Shepherd Center Experts Answer Questions about Participation in Clinical Trials

In laboratories and medical centers across the globe, scientists are uncovering and testing new ways to treat a variety of illnesses and injuries – a rigorous process that often starts in animal models. If and when these treatments move beyond basic or what might be called “bench science,” they must be proven both safe and effective in humans before they can ever become the standard of care.

At Shepherd Center, clinical trials have helped researchers uncover new approaches to help maximize rehabilitation. Two Shepherd Center researchers, Issi Clesson, R.N., director of clinical trials, and Deborah Backus, P.T., Ph.D., director of multiple sclerosis research, weigh in on the basics.

Why do we need clinical trials?

Simply stated, without carefully designed studies (called clinical trials), advances in rehabilitation care wouldn’t happen.

This type of research has paved the way for new drugs, biologics, devices and other therapies to help:

• prevent secondary damage that quickly follows spinal cord injury;
• promote neurological recovery;
• manage symptoms and/or improve function (spasticity, sexual function, walking speeds and bladder management);
• alter the way the immune system works in multiple sclerosis (MS);
• generally make life better for people with disabilities.

Clinical trials also help researchers and clinicians determine the best way to diagnose a problem, administer rehabilitation care (when, how often and how much) and identify which individuals will have the best outcomes.

Why should someone consider participating in a clinical trial?

It depends on your situation, but there can be many upsides. Being part of a clinical trial allows you to take a more active role in your care. It may also give you access to treatments – new medications or devices to improve function and mobility – that wouldn’t otherwise be available. You are also helping researchers answer critical questions that will help others like you in the future.

Are there risks?

Being in a clinical trial doesn’t guarantee that you will improve or receive the investigational therapy. You may, instead, be in the control group, which means you will receive the current standard of care or a placebo (an inactive medication). There can be unexpected side effects, but the research team monitors these closely. It’s really important to also keep up with regular clinical and preventive care while participating in research.

How do I know if I can take part?

All clinical trials have specific criteria about who can and cannot participate (called inclusion and exclusion criteria). For example, researchers may only want to test the therapy in people of a certain age, gender, type of disability or level of injury, or who have not taken certain treatments previously. At Shepherd, an early trial looking at an investigational stem cell therapy was limited to patients with a diagnosis of complete spinal cord injury. The reason? So that researchers are more confident that a response is due to the therapy and less likely because of natural healing.

Can I change my mind once I enroll?

Yes. You can always choose to leave a clinical trial. But you should share your reasons with the research team and you may have to return for a follow up visit. They need to ensure your safety and account for any changes in the number of people enrolled. 

How is Shepherd Center involved in clinical trials?

Shepherd Center is committed to improving the lives of patients and families. “Clinical trials are one avenue to advance this goal,” Clesson says. A trial at Shepherd led to FDA approval of a diaphragm pacing system device that allows ventilator-dependent patients with spinal cord injury to wean off the ventilator full or part-time. “The study not only freed some patients from the ventilator, it also was an improvement on the previous device and invasive surgical techniques,” Clesson adds.

At any given time, about 550 people are participating in research at Shepherd. Many of these studies are conducted in collaboration with leading experts at other hospitals, research centers, medical schools and universities.

For information about research under way at Shepherd, visit shepherd.org/research.

SIDEBAR:  A Phased Approach

Clinical trials are done in phases to ensure new treatments are well-studied. Each phase has a purpose and is required before researchers can seek approval from the U.S. Food and Drug Administration.

Phase I – Is the treatment safe?

Phase II – Does the treatment being studied work the way researchers think it will?

Phase III – How does it compare to the standard treatment? New therapies should be as safe and effective as those currently used. This phase usually leads to data that support a marketing application in the U.S. (FDA approval)

Phase IV – After gaining FDA approval, this phase considers other questions that need to be answered or evaluated (for example, certain outcomes, frequency of side effects, etc.) 

CLINICAL TRIAL RESOURCES

For information about clinical trials, talk with your care provider. You can also visit:

• Shepherd.org/research
• Clinicaltrials.gov
• Centerwatch.com

Watch for These Things When Evaluating Participation in Clinical Trials:

If you are considering enrolling in a clinical trial, it’s important to do your homework first. Legitimate clinical trials:

• will not make claims that treatments are cures;
• should be free of charge (if you are asked to pay, beware);
• should obtain your consent prior to participation. You should be told upfront and in writing of all the potential side effects and treatment alternatives.

Written by Amanda Crowe