The idea for a clinical trial often starts in the lab and requires years of development. As new treatments move through a series of trials called “phases”, researchers learn more information about the treatment, its safety, and its effectiveness. If the drug is eventually approved and made widely available, safety monitoring is continued.
Researchers investigate and discover potential new treatments in the laboratory.
Researchers investigate the efficacy and safety of the treatment without the use of human subjects.
Confirmation of efficacy and safety in humans.
In Phase I of clinical trials researchers test a treatment in a small group of people, typically healthy adult volunteers, for the first time. The purpose is to learn about safety and side effects.
In Phase II researchers study the new medicine or treatment in a larger group of people to further determine safety, effectiveness, and optimal dosage.
In Phase III the new treatment is given to a larger group of participants to confirm its effectiveness, monitor side effects, compare it with standard or similar treatments or a placebo, and collect information that will allow the new medicine or treatment to be used safely.
Filing of an application for approval of the new treatment for the affected community. In the U.S., this is done through the Food and Drug Administration (FDA). Other countries follow a similar development process, though trial results and approvals in other countries may speed up the process in some cases.
In Phase IV, researchers track the safety of the treatment in the general population, seeking more information about the treatment’s benefits and optimal use.
The main event or outcome that is measured to determine whether the therapy being studied is beneficial.
An outcome is an observable or measurable change that is monitored during treatment for change. A core outcome set (COS) is a group of outcomes measured across multiple trials that reflect measures important to the patient. The ACD has established a Core Outcome Set (COS) for GAMT and CTD trials based on input and consensus from both the GAMT and CTD patient and healthcare/research communities. The COS and more information on the development of the set can be found at creatineinfo.org/cos. It is strongly encouraged that all trials include these important outcomes for comparability between trials and to ensure that the outcomes of most importance to the patient are measured.
When the U.S. Food and Drug Administration (FDA) determines that the benefits of the therapy being tested outweigh the known risks. An FDA approval allows for the intended use of a therapy.
The use of a drug for a purposes other than its approved use which carries potential unmeasured risks.
The therapy or process that the study focuses on.
The drug being investigated in a clinical study. Investigational drugs are not yet approved.
Inclusion criteria are defined as the key features of the target population that the investigators wish to recruit to participate in their clinical trial. In contrast, exclusion criteria are defined as features of the potential study participants who present with additional characteristics that could interfere with the success of the study.
Blinding describes the process in which the patient (blind) or both patient and investigator (double blind) do not know if the treatment or a placebo is being given. Blinding prevents members of the research team and study participants from influencing the results. Blinding allows the collection of scientifically accurate data.
Randomization is the process by which treatments are assigned to participants by chance rather than by choice. This is done to avoid any bias in assigning volunteers to get one treatment or another.
The informed consent is a document given to participants and individuals thinking about participating in a study. This document outlines important information about the study including the benefits and risks.
An administrative body designed to make sure that all clinical studies are ethical and that the rights and welfare of the study participants are protected. This includes making sure that potential benefits of the study outweigh the risks and in addition, risks are minimized. The IRB also reviews the informed consent.
A document that describes the study’s background, objectives, and specific research questions.
The primary individual responsible for the clinical study; also known as the lead researcher.
The entity that funds the study. Sponsors can be pharmaceutical companies, academic medical centers, voluntary groups, and other organizations.
A clinical trial is a research study where human volunteers receive a new treatment- a drug, device or procedure- to test if the new treatment is safe and effective.
Clinical trials follow a very carefully planned set of procedures and are described in a document called a protocol. Before patients can be enrolled in a study, the protocol must be reviewed by a panel that includes doctors as well as other members of the community. Any changes to the protocol must also be reviewed and approved by this panel before they can be made.
There may be risks associated with your child’s participation in a clinical trial. The new drug, device or procedure has been tested in the lab and may have been tested in animals to determine how much risk might be involved. Some side effects may be known but some may be unknown. Each child may react differently to a medication or procedure. Also, your child may have to come off his or her usual medication and there could be a risk associated with that. All known risks will be described in a document called an informed consent form. You will be provided with this form.
If you are thinking about taking part in a clinical trial, you should bring up any questions you may have with the researcher and your physician. You should also feel free to discuss any issues throughout the trial. The following questions may give you some ideas of topics to discuss:
The study
Possible benefits and risks
Participation and care
Cost issues
There is no guarantee that your child will benefit from participating in a clinical trial. With a new study medication, you may find that your child’s condition is better controlled or that he or she is experiencing fewer side effects. But this may not occur. Clinical trials sponsored by pharmaceutical companies often provide free medication and study-related care. There are many reasons why parents enroll their children in clinical trials. Often parents say that they want to help other children with the same disease or disorder.
You will be told about the study, including any possible benefits and any known risks, before you decide if a clinical trial is right for your child. In addition, the informed consent will explain what will happen during the trial, how long it will last, how often you will be asked to come to the hospital and how long the visits will last.
You will be given the opportunity to ask your doctor any questions about the trial.
Participation in a clinical trial is voluntary. Only you can decide what is best for your child. No matter what you decide, your child will continue to receive the same high-quality medical care from his or her doctors and nurses. There is no right or wrong decision when it comes to enrolling in a clinical trial.
Before being enrolled in a clinical trial, you will be asked to sign an informed consent form. You may change your mind at any time. Should you decide to withdraw your child from a clinical trial, he or she will continue to be treated by his or her doctor, without prejudice.
Clinical trials have different purposes. The purpose helps define the type of trial it is. On this webpage, we mostly discuss “Treatment trials.”
Some types of trials include:
Clinical trial designs often include some of these standard elements:
*Participants in blinded and randomized studies are eventually informed of the treatment they were given (placebo vs drug). This usually occurs sometime after the study is completed and the data is analyzed.
There is no guarantee that a clinical trial will continue. Researchers carefully examine information collected during a trial before continuing to the next Phase.
After Phase I and again after Phase II of a clinical trial, researchers decide whether to move on to the next phase or to stop testing the treatment because it was unsafe or not effective. If the study continues and a Phase III trial is completed, the researchers examine the information and decide whether the results have medical importance.
Once a new treatment has been proven safe and effective in a clinical trial, it may become a new standard of care. In many cases, if you participated in a blinded or masked study, you will get information about the treatment you received.
Published study results are typically available by searching for the study’s official name or Protocol ID number in the National Library of Medicine’s PubMed® database.
A clinical trial may have guidelines restricting participation in an additional clinical trial. In other words, you may be ineligible to participate in two trials at the same time. You may also need to wait for a period of time known as a “washout period” after ending participation in one trial before being eligible to enroll in a new clinical trial. This will be determined by the trial design of each study.
The following video from Children Hospital of Philadelphia (CHOP) is a patient and family town hall discussing clinical trials, the vital role patients have played in improving treatments, and the often difficult decision patients and families of whether to participate in a trial. The original posting can be found here.
Clinical studies are necessary to move treatments forward. Your participation in clinical studies may provide a valuable contribution to new treatment options for both you and future generations affected by Cerebral Creatine Deficiency Syndromes.
