What are clinical trials?

Clinical trials are experiments using human volunteers, looking at new ways to prevent, detect, or treat disease, with drugs or new surgical procedures. The goal of clinical trials is to determine whether a new treatment is safe, and whether it works. No new drugs or treatments are approved for human use without a rigorous clinical trial to show safety and efficacy.

The idea for a clinical study often originates in a laboratory. After researchers test new therapies or procedures in animal studies, the most promising treatments are moved into human testing by way of a clinical trial. During a trial, a great deal is learned about the experimental treatment, its risks, and its effectiveness.

Clinical research is conducted according to specific protocols, carefully planned to safeguard the participants’ health and answer specific research questions. A protocol describes the following:

  • Who is eligible to participate in the trial
  • The length of the study and what information will be gathered
  • The objective, design, approach, statistical considerations and organization of the trial

A clinical study is led by a principal investigator (PI), who is often a doctor. The research team regularly monitors each participant’s health to determine the study’s safety and effectiveness.

What are the phases of clinical trials?

Clinical trials are conducted in “phases.” Each phase helps researchers answer different questions and is vital to provide sufficient evidence to receive approval from the U.S. Food and Drug Administration (FDA). In order for a treatment or therapy to become available to the public, it must pass through three phases. If the FDA approves, researchers will continue into Phase 4 for ongoing evaluation of safety and effectiveness.

Phase I: Researchers test an experimental drug or treatment in a small group of people (20–80) for the first time. The purpose is to evaluate its safety and identify side effects. No efficacy is expected.

Phase II: The experimental drug or treatment is administered to a larger group of people (100–300) to determine its effectiveness and to further evaluate its safety.

Phase III: The experimental drug or treatment is administered to large groups of people (1,000–3,000) to confirm and fine tune its effectiveness, monitor side effects, compare it with standard or equivalent treatments, and collect information that will allow the experimental drug or treatment to be used safely.

Phase IV: After a drug is approved by the FDA and made available to the public, researchers track its safety, risks, benefits, and optimal use.

Are there clinical trials for spinal cord injury underway?

As we have entered a new era for the field, moving from research to application, clinical trials are being held, or planned, for a number of very promising therapeutic approaches.

At the present time there are clinical trials underway in the U.S. and around the world testing potential treatments for spinal cord injury with others in the pipeline. These studies are evaluating a range of outcomes important to the SCI community from enhanced mobility to autonomic recoveries.

SCOPE = Spinal Cord Outcomes Partnership Endeavor

An academic/industry partnership whose mission is to enhance the development of clinical trial and clinical practice protocols that will accurately validate therapeutic interventions for spinal cord injury leading to the adoption of improved best practices. Find out more about SCOPE and current spinal cord clinical trials.

Does it cost to participate in a trial? Can I get paid to participate?

There are no costs to the participants. There is also no payment or stipend for participating because being paid might affect a participant’s judgment about a treatment’s effect. Some expenses for travel or incidentals may be reimbursed but that is on a case-by-case basis.

How are trials supervised?

Clinical trials are closely supervised by regulatory authorities. Most clinical trials in the United States are approved and monitored by an Institutional Review Board (IRB). An IRB is an independent committee, often based in the treating hospital, that consists of physicians, statisticians, and members of the community who ensure that clinical trials are ethical and the rights of participants are protected.

Informed consent is the process of providing people the facts they need to know about a clinical trial before they decide to participate.

To help someone decide whether or not to participate, members of the research team explain the details of the study and provide an informed consent document that describes its purpose, duration, required procedures, and who to contact for further information. The informed consent document also explains risks and potential benefits.

The participant then decides whether to sign the document. Informed consent is not a contract. Volunteers are free to withdraw from the study at any time, or to refuse particular treatments or tests.

Why do people participate in clinical trials?

There are many reasons people decide to join a clinical trial. People participate in trials to receive the newest treatment and to access additional care from the clinical trial team. Others want to help accelerate science as many studies are delayed or fail to get off the ground due to lack of volunteers. Whatever the reason may be, participating in a study is a personal choice and it is important to understand the clinical trials process so that you can make an informed decision.

Overall, clinical trials offer hope for many people and provide an opportunity to help researchers find better treatments for others.

What is NACTN’s role in advancing clinical trials?

Through a network of hospitals with medical, nursing and rehabilitation personnel trained in the evaluation and management of SCI, the North American Clinical Trials Network (NACTN) works to ensure clinical trials are done in a way that maximizes both patient safety and our ability to gather valid, meaningful data.

Three key roles of NACTN include:

  • Gathering and documenting patient medical information in a data registry to better understand the body’s natural course of recovery after a spinal cord injury
  • Establishing and implementing standardized patient assessment protocols
  • Conducting new trials of promising therapies for spinal cord injury

Now, when researchers get the go-ahead for human trials, they don’t have to build the support system for such studies from the ground up -- NACTN has already built the standing infrastructure to accelerate research with the goal of getting safe and effective treatments for spinal cord injury to our community as quickly as possible.

Source: National Institute of Health