked, study?
A blinded or masked study is one in which participants do not know whether they are in the experimental or control group in a research study. Those in the experimental group get the medications being tested, and those in the control group get a standard treatment or no treatment. The study staff usually knows what group each subject in the study is in.
What is a double-blind, or double-masked, study?
A double-blind or double-masked study is one in which neither the participants nor the study staff know which participants are receiving the experimental drug and which ones are receiving either a standard treatment or a placebo. These studies are performed so that neither the patients’ nor the doctors’ expectations about the experimental drug can influence the outcome.
For safety, a member of the scientific team at the company that is carrying out the protocol, known as the Medical Monitor, does know what each participant is on. If there is a question and the study doctor needs to know which group a subject is in, this information can be “un-blinded” under very strict rules by that safety member.
What are side effects and adverse reactions?
Side effects are any undesired actions or effects of a drug or treatment. Negative or adverse effects may include minor events such as a headache, nausea, hair loss, skin irritation, or major events such as liver or kidney damage or other physical problems. Experimental interventions must be evaluated for both immediate and long-term side effects.
What are the benefits and risks associated with clinical trials?
There are both benefits and risks associated with clinical trials. By participating in a clinical trial, your child can:
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Take an active role in improving health care for children
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Have access to experimental drugs that are not otherwise available
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Help others by contributing to medical research
Clinical trials have risks:
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There may be side effects or adverse reactions that are not known yet to drugs
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The protocol may require a lot of your time for trips to the study site, study interventions, hospital stays or complex dosage requirements
Can My Child leave a clinical trial after it has begun?
Yes. Your child can leave a clinical trial at any time. If you plan for your child to stop participating, let the research team know why.