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WHAT ARE CLINICAL TRIALS?
Clinical research is designed to answer specific questions about dysfunction and its treatment in humans. These questions may concern the cause of a medical or psychological condition or study the effectiveness and safety of a novel treatment or device.
Clinical research involves people who voluntarily give informed consent to serve as research participants without whom the research simply could not be done. Though volunteering for research does not guarantee a benefit, people who take part in clinical trials and studies make important contributions to the knowledge of, and progress toward, new treatments for Spinal Cord Injury (SCI) and its related problems.
A clinical trial is a carefully organized and controlled research study designed to test the safety and effectiveness of a novel treatment or device used for the first time for a particular medical or psychological condition.
The Miami Project conducts basic science research (laboratory setting) with the goal of making new discoveries and then translating them into successful clinical treatments for SCI through our Clinical Trials Initiative.
The Miami Project also carries out many clinical studies that address various problems associated with SCI, such as pain, spasticity, fatigue, fertility, fitness, rehabilitation, and other areas of concern for people with SCI. For more information, see Our Current Studies. Through this research, scientists strive to discover valuable information about SCI that helps in development of new treatments.
How are Clinical Trials Introduced?
Scientists may invest years in basic science research, first in discovery and design of a new treatment and then in testing through experiments that model human disease. Once investigators complete "proof of concept" experiments, which establish a scientifically sound understanding of how a treatment works and potential benefit, the preclinical phase of research can begin.
The preclinical phase involves further research into the treatment's benefit through experimental models but also evaluates whether the treatment has the potential to cause harm. Investigators explore questions about possible damaging effects, such as the formation of tumors or toxicity from higher doses of a drug. These safety studies are very important to achieve approval to begin testing a new treatment in a human clinical trial.
Approval to perform human research at a given institution is received from an Institutional Review Board (IRB). An IRB is comprised of faculty, staff, and community members who are charged with ensuring the design of the clinical research is ethical and that the rights of study participants are protected throughout the study. Some clinical research also requires the approval of a governmental regulatory agency, such as the Food and Drug Administration (FDA) in the United States. Before a new treatment can be marketed as a treatment for a particular condition in the United States, it must be proven safe and effective. The FDA is responsible for ensuring the safety and effectiveness of all drugs, biologics, vaccines, and medical devices.
Clinical Trial Phases
Once a new treatment is approved for first time use in a clinical trial, the treatment will undergo evaluation in a series of clinical trial phases.
A Phase 1 trial is the first step and is designed primarily to evaluate the safety of a drug or treatment in humans. Safe dosage range and side effects are identified. Usually, a Phase 1 trial involves a few healthy volunteers and takes place at only one or two locations, but can include those with the target condition. Investigators use the study results to decide on the best dose and refine procedures for use in further testing.
After completing a Phase 1 trial, investigators will decide whether to seek approval to continue with a Phase 2 trial. A Phase 2 trial will expand the study to a larger group of people with the target condition and compare the results to those of a control group. Those in the control group will receive either the standard treatment or a placebo treatment – an inactive substance. The major goal of Phase 2 is to decide if the treatment is effective and to evaluate further its safety. A few hundred people may participate.
If the results of Phase 1 and 2 are promising, a Phase 3 trial may be sought with an even larger and more diverse group of people with the target condition. The goal of this phase is to confirm effectiveness, monitor side effects, and collect information that will allow safe use of the drug or treatment. Investigators compare the results of the new treatment to that of a standard or placebo treatment. Typically, they will perform a randomized controlled trial in which research volunteers are assigned randomly to either a group that receives the new treatment or a control group that receives the standard or placebo treatment. Several hundred to thousands of people may take part.
The final clinical trial phase is a Phase 4 trial. A Phase 4 trial is conducted to evaluate the ongoing long-term safety and effectiveness of a treatment in large and varied populations and to study additional uses of the drug or treatment. This phase usually takes place after the treatment has been made available to doctors and been approved for standard use in treating at least one condition. Hundreds to thousands of people may take part.
Clinical Research Protocols
All clinical trials and studies have a protocol - a careful set of rules that set forth how the clinical experiment is to be conducted. The protocol specifies the treatment procedures and doses, the route of administration, the number of participants, the specific inclusion and exclusion criteria that define who may enroll in the study, the duration of the study, and the schedule of the tests or interventions that will measure the results. In trials and studies for SCI, examples of inclusion criteria might be the volunteer’s level and type of injury, the time since injury, age, and gender.
An important part of the clinical protocol is the informed consent, which is an ongoingprocess that gives people ample opportunity to learn the important facts about a clinical trial so they can decide whether to begin or continue participation. During this process, an investigator explains what will happen to the research volunteer. Depending on the treatment under investigation, research volunteers may or may not experience a direct benefit because of their participation.