The Different Clinical Trials of Multiple Sclerosis Drugs

Lisa Emrich Health Guide February 09, 2010
  • George Jelinek shared some valuable information in his post - The critical part of the jigsaw of the diet in MS is Professor Swank’s work - which touched on the nature of clinical trials.

     

    When we think of clinical trials, the image of pharma-sponsored drug trials immediately come to mind.  Perhaps this is because we hear most about drugs in trials, drugs which have been tested for use in MS, and drugs which receive that important FDA approval before hitting the market.  But drug trials are not the only types of clinical trials.

     

    Clinical trials fall into several categories, including:

     

    Treatment trials: These studies test new treatments for diseases or conditions. These treatments can include experimental treatments, new drugs, new combinations of drugs, or new approaches to surgery or radiation therapy.

     

    A treatment trial may also study "off-label" uses for an existing FDA-approved treatment. Off-label means the drug is being use as a treatment for a different condition (or indication) than the drug was originally approved to treat.

     

    Screening trials: These studies test for the best ways to detect diseases and conditions.  These trials involve participants who have no symptoms of the condition and can be done on the general population. Or they involve a group of people who have a 'higher than normal' risk of developing a disease or condition.

     

    Prevention trials: Prevention trials look for better ways to prevent disease in people who have never had the specific disease being studied or to prevent a disease from returning. Approaches to prevention may include lifestyle changes, vaccines, vitamins, minerals, or medicines.  As with screening trials, prevention trials can be carried out on the general population, or on a specific high risk group.

     

    Diagnostic trials: These studies are conducted to find better tests or procedures for diagnosing a particular disease or condition.  Diagnostic trials usually involve people who already have signs of the disease or condition.

     

    Quality of Life trials (or Supportive Care trials): These studies explore how your treatment or illness affects you and often seek ways to improve comfort and the quality of life for individuals with a chronic illness.  Many trials include a quality of life assessment as part of the main trial, which may be conducted separately as a 'quality of life study'.

     

    Epidemiology studies: These trials look at causes and patterns of disease, for instance whether a particular factor causes cancer or not, and are primarily observational.  There are three types of observational studies - cohort studies, case control studies and cross sectional studies.  (visit CancerHelpUK for more information)

     

    More specific types of treatment trials:

     

    Pilot studies: Pilot studies (or feasibility studies) are small-scale versions of larger treatment trials. The format is the same but involve only a small number of people and can be completed more quickly.  Not all trials need a pilot study first, but they useful in testing the trial design (protocol) so that investigators can make changes before the larger trial begins.

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    Blind trials: A blind trial is a trial where the participants do not know which treatment they are getting - study drug, standard treatment, or placebo.  All patients receive identical injections or tablets, so they cannot tell which treatment they are getting.

     

    Double blind trials: A double blind trial means that neither the researchers nor the patients know which treatment the participants are receiving. The computer gives a code number to each patient and then the code numbers are assigned to the different treatment groups. Your treatment arrives with your code number on it, so neither you nor your doctor knows if it is the new treatment or not.

     

    The list of patients and their code numbers is kept secret (or blinded) until the end of the trial. If an emergency situation arises, the researchers would be able to find out which trial group a specific patient was in.  But in general no one knows until the trial is complete.  A double blind trial is designed to limit bias in the clinical researchers who work directly with the patients.

     

    A randomized trial that has a control group is called a “randomized controlled trial.”  Sometimes people in the control group take a dummy treatment, called a placebo.  But more often the control group takes the standard treatment for the illness against which the new therapy is being tested.  Avonex or Rebif (interferon beta-1a) are common standard treatments used in MS drug trials.

     

    Sequential trials: In this type of trial, results are worked out as you go along, rather than after the whole study closes. You are treated one by one (sequentially), in one of several groups. Neither you nor your doctor will be able to decide which group you are in. Each group has the same treatment, but in different doses or in different ways.

     

    The first person has their treatment in group 1. The next person has their treatment in group 2, and so on. When all the groups have treated their first patient, they are filled again, one by one. This carries on until researchers can either see which group has the best results, or that there will not be any difference. Sequential trials can show results earlier than other trials, so need fewer people to take part.

     

    Overview studies: Trial overviews, sometimes called meta-analyses, are studies that combine all the results from multiple phase III trials of a new treatment.  The idea in combining results is to get a broader picture of how well a treatment works. The more data (information) you have, the more accurate the results are likely to be.

     


    SOURCES:

    Understanding Clinical Trials from ClinicalTrials.gov

    Types of Trials from CancerHelp.org

     

    MORE INFORMATION ON HEALTHCENTRAL:

    MS Research and Registries

    Clinical Trials I: What You Need to Know

    Clinical Trials II: Phases and Protocols

    Clinical Trials III: Patient Participation

     

    Lisa Emrich is author of the blog Brass and Ivory: Life with MS and RA and founder of the Carnival of MS Bloggers.