Clinical Trials

Clinical trials, what you need to know

• For specific trails related to gastroparesis 

• For all digestive diseases 

 

 

On this page you will learn more information about drug clinical trails, some interesting facts and important questions for you to consider if you are thinking you may want to participate in a clinical study.

What is a clinical trial?
Clinical trials are well defined and controlled scientific studies, held at multiple centers, which help to answer two vital questions regarding advancing a new treatment:
        • Is the treatment safe?
        • Does the treatment work?

Most commonly, the treatments studied are for new drugs, but clinical studies are also done for biologically active agents and for medical, implantable devices, as well as new diagnostic tools.

The approach used for clinical studies is time honored and follows a similar approach worldwide.

Any new medication will have undergone years of lab investigations before advancing to human trials. Once poised for human investigation, the sponsoring company (often a pharmaceutical company) must apply for an Investigational New Drug (IND) application.

Trial design begins.
The trials progress through 3 stages or what are called: phases. In every step, new volunteers are recruited; they are informed regarding the expected risks and benefits; then they are free to either -- choose to decline from the study, -- or consent to participate.

Every effort is made to insure safety for those who participate. In the United States, the Food and Drug Administration sets forth stringent guidelines. Further, an Institutional Review Board (IRB), composed of independent scientists, community advocates and others, must initially approve the clinical trial for their institution, and periodically review the progression of the clinical trial.

Who can participate?
Before you can join a clinical trial, you must meet the criteria for the study. Each clinical trial design has inclusion criteria and exclusion criteria. These guidelines are based upon age, medical history, stage of your disease, ability to understand and consent to the study, and other factors.

In phase I of the clinical trial, usually a small number of healthy volunteers are recruited. Many aspects of the drug are investigated as well as determining dosage ranges.

In Phase II studies, a larger number of volunteers are needed. Anywhere from 10 to 400 people, who actually have the disease for which the treatment is being develop, are asked to participate. In this phase, usually a placebo is used and the patient is unaware (single blinded) if they are receiving active medication or an inactive look alike (placebo).

In Phase III studies, even larger numbers of patients are recruited. In this phase a double blinded and cross-over method is employed. Neither the clinical investigator or the patient know which drug is active and which is placebo.

An informed consent is critical with every phase. The consent process allows you to learn how the test will affect you; what benefit and risks you can expect; how long and what requirements you need to full-fill the study; and finally, that you have the right to discontinue the trial at anytime.

You should be allowed to take the consent form home and discuss it with your family before you sign the document. Further, the consent is on-going and you are encouraged to ask questions throughout the study. You always have the right to withdraw at anytime.

Some clinical trials will reimburse you for expenses associated with participation in the research. Usually this is transportation costs, meals and perhaps even accommodation.

Once all phases of the trial are completed and the volumes of data are evaluated, the sponsor then applies to the Federal regulator for approval to market the drug. This is called a New Drug Application (NDA)

Any new treatment which is approved for marketing continues to be monitored under a post marketing surveillance system.

When a new medication is fully approved for marketing -- then physicians are permitted to prescribe this new drug for other medical indications in which the drug has not been clinically tested. This is called an "off-label" use of the drug.

Off-label uses may mean that your medication will not be covered by insurance providers.

An example of off-label use for treating gastroparesis is erythromycin.

This antibiotic was initially brought to the market for the treatment of infections, but found to have a side-effect of causing the stomach to contract and empty. Doctors prescribe this antibiotic for treating gastroparesis for its stomach emptying side-effects.

Off-label prescribing of medications is done frequently. When these practices become more widespread, it often will stimulate clinical investigations to establish evidence for new uses of the medication. If the drug is not a generic medication, then a sponsoring company frequently will help support further clinical inquiry. The sponsoring company may also take a further step and apply to the federal regulators for a lateral approval to market their medication for the new indication. Clinical evidence must be demonstrated for granting this lateral approval.

It can take a decade or longer for a new drug to progress from the initial patent application to pre-clinical studies, through the various clinical trials, and then may take up to 3 years for the federal regulator to review all the data before an NDA is granted.

Generic drugs enter into the market once the patent life of the new drug has expired. Patent life in the United States is 20 years.

Did you know...
Currently, there is not one drug in North America which has specifically been brought to the market for the treatment of gastroparesis.

The National institutes of Health reports: 5 million Americans suffer from gastroparesis.

All drugs currently used to medically treat gastroparesis are prescribed "off-label".

Should I Participate?
Taking part in a clinical trial means that you get to play an active role in your own health care and gain access to a new treatment which is not yet available to the general public. Further, it usually means you gain access to medical care from some of the top physicians for your disease. But, perhaps most importantly, you have the potential to play a vital role helping others by assisting in bringing a vital new treatment to the market for all.

Clinical trials do potentially have risks, though this is greatly minimized through a careful patient selection process and careful monitoring.

Important questions to ask:

• What is the purpose of the study?

• Why does the treatment research team think this treatment will work?

• What side-effects might I experience?

• What benefits can I expect?

• How long will the trial last?

• How often do I need to return for follow-up?

• What tests will be done to monitor my progress?

• Who will provide my medical care once the clinical trial ends?

• Will I be hospitalized during the study?

• Will the clinical team work with my doctor?

• What are my responsibilities?

• Will I find out the results of the trial?

• Will I have to pay for anything?

• Will my insurance cover expenses?

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