A clinical trial is a research study to determine whether an experimental drug, device or treatment is beneficial to the advancement or improvement of patient care.  Activities during a clinical trial are different from those of routine standard of care. Clinical trials are designed to answer several essential questions.

• Is the new drug able to treat people?

• If so, is it better than the drugs currently available?

• If it is not better, then what are the side effects of this drug?

• Is the drug safe?

• There is no treatment without side effects. Do the benefits of the drug outweigh the risks?

You or your child may wish to participate in a clinical trial for a variety of reasons. Researchers cannot develop new treatments without your help. It can be a rewarding and satisfying experience, as well an opportunity for you to contribute to the development of innovative, “cutting-edge” medical treatment. You could be part of a process that is critical in the development of medications. All medications must go through this process to become FDA approved. These studies are performed to determine how well the medication works or how long its effects can last.

You will gain unique access to experts in a particular disorder, especially in rare neurological disorders. Consulting physicians who are affiliated with a particular study will provide you with an opportunity to ask questions and discuss either your, or your child’s disorder. You will be able to ask detailed questions, free of charge.

Individuals who volunteer for phase II and III trials can gain access to promising drugs long before these compounds are approved for the general public. Study participants will be closely monitored and will receive comprehensive medical attention from highly skilled professionals who are readily available to answer questions. In most studies, you will receive diagnostic testing, examinations, and treatment without charge. This can help offset expenses that would otherwise make it difficult for you to receive care without insurance coverage. We may be able to compensate you for your time and travel.

Participating in a clinical trial can entail both benefits and risks. However, the risk is present everywhere, in every area of ​​life, including medical care. As for clinical trials, most side effects are temporary and generally disappear over time once treatment has ended. While in some studies participants can experience long-term adverse reactions or require medical attention. Some side effects may show up during treatment, but others may not appear until after treatment has finished.

Some investigational products can cause serious or even life-threatening side effects. Thus, clinical trials can pose health risks to participants, as the safety and side effects of the investigational products being tested are not yet fully known.

Potential risks of participating in a clinical trial are described in detail in the informed consent form (ICF), which has to be signed prior to any study assessments are performed. Additionally, you will be able to discuss all of your questions with a member of the research team, who will explain all possible side effects or other risks of the treatment. You should think carefully before deciding to take part in the research. It is important to understand that participating in a study may not be of direct benefit to you. However, your participation can help drive medical research forward to benefit others.

The criteria for participation in clinical trials depend on the drug, the type of study and the disease condition that is being studied. For example adults are often excluded from the drug trial for Duchenne muscular dystrophy (DMD). The opposite is the case when it comes to prostate cancer treatment. The criteria are stated in the clinical trial protocols. They also feature "exclusion criteria," which are characteristics that disqualify patients from participation. Pregnancy and breastfeeding are very common among these “exclusion criteria.”

Clinical trials are a necessary pathway to study and develop new medical treatments. Your participation in any clinical trial will provide invaluable information, helping researchers determine whether the particular treatment would be beneficial or not.

For anyone who is interested in participating in a research study, knowledge and preparation are important. Based on the study protocol, your Clinical Research Coordinator will inform you of any steps you need to take to prepare for the study. Most studies will require you to provide medical records during the pre-screening process.

• Patient Bill of Rights: as a research study participant, your rights and protection are our priority. We will work with you in understanding of the protocol and study procedures, ensuring that all questions or concerns are answered.

• Confidentiality: The Health Insurance Portability and Accountability Act (HIPAA) prohibits health care providers from using or disclosing “Protected Health Information (HPI)” to others without the consent of the patient.

• Inform consent process: Informed Consent is a voluntary agreement to participate in research. It is a process as outlined by FDA (21 CFR 50) and not merely signing a document. The participant will need to learn about the purpose of the study, the procedure involved, the potential risks and benefits.

In many cases, your physician will be able to offer you information about opportunities to become participants in a drug trial. This is often the case if the drug’s possible effectiveness is greater than traditional methods of treatment.

A great online source is ClinicalTrials.gov. This is a federally supported and searchable registry for clinical trials pertinent to your condition: www.clinicaltrials.gov

Additionally, patient advocacy groups can offer helpful information about clinical trial opportunities specific to your condition.

• Read the inform consent and other documents carefully. Ask questions about the study and make certain that you fully understand the risks and benefits of the study.

• It is important to adhere to the guidelines set forth in the protocol, such as the duration of the study, report any potential side effects, and inform the research team of any changes. These guidelines will be discussed with you by the Principle Investigator and Clinical Research Coordinator who are responsible for conducting the clinical trial.

If you decide that you no longer want to participate in a clinical trial, you are able to do so at any time. Simply inform the research team, and they will discuss the process of withdrawal from the study. Withdrawal will involve stopping the investigational treatment and may involve proceeding to the End of Study (EOS) and follow-up visits.

Your insurance should never be billed for study-related assessments.

The methods, duration, participant requirements, and further details on the clinical trial are specified in the study protocol. The research team will explain the protocol to you, and detailed information about all study procedures will be included in the informed consent form (ICF). Patients should thoroughly review the ICF before deciding to participate. If you sign the ICF, you confirm that you have read all the documents and have voluntarily decided to participate in the clinical trial.

The schedule of clinic visits is developed individually for each clinical trial and will be detailed in the informed consent form (ICF). In some cases, the participant needs to see a doctor several times a week; in other studies, visits can be no more than once a month. Therefore, it is vital to become acquainted with the details of the ICF and discuss any concerns with the research team.

The care and investigational treatment that you receive as part of a clinical trial are provided at no cost to participants. Some studies also provide reimbursements or stipends to lessen the burden of travel to the study site, meals during clinic visits, and other expenses associated with your trial participation. In this case, the Institutional Review Board (IRB) determines the amount and schedule of all payments to participants before the start of the study to ensure that the proposed method and timing will not result in any undue influence as guided by FDA (21 CFR 50.20).

Clinical trials employ different methods of recruiting study participants. The research center can review its existing databases of patients or medical charts to determine potentially eligible patients. Other recruiting methods include advertising in mass media (newspaper, radio, television, or Internet), public workshops, free health screenings, or forums.

During this process, the study staff will ask you a series of questions to confirm your willingness to engage in the clinical study and verify whether you meet the criteria for participation or not. This process typically happens during a phone call with site staff.

If you have been determined eligible during the pre-screening process, you will be scheduled for an appointment at the research site and meet with a member of the research team. Prior to initiation of any clinical screening procedures, you will discuss and be asked to sign an ICF. This document provides a detailed overview of the study, the possible risks and benefits, emergency contacts, and your rights as a research participant. The informed consent process also allows you to ask any questions you might have about participating in the study. You will receive a copy of the signed ICF to take home and keep for your records.

After you have given consent to participate in the clinical trial and signed the ICF, the site staff will begin guiding you through the screening procedures. These assessments are designed to determine if you meet the specific inclusion and exclusion criteria for the clinical research study you decided to join. The screening process includes completing questionnaires and answering a series of questions about your medical history, current and previous treatments. To confirm that you meet the study’s requirements, you may also have to undergo a physical exam, electrocardiogram (ECG), as well as blood and urine tests to assess your general health. The investigational product is not usually provided during the screening visit.

Upon satisfying the study eligibility criteria (which include acceptable laboratory and medical test results), you will be asked to come to the clinic for a series of study visits, also called “treatment visits.” Some of these visits may be conducted virtually. Study participants start receiving the investigational product or the comparator product (sometimes a placebo) during treatment visits. The frequency and duration of visits varies for every study.

The End of Study visit occurs once you are no longer receiving investigational treatment. This stage requires the same or similar tests conducted before receiving the first dose of the investigational product. The research team will also discuss your follow-up treatment options (which may include standard of care treatment for your condition or open-label extension study of the investigational product). Please note that you may be asked to return to the research site for a follow-up safety visit if required.

The number of follow-up safety visits and intervals may differ according to the type of study. The purpose of this stage is to make sure that you are not experiencing any persistent side effects from the investigational product or the overall study participation.