What Are Clinical Trials?
Clinical trials are research studies conducted to evaluate the safety and efficacy of a potential intervention or diagnostic tool. Each trial has specific criteria for participation, as well as a detailed protocol of assessments, timelines for participation, and drug administration (if applicable). Clinical intervention trials span an array of modalities or therapies, such as pharmaceuticals and medical devices, and may provide insight into how an experimental treatment compares to standard treatment in terms of efficacy and side effects. Some clinical trials are designed to test ways to detect a disease early, even before symptoms are present. Prevention-based clinical trials and quality of life improvement clinical trials may yield important information about the role of support groups, caregivers, or lifestyle factors on a specific medical condition.
Role of the FDA in Clinical Trials
All clinical trials are conducted by a qualified team of professionals. Experimental treatments and protocols are designed by scientists who may work at a university or a pharmaceutical biotech company. These entities contract with qualified professionals and sites to conduct clinical trials. Clinical trial research is highly regulated by the Food and Drug Administration (FDA) and Institutional Review Boards (IRB), with subject safety being of paramount importance.
Clinical Trials at The CRCNJ
At The CRCNJ, we conduct clinical trials focused on memory loss, Alzheimer’s disease, mild cognitive impairment, and dementia. Our goal is to advance the state of research and treatment for these diseases, help discover the most effective new treatments, and eventually contribute to eliminating them altogether. For more information on the clinical trials we are conducting at The CRCNJ, please click here.
Four Phases of Clinical Trials
The FDA requires clinical trials to be conducted in graduated phases. If a potential treatment successfully passes through the first three phases (proving safety and efficacy), the FDA approves the stage 4 phase for clinical use. The FDA continues to monitor the treatment’s effects.
Phase I
In a Phase I clinical trial, an experimental treatment is tested on a small group (20 to 80) of generally healthy people (ages 20 to 80) to evaluate its safety and side effects as well as to begin to find the correct dosage if the treatment is a drug. The emphasis during Phase 1 is on safety.
Phase II
This phase requires more people (100 to 300). The emphasis of Phase II is on dosage and preliminary data on effectiveness. The goal is to acquire additional information about dosage and safety in the population for which the intervention is indicated. In addition, preliminary information on whether a treatment works in people who suffer from a particular disease is collected. As in all stages of clinical trials, safety and side effects are still studied. Phase II can last several years.
Phase III
The primary goal of a phase III study is to prove efficacy, which means that a drug or intervention has a positive, expected effect. The number of people in these trials is much larger (several hundred to 3,000). If the FDA deems the trial phase positive, the treatment is approved in the United States as experimental.
Phase IV
This final trial phase is completed after the FDA approves the use of the experimental treatment. A medication or medical device’s efficacy and safety is monitored using large, diverse populations. In some cases, side effects of a medication are unknown prior to this phase and become evident with the large amount of people using the medication over a longer time span.
Why Participate in a Clinical Trial?
Each person’s decision to participate in a clinical trial is different. They may wish to join a trial because all standard treatments have not worked for them, or because there is currently no approved treatment for their health problem. Another benefit of participation includes the possibility of receiving a treatment early, before it becomes widely available, which can take years. Some healthy people join clinical trials because they want to help find a cure or treatment for a disease or condition.
By participating in a clinical trial, people are playing a more active role in their healthcare and have access to potential treatments they wouldn’t otherwise have. They are also playing a role in scientific discovery that leads to helping many people. Major medical breakthroughs would never happen without trial participants.
Importance of Diversity in Clinical Trials
Diversity in clinical trials is extremely important and includes people of different ages, sexes, races, and ethnicities. When research only involves a similar group of people, the findings can be skewed and may not be useful for everyone, or even harmful to some others. A diverse clinical trial has much more accurate data resulting in wider use in the general population – in essence, the treatment works for more people.
Researchers are aware that willingness to participate may be hard for certain people, and that reluctance, and even fear, is grounded in legitimate concerns. Acknowledging concerns and finding a way to bridge that gap continues to be a challenge, but one that is surmountable when the medical and research community is empathetic and willing to spend the time needed to change, and to meet, the needs of these populations.
Will I Know if I Receive the Treatment or a Placebo?
There are different types of trial designs. The details of the design of the trial, including group assignment and number of groups or participants, are determined by scientists and statisticians prior to the onset of a trial. Often, neither the study participant, nor the members of the team at the research site, know whether a participant is receiving an active dose of the drug or a placebo. This way, data cannot be accidentally biased. Assignment to the drug-treated or placebo group is random, often conducted through a computer algorithm. The procedures and trial experience for all participants is identical, regardless of group assignment.
While participants won’t know if they are receiving the treatment, if they do not participate, there is zero chance of receiving the treatment. There is also ample evidence to show that people who are in clinical trials, including those who received the placebo, have better outcomes than people who are not in clinical trials.
What Happens When a Clinical Trial Ends?
After a trial is complete, researchers collect and analyze all the trial data There may be a long lapse of time in between a person’s completion of the trial and the publication of any findings because data is not analyzed until all participants at all sites have completed the study. At this time, group assignment may be released by the study team.
Some Phase III studies include the possibility of participating in an open label extension after completing the double-blinded portion of the trial. With their informed consent, the participant will be given an active dose of the drug. Both the participant and the study team will know that the person is receiving the drug, but may not know the exact dose received. The participant will continue to be followed by the study team as per the open-label trial protocol.
How Do I Know If I Qualify for a Clinical Trial?
After consent, participants are screened by a clinician to determine whether they are eligible for a particular trial. Inclusion criteria vary widely based on the type of clinical trial. Even though a participant may not be eligible for one trial, they may qualify for a different one. There are also exclusion criteria that may include things like specific health conditions or medications that have contraindications to the treatment being used in the trial.
The CRCNJ is devoted to helping to find new treatments and a cure for Alzheimer’s disease, dementia, memory loss, and other cognitive disorders. We can only honor this mission through clinical trials.
One of the best ways to determine whether you qualify for a clinical trial at The CRCNJ is to take part in a memory screening. Memory screenings are offered at no cost to you. After evaluation and review of the results of your memory screening, you will be notified if you qualify for a trial. To determine if you’re eligible for a no cost memory screening, complete our contact form or call us at 973-850-4622.
