What Are Clinical Trials?

A clinical trial is a medical research study involving volunteer participants. Clinical trials are a standard research practice and are done on all medications before they are approved by the Food and Drug Administration (FDA).  Some trials evaluate the effects of new or existing medications. Some may study new forms of interventions such as speech therapy, psychotherapy (talk therapy) or the use of assistive technologies such as computer-assisted learning devices.  Other trials may study a combination of therapies.  Clinical trials are most often conducted by universities, but may also be conducted by government agencies or private companies.  Many trials are funded by governmental and/or pharmaceutical company grants.

Clinical trials follow a structured plan developed before the trial begins.  Most trials have an Institutional Review Board (IRB) that is comprised of doctors, university representatives, patient advocates and others, which is in place to help ensure patient safety and to make sure the study is conducted ethically and within certain guidelines.  KS&A encourages all patients (or the parents of patients who are minors) to ask if a trial you are considering has been reviewed by an IRB.

It is important to understand that the objective of a clinical trial is research, not patient care. This does not suggest that trials are designed to harm people, in fact most clinical trials are carefully designed with patient safety in mind.  However, individuals participating in the trial cannot be guaranteed of receiving any new treatments. Some individuals may not receive any treatment at all. The contribution made by participating in a clinical trial is to science first and to the patient second.

During a Trial

In a typical clinical trial evaluating medications, one group receives a new treatment, while a second control group receives a treatment already in use.  In some trials, another group will receive a placebo – a sugar pill that has no medicine in it.  Most of the time, participants do not know which they are receiving.

Other clinical trials involve a “crossover” design, where participants are randomly assigned to take a new treatment, a treatment already in use, and/or a placebo for a specified time period. When that portion of the study ends, participants “crossover” to one of the remaining treatments for another specified time period.

In trials that test new forms of psychotherapy, interventions and assisted technology, one group may be randomly assigned to a new form of therapy and another group may receive a standard method or a combination of that method and medication.

Benefits of Participation

Any decision to participate in a clinical trial should be made after discussing the pros and cons with your doctor. People participate in clinical trials for a variety of reasons, including:

•   A desire to help advance scientific knowledge about X and Y chromosome conditions and their treatments.



•   In the majority of studies, participants are not charged for medications, visits or tests.



•   There may be the chance to receive the attention of multiple medical experts or to receive care at a well-respected medical facility.



•   Where new medications are involved, the medication being tested may lack the side effects of the medication you are currently taking.



•   It could be an opportunity to try a new medication before FDA approval.  However, there is no guarantee that you will receive the new medication in the trial.

Drawbacks of Participation

Every clinical trial involves a trade-off. Remember that any decision to participate in a clinical trial should be made in consultation with your doctor. Some drawbacks of participating in clinical trials include:

•   There is no guarantee you will receive a new medication or that the medication you receive will be better than something you may already be taking.



•   Some clinical trials do not permit the use of medications other than those administered as part of the study. You may have to stop taking medications you are currently taking.



•   Some trials involve hospitalization or numerous hospital visits for laboratory tests.



•   Some trials last a long time, ranging from several months to several years.

Requirements for Participation

Admission into a research study or clinical trial is based on a rigid set of requirements (generally more in the instance of a clinical trial).  For example:

•   You must be diagnosed with the symptoms associated with the condition that the treatment under study may remedy.



•   Sometimes admission requirements will specify a particular subtype of an illness or certain symptoms. You can be severely ill and fail to qualify for a clinical trial if you do not meet these requirements.



•   Excessive alcohol intake or “street” drug use may disqualify an applicant from participation.

Informed Consent

You will be asked to sign an informed consent form after you discuss the clinical trial with study personnel. Carefully read the entire form and ask questions if there is anything you do not understand. KS&A encourages you to request the informed consent form in advance so you can take your time to become familiar with its contents and discuss it with others, if necessary, or alternatively to take the consent form home so that you can consider it overnight before executing it.

Informed consent is not a one-time event, but a continuing process. Throughout a study, the research team must provide information about your participation in the study. They must respond to any questions you have about the research and inform you if any new risks are identified. At any time you may withdraw your consent and end your participation in the study.

The informed consent process must include the following items:

•   A through description of the research study or clinical trial, including all the possible benefits and risks that may be involved. This is done in person with a researcher and in writing.



•   The reasons why you may be dropped from the study and how you can voluntarily leave the study.



•   A list of any charges you may have to pay to take part in the research.



•   A description of what kind of treatment you will receive following the trial.



•   A statement that you will be told of any important results of the research which may help you to decide whether to continue taking part in the study.

Questions to Ask

Before agreeing to participate in a research study or clinical trial, ask questions.  Understand the study’s protocol (i.e., the plan to be followed).  It’s crucial you feel comfortable with the protocol because, to protect the scientific integrity of a trial, straying from protocol is seldom allowed.  Be sure you completely understand what is involved in the study before you begin.  Do not be afraid to ask questions before you begin or anytime during the trial.  Questions you may want to consider asking include:

•   What treatment is the clinical trial studying?



•   Who is paying for the trial?



•   How long will the trial last?



•   Where will the trial take place? Is hospitalization required?



•   Will I have to discontinue any present medication(s) and/or supplements? If so, could this harm my health?



•   If I can stay on my present medication(s) and/or supplements, will taking experimental medication be bad for my health?



•   If I am involved in a “crossover” clinical trial, can I go back to the first treatment I received if it worked better than the second treatment?



•   Can I continue to see my own doctor while participating in the trial?



•   Will everyone involved in the clinical trial get the same treatment? If not, what chance do I have of receiving an existing treatment or placebo?  What else will I have a chance of receiving?



•   How much time will I have to commit to participate in the clinical trial? How often will the study personnel need to see me? How long will the visits be? What time of day will I need to schedule appointments? Do these need to be in person, or can at least some be conducted by telephone?



•   What are the possible side effects and/or risks? What will be done if I experience side effects during the clinical trial?



•   If I don’t receive a new treatment during the clinical trial, will I have an opportunity to try it at the end of the trial?
 


•   If I receive the new treatment and it helps me, will I be able to use it after the clinical trial ends?



•   Are there any fees associated with the study? Will I be paid for my participation? Will I be reimbursed for any related costs I incur?



•   What procedures are in place to ensure my confidentially?



•   Will I be promptly informed of any findings that might make me want to drop out? For example, if the experimental medication can make me ill.



•   Can I drop out at any time, for any reason?



•   What kind of follow-up care will I receive after the trial is completed?



•   Can I sue if I suffer injury?

Where to Look for Relevant Research Studies and Clinical Trials

First, ask your physician if he or she is aware of any research studies and/or clinical trials that relate to your condition and/or symptoms.  Research organizations conduct clinical trials in most cities.  Some research studies and clinical trials are identified on this website.

For another source, call the Office of Public Inquiries at the National Institute of Mental Health (NIMH), (301) 443-4513, or visit the NIMH’s web site.

Ads for clinical trials appear in newspapers, magazines, on billboards, the radio and television. These ads usually do not adequately describe the trials, but usually give you contact information. Be sure to review possible risks and benefits with your doctor, family and friends before participating in any research study or clinical trial.

Disclaimer

KS&A does not endorse nor recommend any particular research study or clinical trial.  Patients should discuss all options with their health care providers and family members before beginning any study or clinical trial.

Many people think that participating in a research study or clinical trial means they will get better treatment for their condition. While this may be true, it’s also important to remember that such studies and clinical trials are conducted for research purposes, and does not ensure better or safer treatment. Taking part in a research study or clinical trial does not guarantee individual benefits to the participant in the form of newer or safer treatment. The contribution made by participating in a such a study or in a clinical trial is to science first, and to the patient second. KS&A, its Scientific Advisory Committee members, Board of Directors, executive officers, staff and advisors do not endorse or recommend the use of any specific treatment or medication. For advice about specific treatment or medication, patients (or in the case of minors, an adult family member on the patient’s behalf) should consult their physicians and/or health professionals.