This content was produced by SiteRx writers. These materials provided by SiteRx are for informational purposes only, not intended to be used to diagnose or treat any disease or illness, and not an alternative to medical advice from your doctor or other qualified healthcare providers. Participation in a clinical research trial is voluntary and subject to risk. SiteRx does not make any representation as to the risks or benefits of participating in a clinical research trial, and you should always consult your doctor or other qualified healthcare professional to discuss your treatment options. If you think you may be suffering from any medical condition, please seek immediate medical attention. You should not delay seeking medical advice, disregard medical advice, or discontinue medical treatment because of the information contained in these materials.

Medically reviewed by Jay Rubin, M.D.

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A Patient's Guide to Clinical Trials

Living with a neurological condition may be disabling and very challenging for you and your family. For some diseases there may be few treatment options, responses may seem limited, or there might be bothersome side effects associated with available medications.

Clinical trials offer advanced aspects of medical science and technology often with options available in convenient locations and typically without cost. These clinical trials are particularly important because the incidence of neurological diseases is steadily increasing.


Clinical Trials Are An Option

Clinical trials can provide you access to new treatments that are not yet approved for widespread use by the general public and typically without cost. Participating in clinical trials may also contribute to the advancement of medical science and the development of new potential treatments, which may help other patients and future generations living with neurological disorders.

site rx brain

What is a clinical trial?

A clinical trial is a type of research study that is designed to understand new medical interventions. These interventions could include drugs, devices, or changes in behavior. Clinical trials require volunteer participants to receive these interventions. Participation is completely voluntary at all times. Informed consent is a vital component of the continuous patient and caregiver education and participation process. You have the right to withdraw from a study for any reason. Some portions of a clinical trial may be optional.

When researchers conduct these studies, they are typically studying 1) the effectiveness and safety of a new drug or therapy, and 2) comparing a new drug or therapy to a widely-available treatment to determine if the new drug or therapy represents an improved treatment option.

What are the types of clinical trials?

New drugs or therapies require extensive research and multiple staged studies. One difference across these study stages is the level of involvement of the study participants and scientific experience with the new drug or therapy.

Observational Studies

In these types of studies, participants are observed and monitored over a period of time to understand how a disease progresses. While researchers have learned a lot about neurological conditions in recent years, there is still so much that is not fully understood.

In some observational studies, researchers study either changes in participant behavior or signs and symptoms of a disease’s progression over time without the participants receiving any new treatment.

Genetic Studies

Within any group of patients living with the same condition, there may be differences in genes. These differences may be observed between individuals or from one group of patients to another. The genetic differences could result from gene mutations or simply hereditary factors

This is why researchers conducting genetic studies need to better understand the connections between genes and medical conditions. Using this information, the researchers can design new treatments that can cater to the diversity of patients living with neurological conditions.

Interventional Studies

With these studies, researchers provide experimental treatments to participants and then study the participants over time to understand how the treatment works. It is common in these studies for a percentage of participants to be given a placebo, while the others are given the experimental drug or therapy.

The goal of interventional studies is to identify a treatment that is safe, effective, and possibly better than currently available treatments.

Phases of Clinical Trials

Clinical trials are typically divided into multiple study phases. In each of these phases, researchers answer important questions that help them determine whether the research of a new drug or therapy should continue to the next phase. The following is quick and simplified overview of possible clinical trial phases:

Clinical Trial Phases

Phase 1: Is the treatment safe?

Researchers start by testing a new treatment on a small number volunteers to understand whether it is safe at low doses

In Phase 1 of a study, researchers may also try to find possible ways to administer a new drug or therapy to patients while reducing potential risks. Researchers also work to identify any side effects at this point before the study moves on to Phase 2.

Phase 2: Does the treatment work? Are there any side effects?

Phase 2 of a study usually involves several hundred patients and can last anywhere from a few months to a few years. Of course, the safety of any new treatment is paramount throughout the clinical trial process. However, in Phase 2 of a study, even though researchers continue to focus on the safety of the treatment and potential side effects, they also more closely consider the effectiveness of the treatment.

The data researchers obtain from Phase 2 of a clinical trial may also help in designing Phase 3. This data also helps to guide researchers in how they make decisions regarding dosage for the new drug or therapy. All of this impacts how the treatment will affect patients.


Phase 3: Is the treatment better than what is currently available?

As you may have noticed, the number of participants typically increases with each stage of a clinical trial. Phase 3 of a study may involve anywhere from a few hundred to thousands of patient volunteers living with the neurological condition for which the treatment was developed.

In addition to studying the benefits of the new treatment over current ones, researchers may also study patients for any long-term or rare side effects. Given that this phase lasts several years, researchers are in a better position to study the different ways participants respond to the new treatment.


Phase 4: Is there anything else to learn about this treatment?

Phase 4 studies are conducted after the new drug or therapy has been approved by the U.S. Food and Drug Administration (FDA). At this stage, the FDA may approve further testing to address additional indications for the drug or therapy, or the FDA may require additional research on long term patient safety and tolerability. The FDA approves a drug for further testing in this phase only when the treatment has satisfied all the criteria required to progress from Phases 1 to 3.

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Talking To Your Doctor About Clinical Trials

Beyond the potential benefit of a new treatment option, your participation in clinical trials may assist in developing new treatments that help others. Unfortunately, while hundreds of neurological clinical trials are active at a given time, most struggle to find volunteer participants

If you are interested in participating in a clinical trial but are unsure where to start, discuss the option with your doctor who can help connect you to a promising trial.

Here's how to ask your doctor

A good starting place in discussing clinical trials with your doctor is to review the benefits, which includes access to:

  • State-of-the-art medical treatment, care, and vigilance
  • In-depth education about your condition
  • Innovative treatments free of charge
  • Support and extra attention for you and your caregiver(s)
  • Networks of highly motivated healthcare providers
  • Explanation of potential known risks and benefits.
  • Attention to your safety as a respected patient volunteer

Your doctor is important in the referral process

When you talk to your doctor about clinical trials as an additional care option, you should emphasize that his or her participation in this process is important and necessary. Your doctor is your trusted expert in providing you medical care and will help guide you in exploring clinical trial options. In fact, studies show that patients have improved experiences in clinical trials when their doctor is involved.   

Questions to Prepare

It might also be helpful to consider asking some questions to start the conversation about clinical trials with your doctor. Questions to consider include: 

  • Have you referred patients to clinical trials before?
  • Do you have any interest in referring patients to a clinical trial if the process is simple?
  • Are there clinical trials studying my condition?

If your doctor says he or she has explored clinical trials for patients before, this is an excellent opportunity to express your interest in participating in a clinical trial.

If your doctor is interested in participating in the clinical trial process, let them know that SiteRx works with doctors to match patients to clinical trials. Most importantly, your doctor remains the primary educator and source of medical guidance throughout the process.

Your Rights 

As a patient, you have the right to know all possible treatment options available to you. In addition, you may withdraw from a clinical trial at any time. Your doctor is responsible for sharing this information with you and can help guide you through this process.

Requesting Access Locally

Your doctor may be aware of local opportunities to participate in clinical trials. For example, hospitals, research centers, and clinics often recruit patients for clinical trials. Your doctor may also have access to information within a network of fellow medical providers. By requesting local access, your doctor may share some of this information.


people and a computer

Finding Clinical Trials For Yourself

You might prefer to find clinical trials for yourself. There are several resources you can try.

Sources to Find Information 


Websites are generally good places to start looking for information about clinical trials, but these might not be the best for local options. Be wary of websites that have outdated information because some are not updated frequently. In addition, many websites containing clinical trial information might use technical language that may be overwhelming. In such cases, you should ask your doctor to explain any information you do not understand.

Good websites to try to include: 

Friends and Family

Another way to find clinical trials is by talking to friends, colleagues, or family. You may very well know someone that has participated in a clinical trial or knows someone who has.

Communicating With Your Doctor

As you search for information on clinical trials, stay in communication with your doctor about the process. You can start by asking your doctor what he or she thinks is the best way to participate in a clinical trial, if recommended. Your doctor not only has a medical understanding of your condition but is also familiar with how your condition has progressed since your diagnosis.

It is beneficial to keep this line of communication open throughout the trial. Studies show that ongoing communication between patients and doctors during a clinical trial makes it easy for doctors to continue providing care to patients even after a trial ends. In addition, this communication keeps your doctor informed to make decisions about drugs and therapies, tests, and procedures, and allows your doctor to monitor changes in signs and symptoms during and after the clinical trial.

Finally, if you encounter any issues during your participation in a clinical trial, you can also reach out to your doctor for assistance or guidance.

SiteRx can help you!

Working with your doctor, SiteRx can help match you to a clinical trial looking to enroll participants. Your doctor can use SiteRx's easy sign-up process to get started. Your doctor does not need to be a Principal Investigator, as some doctors presume. SiteRx can assist your doctor in recommending clinical research opportunities to you (and others).

SiteRx’s technology will assist your doctor in certain administrative tasks and will help your doctor effectively match you to a clinical trial looking to enroll patients. Studies show that one of the most significant barriers to patients participating in clinical trials is doctors not having enough time to recommend options for patients. SiteRx provides an excellent opportunity to break down these barriers by assisting doctors in matching patients to clinical trials suited to your particular condition.