About Multiple Sclerosis Clinical Trials

WHAT IS A CLINICAL TRIAL? ARE CLINICAL TRIALS RIGHT FOR YOU? 

Clinical trials, sometimes referred to as clinical research, are studies in which people, generally patients who have the illness or disease being studied, give consent to test efficacy and/or safety of a drug or device. Medical professionals use clinical trials to gather data and determine whether a new investigational product/medication, medication or device is effective and safe for humans.  

At Rocky Mountain MS Research Group, we are generally involved in studies on new investigational medications to be used for disease modifying therapy (DMT) or ways to make existing or current drugs safer.

Deciding to become involved in a clinical trial can be difficult if you have an illness. If there is a clinical trial available for which you qualify, it is worth at least exploring the possibility. Much has changed over the last few years, and people should know as much as possible about all of their options before making the decision to participate in clinical research.

We do not know how research will turn out. Sometimes the question we are trying to find an answer to reveals something completely new and unexpected. There is no simple answer to the question, "Should I take part?"

You usually don't hear about research unless something has gone wrong in a clinical study. For instance, the evening news will report when a volunteer in a study is harmed. Although it is very rare, people have been harmed or even died while taking part in a clinical trial. Reports of these tragic outcomes are important, because they help to expose problems in the system. These problems can then be handled so that they do not happen again. Due to the serious issues in past research, there are now laws, requirements, and procedures in place to protect the rights and the health of study participants.

There are thousands of people who are helped each year because they decided to take part in a clinical trial. There are millions of people who benefit from others' participation in clinical trials.

The decision to participate in a clinical trial is a very personal one and depends on many factors. The benefits and risks of the study have to be carefully evaluated. It is important to think about what you hope to achieve by taking part. Participating in a study also depends on your own values, preferences, schedule, and priorities.

Knowing all you can about research studies and clinical trials in general, as well as the ones you are thinking about taking part in, can help you feel better about your decision. If you do decide to take part, knowing what to look for and what to expect ahead of time can be helpful too. Participating in clinical research does take time and it is a responsibility to make sure the guidelines of the study are followed as instructed.

There are several types or phases of clinical studies.  At Rocky Mountain Multiple Sclerosis Clinic, and now Rocky Mountain MS Research Group, the new research arm of the Clinic, we have participated in studies from Phase 1 through Phase IV.  Here is a brief description of the various phases of clinical trials:

PHASE 0 STUDIES

Phase 0 studies are the earliest stage of drug development.  These exploratory studies often use only a few small doses of a new drug in each patient.  Phase 0 studies are not common and generally involved fewer than 20 people. Phase 0 studies are not a required part of testing a new drug, but they may help to focus the process.

PHASE I STUDIES

Phase I is much more common than the Phase 0 trials. Phase I studies are usually the first studies of a new drug that involve a small number of people anywhere between fifteen to fifty people.  There are most likely healthy volunteers involved at this stage of research as well. The main reasons for doing Phase I studies are to find out the highest dose of the study medication that can be given safely without serious side effects, and the best way to give the new investigational product/medication. The first few people in the study often get a low dose of the investigational product/medication and are watched very closely. If there are only minor side effects, the next few patients may get a higher dose. This process continues until medical professionals find the dose that is most likely to work while having an acceptable level of side effects.

PHASE II STUDIES

Phase II studies are conducted if an investigational medication is found to be reasonably safe in Phase I clinical trials, the investigational product/medication can then be tested in a Phase II clinical trial to see if it works the way researchers think it will.

Usually, a group of 25 to 100 patients with the same type of illness gets the new investigational product/medication in a phase II study. They are treated using the dose and method found to be most safe and effective in phase I studies. In a typical Phase II clinical trial, all the volunteers usually get the same dose, and no placebo is used.

But some Phase II studies do randomly assign participants to 1 of 2 investigational product/medication groups, much like what is done in Phase III trials (see below). These groups may get different doses or get the investigational product/medication in different ways to see which provides the best balance of safety and effectiveness. Phase II studies are often done at major cancer centers, but may also be done in community hospitals or even doctors' offices.

Doctors look for some evidence that the investigational product/medication works. The type of benefit or response they look for depends on the goals of the clinical trial. If a certain percentage of the patients benefit from the investigational product/medication, and the side effects aren't too bad, the investigational product/medication is allowed to go on to a Phase III clinical trial. Along with watching for responses, the research team keeps looking for any side effects. Larger numbers of patients get the investigational product/medication in Phase II studies, so there is a better chance that less common side effects may be seen.

PHASE III STUDIES

Phase III Investigational product/medications that have been shown to work in phase II studies usually must successfully go through one more stage of testing before they are approved for general use. Phase III clinical trials compare the safety and effectiveness of the new investigational product/medication against the current standard treatment.

Phase III clinical trials usually have a large number of patients, at least several hundred. These studies are often done in many places across the country (or even around the world) at the same time.

Because doctors do not yet know which treatment is better, patients are often chosen at random, (called randomized) to get either the standard treatment or the new investigational product/medication. When possible, neither the research staff nor the patient knows which of the investigational product/medications the patient is getting. This type of study is called adouble-blind study. As with other studies, patients in Phase III clinical trials are watched closely for side effects, and investigational product/medication is stopped if they are too bad. Placebos may be used in some Phase III studies, but they are never used alone if there is already a treatment available that works.

PHASE IV STUDIES

Phase IV studies help to answer any remaining questions. Even if thousands of people have successfully completed a Phase III study are there rare side effects that haven't been seen yet, or side effects that only show up after a person has taken the drug for a long time?

Phase IV studies look at drugs that have already been approved by the FDA. The drugs are already available for doctors to prescribe for patients, but these studies are still needed to answer important questions.

When thinking about taking part in a Phase IV trial, you should know that the drug has already been approved for use. You do not need to enroll in the study to get the medicine. At the same time, the care you would get in these types of studies often is very much like what you could expect if you were to get the drug or treatment outside of a clinical trial. You should be reassured that in taking part you would be getting a form of investigational product/medication that has already been studied a lot and that you would be doing a service to future patients.

Phase IV studies are frequently what we call observational studies.  This means that scientists and researchers are simply looking at some aspect of the disease or medication generally this entails taking blood, urine or cerebrospinal fluid or answering surveys and questionnaires.

SHOULD I PARTICIPATE IN A RESEARCH STUDY?

When trying to decide, first ask yourself some basic questions:

+ Why do I want to take part in a clinical trial?
+ What are my goals and expectations if I decide to take part? How realistic are these?
+ What is the chance of benefit versus risk?
+ What are other possible factors, such as whether I have the time for the scheduled study visits?
+ Will I be compensated for my visits? Does this matter to me?
+ What are my other possible options?
 

Some of these questions may not have clear-cut answers, but they should help you begin thinking about some important issues. Each person's situation is unique, and each person's reasons for wanting or not wanting to take part in a study may be different.  There is no right answer. 

Participating in a research study is completely voluntary.  Once a person signs the Informed Consent they can still change their mind and withdraw at any time.  If you discontinue a study for whatever reason there may be a follow-up visit to make sure you are okay but it is always your decision whether you wish to participate or not.

You can always speak with your provider here at Rocky Mountain MS Clinic or with any of the staff in the Research Office.