WHAT ARE CLINICAL TRIALS?

A clinical trial  are often referred to as drug studies, drug trials, clinical research trials, medical device trials, medical research studies and so forth. In general, these trials have as their goals to provide information regarding the safety and the efficacy of a medication, a medical procedure or a medical device.

The efficacy of a drug or a device is measured in various ways depending on the focus of a specific trial. In some cases it can be measured physically thru blood pressure or insulin levels, or in other ways such as daily report of pain, psychological assessments or by Xray or MRI. The safety of a drug is determined by the assessment of physical and psychological symptoms that occur during the course of the trial. These assessments help answer questions about side effects, superiority to current treatments and degree of positive effect.

The FDA (Federal Drug Administration) ultimately decides weather a drug should be made available to the public. In general, the FDA insists that a new drug must be safe and effective. The safety aspect is clear. The efficacy may be demonstrated by the proposed drug being superior to currently available treatments, or by providing equally effective treatment with fewer side effects.

TYPES OF TRIALS

There are three different types of clinical trials. Successful completion of these trials is required before a drug can gain regulatory approval by the FDA. The trials are divided into four categories: Phase I, II, III and IV.

PHASE I

Phase I  clinical  are the first to be conducted in humans. Prior to this, the study drug (compound) has been thoroughly tested in a laboratory and used in animal populations. These trials are the first time humans are exposed to the drug (compound). There are often as few as thirty study participants in these trials. These are small trials that seek to understand the safety of the drug, the ways in which the drug is handled in the body (pharmacokinetics and pharmacodynamics) and to establish potential dosage ranges. Normal healthy humans are usually the target population for these trials. There are exceptions to this such as oncology trials where cancer patients must be the study subjects, or populations with other specific diseases.

The first patients will receive a small amount of the drug and as the trial proceeds study subjects will receive an increasing dose. The effects of the dose are monitored in various ways such as laboratory testing (blood draws), vital signs, and ECGs. Because of patient safety, these trials are usually conducted on an inpatient basis.

PHASE II

Phase II trials look to the population of individuals who suffer from the illness targeted by the drug. About 70% of Phase I trials continue into phase II trials. Usually these trials enroll several hundred participants. Phase II trials again look to the safety of the drug as well as efficacy and dosage of the drug. In other words, how well does the drug work (efficacy)? At what dose is it most effective (phamacodymanics)? What are its side effects? Can these side effects be managed?  Is this drug as good as or better than currently available treatments?

PHASE III

If these questions are answered positively or if they are a bit uncertain but indicate further investigation, then Phase III trials are begun. These trials often enroll a thousand or more patients. They are also placebo controlled trials. They often have a currently available treatment as a comparator for the study drug. And so, these trials will have three arms (placebo, study drug, comparator). Often the trials might have four arms, two being different doses of the study medication.

In Order to make certain that the information collected from the trial is objective an unbiased, these trials are randomized and blinded. This means that we do not know which participant is on which arm of the trial. This information is available if there is a medical crisis. Otherwise, it is only made available after the trial is complete (unblinded).

In Phase III trials the larger population may be composed of people in many different countries.  On occasion, these trials will have an extension. This means that after a trial is complete, participants may be able to continue on the study drug  for a period of time, often until the drug becomes available to the public (after regulatory approval by the FDA).

PHASE IV

Phase IV trials are large and can enroll many thousands of patients. Primarily, these trials are conducted to monitor the safety of the newly approved over a longer period of time.

ARE STUDY DRUGS SAFE ?

Clinical trials are not thought to be treatment guarantees, nor are clinical trials clearly determined to be safe and efficacious. This means that there is always a degree of uncertainty. The degree is greater when the drug has been tried in a fewer number of humans. The larger the number, the more is know about the drug and its workings in the human body.

This information is made know to each study participant in the INFORMED CONSENT, and if anything unanticipated occurs during the course of the trial, each participant will be informed.

In general, the potential harm that may come to study participants is anticipated. A lot is know about each drug and what its potential side effects may be. And so before enrolling in a trial, you are made aware of these know or anticipated side effects to the medication. This is research and so until a drug is thoroughly tested. There will always be a degree of uncertainty. This is the RISK involved in all Clinical Trials.

IS MY INFORMATION GOING TO BE CONFIDENTIAL?

In all trials, the information collected about each study participant is revealed to a limited number of entities. These are disclosed at the beginning of the trial. This information is linked to the study participant by their initials and Date of Birth. The FDA requires that we obtain medical records and record contact information separately. This provides evidence of the unique nature of each participant as well as the ability to contact each in the future should this be necessary. And so, we have your information with your identity. You can authorize its release to your physician are to any other agency. The Pharmaceutical company (Sponsor) will not have access to your identity but only to the data supplied about your physical and medical health before and during the trial.

WHAT HAPPENS IF I GET SICK DURING THE TRIAL?

If you become ill during the trial and this illness is related to the study drug, the Sponsor will pay for any and all medical treatment provided to you until the illness is resolved (Indemnification). Of course, if your illness is not related to study drug, you must utilized your private health Insurance.

No one wants any harm to come to any study participants.  Their goal is to establish better treatments for individuals.

WHAT ARE THE ADVANTAGES TO BEING IN A TRIAL?

In order to make in informed choice about participating in a clinical trial, you must weigh the risks and benefits of a clinical trial. This means that from the outset you are aware of what may likely happen during the course of the trial, and you feel it is worthwhile to move forward.

The risks are the side effects, the chance of being on placebo,  and, finally, the ever present degree of uncertainty.

The benefits can be efficacious treatment, helping the advance of science and thereby helping others, thorough health assessment and physical monitoring before and during the trial and financial compensation for your time and travel.

HOW DO I APPLY TO BE IN A CLINICAL TRIAL?

Its quite easy. Please call us (504-363-7448). Our staff will discuss the trial that interests you and see if you qualify. Often, people have more that one diagnosis and may be able to participate in several trials. You cannot participate in more than one trial at a time. You can participate in one, complete and enter another. Usually there is a waiting period of 30 days between trials. This is regulated by the Sponsor of trials.

In every case, we will be pleased to add your name and address to our data base so that we can contact you in the future.

WHAT IS EXPECTED OF ME DURING THE TRIAL?

It is of the greatest importance that all study participants realize that they are collaborating with us to understand the effects of this study drug. This means that each is primarily responsible for taking their medication as they are told, attending all visits, answering all questions and telling us the absolute truth about you experience physically and emotionally during the trial.

Study drug is referred to as INVESTIGATIONAL PRODUCT. It is crucial that each study participant maintain the study drug in a safe place, take the medication exactly as the Doctor orders it, and to return all empty containers. This is the only way we can document that you have taken the correct amount of medication and in the correct manner.

You may have to avoid taking certain medications and ingesting certain food or drink items. Pregnancy should always be avoided.

We DO NOT EXPECT that you will get better or get worse. We do not know. We are relying on you to tell us what happens to you during the trial. The truth is what we are looking for it can by positive or negative.

If you give us biased information  based not on what you are actually experiencing but based  on some other belief that you think we expect you to get better, then the information you give us will be inaccurate, and would do more harm than good.  In fact, in order to get approval, each study drug must demonstrate that it is superior to placebo. So, if you are on placebo and you are telling us you are getting better, than it will be more difficult for the drug to get approval because the biased information you reported will make it more difficult for the data on the study drug to achieve statistical superiority over the placebo.

In general participating in trials implies medical monitoring of every kind. You can anticipate laboratory testing, ie blood and urine sample collection, screen for dangerous and illegal drugs, vital signs ie temperature and blood pressure, occasional Electrocardiogram, sometimes MRI or x ray, physical exam, and verbal assessments.

HOW OFTEN DO I HAVE TO COME TO THE CLINIC?

The protocol establishes the frequency of visits. In general, there is a screening visit during which assessments are make a laboratory samples collected. A week or two later, if all is well, the baseline or randomization visit occurs (initiation of study drug). Most often the next two visits are weekly. In some trials weekly visits may continue for two months. Most often, the space to every other week, and eventually every month.

CAN I CONTINUE TO SEE MY OWN DOCTOR?

We are happy to work with your Doctor, and provide him/her with any information they might need or request. There is no conflict with your being followed with your Doctor. We ask that you make him aware that you are in a clinical trial and that before he changes your medications, that he contract the Investigator here at the Clinic. This is important because there are some medications that are not allowed in the trial, and another drug could be prescribed that could have the same therapeutic effect.

CAN I GET TRETMENT ELSEWHERE?

No Clinical Trial offers any guarantee of treatment. At the outset you will know that there are most likely other forms of treatment for your illness. These treatments will likely be generally available in the community. To participate in a clinical trial implies that you may not receive active medication for treatment of the targeted illness.

WHERE CAN I GET MORE INFORMATION ABOUT CLINICAL TRIALS?

You can get more information about clinical trials at the US Government’s clinical trial’s registry website at  http://www.clinicaltrials,gov/ct/info/resources.

WHO ORGANIZES  CLINICAL TRIAL?

All clinical trials involve the efforts of different organizations. Each has its own task. There is the SPONSOR. This is the pharmaceutical company that has produced the study drug and who supports the series of trials that  could result in approval of the study drug by the FDA. The FDA (Federal Drug Administration) is an agency of our government that determines wheather a drug should be made available to the general public. This means that they have to evaluate all the information about the study drug and determine if it is safe and efficacious. The information upon which this decision is made is provided to the FDA by the SPONSOR. As part of the approval process, the FDA must determine if the SPONSOR has conducted the clinical trials according to the principals the FDA has mandated.

Often a SPONSOR may be a very small company and may not have the staff and resources to conduct a clinical trial. They then form a relationship with a Clinical Research Organization (CRO). These are often very large world wide corporations that conduct clinical trials for Sponsors.

The Investigational Review Board (IRB) is an independent group of research and community professionals. They are regulated by the FDA. They are mandated to insure patient safety before and during and after the close of a clinical trial. They are to approve the Informed Consent document. This document contains their contact information (You may contact them at any time for any reason in order to gain more information or to report a concern you may have about the conduct of the clinical trial). The IRB approval indicates that the document accurately and thoroughly details the risks and benefits of participating in a clinical trial. During the course of the trial all information about side effects and adverse events are reported to the Investigational Review Board. Should any unforeseen medical events occur, the Review Board will contact (through the Study Site) each study participant to inform them on the new significant event. The Sponsor pays the IRB

The Study Site is the final group. The study site has a contract with the Sponsor or the CRO. The site agrees to conduct a clinical trial according to the dictates of the study protocol. The protocol delineates all the actions and procedures that take place during the conduct of the clinical trial. The Sponsor pays the Site to conduct the clinical trial.

WHAT IS A PLACEBO?

A placebo is a sugar pill (it does not actually have sugar in it), a tablet that contains no active medication. In many clinical trials the FDA requires that a placebo be used in order to assess the efficacy of the study drug. In order to get approval by the FDA, the study drug must be more efficacious than the placebo group.

The extent of the possibility of your being on a placebo is discussed in the Informed consent.

CAN I LEAVE A TRIAL?

Participation in a Clinical Trial is always voluntary. This means that anyone at any time and for any reason can choose not to participate in any trial. If you decide to discontinue, we ask only that you return all study drug to us and allow us the opportunity to conduct end of trial procedures which are important for your safety.

WHO DO I CONTACT?

Site staff and the Principal Investigator (Doctor) are available to you on a 24 hour a day basis. They will provide you with contact information.

DO I GET PAID?

Each study participant is financially rewarded for their participation in a clinical trial. In general the amount is determined by the length  and frequency of study visits, and the extent of the risk (Phase I trials involve more risk than Phase II etc.). The amount that you will be paid is clarified in the Informed Consent.

ARE THEIR THINGS I CANNOT DO DURING THE TRIAL?

During a trial you will not be able to take certain medications, and sometimes must abstain from certain types of food or drink. Alcohol and street drugs are strictly prohibited. In most trials each person must avoid pregnancy. This requires each participant to use one or more methods of birth control.

WHAT IS AN INFORMED CONSENT?

An Informed Consent is a document that you must sign that the beginning of  any Clinical Trial. This document has been approved by an Investigational Review Board (a group of experts in research and patient safety) and spells out clearly (and in language that all can understand) the risks and benefits of participating in this specific clinical trial.

You should take you time in reviewing this document. You may take it home with you and discuss it with your family or with your doctor. You may ask any questions and speak to the Doctor in charge of the trial. Once your questions and concerns have been addressed, all parties sign the document. No procedures related to the conduct of the trial can be performed prior to the time you sign the informed consent.