Understanding Clinical Trials

Choosing to participate in a clinical trial is an important personal decision. The frequently asked questions (FAQs) below and linked pages provide detailed information about clinical trials. In addition, it is often helpful to talk to a physician, family members, or friends before deciding to join a trial. For answers to your specific questions, contact the study research staff or one of our neurosurgeons.

What Are Clinical Trials?

A clinical trial is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the safest and fastest way to the discovery of effective treatments and therefore new ways of improving health.

Clinical trials are conducted according to a plan called a protocol. The protocol describes what types of patients may enter the study, the schedules of tests and procedures, the drugs and dosages, and the length of study, as well as the outcomes that will be measured. Each person participating in the study must agree to the rules set out by the protocol.

The Food and Drug Administration’s job is to make sure medical treatments are safe and effective for people to use. The FDA, however, does not develop new therapies or conduct the clinical trials to demonstrate safety and effectiveness. FDA staff members meet with researchers and perform inspections of clinical trial study sites to protect the rights of participants and to verify the quality and integrity of the data.

For more information about clinical trials and how to participate in one of our trials, click on the links below:

  • Types of Clinical Trials
  • Phases of Clinical Trials
  • Protocols and IRBs
  • Participating in a Clinical Trial
  • Participating in a Clinical Trial
Types of Clinical Trials

There are many different kinds of clinical trials, including:

  • Treatment trials study new drugs, combinations of drugs, or new approaches to surgery or radiation therapy. This could include, for example, pain medicines, steroid injections, radiation treatment, or spinal instrumentation.
  • Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
  • Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
  • Screening trials test the best way to find certain diseases or health conditions through screening tests such as magnetic resonance imaging, for example.
  • Quality of Life trials study ways to improve comfort and the quality of life for individuals with a chronic illness.

Usually, clinical trials compare a new product or therapy to something else to see if it works as well or better to treat or prevent a disease or condition. In a blinded study, a participant may be randomly assigned to receive the test product or an existing, approved therapy. In some studies, participants may be assigned to receive a placebo (a product with no therapeutic action that looks like the test product). Comparison with a placebo can be the fastest and surest way to demonstrate therapeutic effectiveness of new products. Placebos are not used, however, where a patient would be put at risk, particularly in the study of treatments for serious illnesses. Most studies of this kind compare new products to an approved therapy. Potential participants are told before they enter a trial whether placebos are going to be used in the study.

Phases of Clinical Trials

The four phases of clinical trials are:

  • Phase I: These trials are the first stage of testing in human subjects. Normally, a small (20–100) group of healthy volunteers is selected. This phase includes trials designed to assess the safety of a drug, procedure, or device (the treatment). In Phase I trials, little is known about the treatment’s effectiveness in humans.
  • Phase II: Once the initial safety of the study treatment has been confirmed in Phase I trials, Phase II trials are performed on larger groups (20–300) to test the effectiveness (efficacy) of the treatment. When the development process for a new treatment fails, this usually occurs during Phase II trials when the treatment is discovered to not work as planned. When the benefits of treatment outweigh side effects and/or risks, the next phase of testing can begin.
  • Phase III: These studies are randomized, controlled, multi-center trials on large patient groups (300–3,000). The trials in this phase are aimed at being the definitive assessment of how effective the treatment is in comparison with the current standard proven treatment.
  • Phase IV: These trials are also known as Post Marketing Surveillance Trials. Phase IV trials involve the safety surveillance and ongoing technical support of a FDA-approved drug, device, or procedure. Phase IV studies may be required by the FDA or may be undertaken by the sponsoring company (manufacturer). An example of this type of study is the study of aspirin, which was initially approved as a pain reliever, but was later found to be useful as a blood thinner.
Protocols and IRBs

A clinical trial requires a well-designed protocol, collaboration among various medical specialties, and timely and accurate reporting of the findings. It is conducted under the direction of a principal investigator (PI) and approved by an Institutional Review Board.

What Is a Protocol?

A protocol is the study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment. Every doctor/researcher and research facility involved in the clinical trial are required to follow the protocol.

Role of the Institutional Review Board

An institutional review board (IRB) is a committee that has been formally designated to approve, monitor, and review biomedical and behavioral research involving humans with the aim of protecting the rights and welfare of the research subjects. The purpose of an IRB review is to assure, both in advance and by periodic review, that appropriate steps are taken to protect those individuals participating as subjects in a research study. To accomplish this purpose, IRBs review research protocols and related materials (e.g., informed consent documents and investigator brochures) on an ongoing basis throughout the duration of the clinical trial.

Participating in a Clinical Trial

Considering Participation in a Clinical Trial?

Participation in a clinical trial is strictly voluntary. You may decide to participate for access to a medical treatment that is not yet available; however, remember that treatment will not benefit all patients. If you decide to participate in a clinical trial, you will be helping doctors learn more about specific conditions and treatment options which may, in the future, help others with the same disease.

Benefits of Participating

By participating in a clinical trial, you will get to:

  • Play an active role in your health care.
  • Gain access to new research treatments before they are widely available.
  • Obtain expert medical care during the trial from physician investigators who are specialists in disease being studied.
  • Help others by contributing to medical research.

Risks of Participating

Before participating in a clinical trial, you need to be aware of the following risks:

  • There may be unpleasant, serious, or even life-threatening side effects to experimental treatment.
  • The experimental treatment may not be effective for you, and/or it may not be better than the standard treatment.
  • In a randomized study, participants may not choose which treatment they will receive.
  • The protocol may require more of your time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays, or complex dosage requirements.

For listing of current national clinical trials and additional resources, you may visit these websites:

Are Clinical Trials Safe?

The FDA works to protect participants in clinical trials and to ensure that people have reliable information as they decide whether to join a clinical trial. The federal government has regulations and guidelines for clinical research to protect participants from unreasonable risks. Although efforts are made to control the risks to clinical trial participants, some risks may be unavoidable because of the uncertainty inherent in medical research studies involving new medical treatments. The government requires researchers to give prospective participants complete and accurate information about what will happen during the trial.

Current Clinical Trials

For Our Research Colleagues