This content was produced by SiteRx writers.

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 Are An Option

Clinical trials can provide you access to new treatments that are not yet publicly available and without any expense. Participating in these studies contributes to the advancement of medical science and the development of new potential treatments; this helps other patients and future generations living with neurological disorders.

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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 always have the right to withdraw from a study for any reason. Some portions of a clinical trial may be optional. Strict confidentiality of your medical information is another important feature of your participation. (Learn more in our post What You Need to Know About Participating in Neurology Clinical Trials.)

When researchers conduct these studies, they are carefully and consistently studying 1) the effectiveness and safety of a new treatment and 2) comparison to any available treatment.

What are the types of clinical trials?

New treatments are introduced in stages. The main difference across these stages is the level of involvement of the study participants and scientific experience with the new treatment.

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 changes in participant behavior, signs, and symptoms of the disease over time without giving the participants any treatment. 

Genetic Studies

Within any group of patients living with the same condition, there will 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 better 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 active drug or treatment.  

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 can be broken down into four phases. In each of these phases, researchers ask vital questions that help them determine whether the clinical trial moves on from one phase to another.

trial phases

Phase 1: Is the treatment safe?

Researchers start by testing a new treatment in 20 to 100 healthy volunteers to understand whether it is safe at low doses. Researchers also study how the treatment interacts with the body to determine which dose is the safest and most effective. 

In Phase 1, researchers will also try to find the best possible ways to administer the treatment to patients while reducing potential risks. It is crucial for researchers to identify any side effects at this point before the clinical trial moves on to Phase 2.

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

Once researchers have determined that the treatment is safe at low doses, they go ahead and test this treatment in patients who actually have the diseasePhase 2 usually involves several hundred patients and can last anywhere from a few months to two years. 

The safety of any new treatment is paramount throughout the clinical trial process. In Phase 2, researchers continue to determine the safety of the treatment. Here, they pay special attention to any side effects patients may experience. In addition, with larger numbers of patients (several hundred), researchers are in a better position to identify any safety issues that smaller studies did not reveal.  

The safety data researchers obtain from Phase 2 of the clinical trial also helps in designing Phase 3. This data also helps to guide researchers in how they make decisions regarding treatment dosage, sample collection, or even the types of research tools used during the clinical trial. Researchers must be using the proper methods to collect samples and obtain data. 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 numbers of participants increase as a new treatment progresses along with the clinical trial from one phase to the next. Phase 3 involves anywhere from 300 to 3,000 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 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 includes Food and Drug Administration (FDA) review and further testing. The FDA approves a drug for further testing in this phase when the treatment has satisfied all the criteria required to progress from Phases 1 to 3. At this level, the treatment is now considered safe, effective, and beneficial to patients.

After a new treatment has been approved, it is still considered new and experimental. Researchers need to study participants further to determine if some side effects show up many years later. There can also be varying responses to the treatment across patients from different demographics and populations. Researchers continue to study the treatment in Phase 4 with thousands of patient volunteers over a more extended period.

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Talking To Your Doctor About Clinical Trials

Beyond the benefits to your personal health, your participation in clinical trials is important 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/her participation in this process is beneficial. Your doctor is your trusted expert in providing you medical care and can 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.   

Research opportunities for your doctor

Your doctor's involvement in referring you to a clinical trial also opens up research opportunities for him/her which, in most cases, is not easily accessible. Many doctors prefer to be involved in research for professional reasons and to add new knowledge and tools to their practice. In some instances, participation in clinical trials also provides an opportunity for your doctor to generate new income for their practice

Some doctors may have reservations about the time and effort involved in recruiting patients for trials or wish to avoid tedious administrative tasks. SiteRx actually removes this burden by taking care of the patient matching process, allowing your doctor to focus on your care

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/she has referred 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 a referral process, let them know that SiteRx works with doctors to match patients to clinical trials, with doctors reimbursed for their time. But, more 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, teaching hospitals, academic research centers, and clinics often recruit patients for clinical trials. Your doctor may also have access to the information within a network of fellow medical providers. By requesting local access, your doctor may share some of this information.


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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 clinical trials, but these might not be the best for local options. Be wary of websites that have outdated information, as 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 could ask your doctor to explain any information you do not understand.

Good websites to try to include: 

You can also refer to this resource which provides detailed information about clinical trials, including where to search for trials currently enrolling patients.

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 You 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/she thinks is the best way to engage in a clinical trial. Your doctor not only has a good 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 doctors informed on how to make decisions about drug prescriptions, testing procedures, or monitoring changes in signs and symptoms during and after the clinical trial. Finally, if you encounter any issues during your participation in a 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. While participating in the referral process, your doctor can access new clinical research opportunities and get reimbursed for the time spent referring you and other patients. 

SiteRx takes care of all the administrative and recruitment tasks and matches 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 refer patients. SiteRx provides an excellent opportunity to break down these barriers by actually matching patients to clinical trials suited to your particular condition.