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Clinical_Trials Powered By Docstoc
an Expert:      volume 2/spring 2007

Clinical Trials                                                                                                 Joyce Ingold, RN, MSN, OCN.

 Kim Johnke (KJ): What are Clinical Trials?
 Joyce Ingold (JI): A clinical trial is a
                                                  “Ultimately, to bringclinical trial participants
                                                   are helping
                                                                        potential new life-saving
 research study in which human volunteers
                                                   treatments to other individuals facing cancer.
 help investigators answer specific medical
 questions. Treatment trials test for safety
                                                   staffed with a research nurse who keeps                 (where comparison is necessary) participants
 and efficacy of investigational new drugs
 (IND) for diseases and conditions. In the         close track of patients. Because the IND is             are randomized into treatment groups
 United States, clinical trials are regulated      being tested in humans, it is necessary to              (trial arms), and the PI compares
 by the National Institutes of Health (NIH)        know every detail about the patient’s                   differences between groups. For example,
 and Food and Drug Administration (FDA).           experience. In the long-term, the data we               one trial arm may receive the SOC, while
 All drugs – whether over-the-counter or           collect from participants in clinical trials            another arm receives SOC therapy plus
 prescription – must pass through clinical         will help establish the minimum effective               the IND. In this type of trial, all cancer
 trials before receiving FDA approval and          dosage, as well as which cancer(s) it will              patients would receive the standard of care.

                                                                                                                                                                    THE LUSTGARTEN FOUNDATION FOR PANCREATIC C ANCER RESEARCH
 becoming available for public consumption.        effectively treat. Ultimately, cancer clinical
                                                   trial participants are helping oncologists to           KJ: Why are Clinical Trials particularly
 As a form of research, clinical trials follow     bring potential new life-saving treatments to           important to people with pancreatic cancer?
 an action plan called a protocol, which           other individuals facing cancer.
 is carefully designed by a principal                                                                      JI: Unfortunately, pancreatic cancer is
 investigator (PI) and approved by an              KJ: How do Cancer Clinical Trials differ                often detected after it has metastasized.
 Institutional Review Board. Clinical              from non-cancer Trials?                                 The standard of care therapy for pancreatic
 trials exist in all areas of medicine.                                                                    cancer is Gemcitabine (Gemzar). To date,
                                                   JI: Generally, individuals are more likely              Gemzar has increased survivability by a matter
 KJ: How do Clinical Trials benefit individuals    to participate in non-cancer clinical trials            of months, and investigators want to improve
 in the short-term and the long-term?
                                                   as first-line therapy, whereas cancer                   this rate. Therefore, many pancreatic cancer
 JI: It is important to emphasize that             clinical trials are usually reserved for when           patients seek clinical trials as first-line treat-
 patients do not need to join a clinical trial     the cancer no longer responds to the                    ment. Scottsdale Healthcare offers first-line
 to receive quality cancer care, and not all       standard of care (SOC) therapy. Also,                   Phase I and II clinical trials for pancreatic
 trials offer direct benefits to patients. At      in quality cancer clinical trials, placebos             cancer patients who have never received
 Scottsdale Healthcare, we find that               are usually not used. Be sure to ask your               chemotherapy. Clinical trials are helping us
 a short-term benefit of clinical trials is        doctor whether a placebo will be used in a              learn which other drugs can be combined
 personalized care. Also, clinical trials are      particular trial. In a cancer clinical trial,           with Gemzar to improve survivability.
*Words in bold are defined in the glossary.                                                                                                     (continued) |                                                                      | 1111 Stewart Avenue • Bethpage, NY 11714 | 516-803-2304   1
    an Expert:                         Cancer Clinical Trials
    KJ: How does the Clinical Trials process       KJ: What is meant by ‘accrual?’                   and cooperate with staff, and to participate
    work?                                                                                            in their health care decisions. Participants
                                                   JI: The sponsoring agency pre-determines          also have a responsibility to disclose any
    JI: The Clinical Trials process involves       the total number of participants that a           and all types of medications or drugs that
    four distinct phases. Phase One brings         trial will accommodate. The process of            they are taking (including herbal, over the
    an IND to humans, and the PI seeks to          enrolling participants is called ‘accrual.’       counter and alcohol). They are also asked to
    understand the safest dosage required.         The accrual process works in phases, where        promptly and honestly report unexpected
    Phase One trials often include patients        several participants are accrued together         problems or concerns to the treatment team.
                                                   into a cohort. Cohorts are ‘mini-groups’
    with various forms of cancer to learn which
    tumor types have the most consistent
                                                   of participants who start a trial together        KJ: Who comprises the ‘treatment team?’
                                                   and receive the same treatment dosage.
    response to the IND. These early-phase                                                           JI: A host of professionals may comprise
    trials usually involve small groups of         Participants cannot move between cohorts          a treatment team, including medical
    volunteers (20-30) and last one to two         during the study. For example, a patient          oncologists, radiation oncologists, surgical
    years. During Phase Two, the PI continues      who starts a clinical trial drug at a dosage      oncologists, pathologists, nurses, social
    testing the safety of the IND and begins       of 10 milligrams will receive this dosage         workers, nutritionists, psychologists and
    to evaluate how well the new drug works.       until the cancer stops responding. If the         more. The principal investigator (PI) is
    Phase Two trials usually focus on a specific   patient decides to leave the 10-milligram         the physician who designs and conducts
    type of cancer. This phase brings the drug     cohort, s/he could enroll in a different          the trial. In our office, we also have a Nurse
    to a larger number of participants.            trial, but could not skip to a new cohort         Practitioner who functions as a primary
                                                   within the same trial.                            care provider in collaboration with clinic
    Phase Three compares data from people                                                            physicians. Each clinical trial also has a
    taking the IND with data from people           KJ: What happens if, during the course of         research nurse. This is the person with
    receiving standard treatment. These trials     a trial, a participants’ medical status drops     whom participants typically develop
    depend on large groups of volunteers           below the baseline requirement? Could this        a close rapport, because the research nurse
                                                   person remain in the trial?                       keeps a very close eye on study partici-
    (up to 3,000) to confirm efficacy, monitor
    side effects, compare commonly used                                                              pants. S/he will ask questions about issues
                                                   JI: We will do everything possible to keep        such as diarrhea, eating habits, nausea,
    treatments and collect information that        a person eligible for a trial, including blood
    will allow the IND to be used safely.                                                            pain, etc. A pharmacist assists in monitor-
                                                   transfusions and/or providing support to
    The time frame for Phase Three trials is                                                         ing additional medications that an individ-
                                                   maintain performance status. If we cannot
    usually several years. After Phase Three                                                         ual may be taking, such as antiemetics,
                                                   maintain an acceptable performance status,
    trials are completed, the company may                                                            antidepressants and narcotics for pain.
                                                   then an individual would need to discon-
    apply for FDA approval of the IND.                                                               Supportive services will often include a
                                                   tinue participation in the clinical trial.
                                                                                                     social worker to help address various social,
    Phase Four trials represent the final phase    KJ: What are the rights and responsibilities      emotional and financial concerns.
    of the trials process, and are conducted       of Clinical Trial participants?
                                                                                                     KJ: What happens if, for any reason,
    after the treatment has been FDA
                                                   JI: Every clinical trial participant has a Bill   a clinical trial is discontinued? Can the
    approved. Phase Four trials seek to
                                                   of Rights, which includes receiving a copy        patient continue to receive the IND?
    ensure that the newly approved drug
                                                   of the signed informed consent docu-
    is indeed effective.                                                                             JI: A clinical trial may be stopped when
                                                   ment. Informed consent is the process by
                                                   which potential trial participants learn key      data overwhelmingly demonstrates a
    KJ: Can anybody join a Clinical Trial?                                                           benefit to the IND and the trial sponsor
                                                   facts, including potential risks and benefits
                                                   (See Box) before deciding whether to join.        proceeds to FDA application. A trial
    JI: Every clinical trial has eligibility                                                         could also be stopped because it’s not
    criteria and inclusion/exclusion               The process includes reviewing a document
                                                   that explains key details of the study, contact   demonstrating a benefit. If an individual’s
    criteria; these criteria ensure that                                                             clinical trial is discontinued, s/he can
                                                   information, and required procedures
    researchers are comparing ‘apples to                                                             apply for continued/compassionate use
                                                   (patient responsibilities). The document is
    apples.’ Patients must also meet a                                                               of the study drug, or begin a new clinical
                                                   not a contract, and participants may leave a
    performance status requirement to                                                                trial after meeting a new set of eligibility
                                                   study at any time, and for any reason.
    be part of a clinical trial. Examples of                                                         criteria. Between clinical trials, however,
    performance status criteria include, but       In order to preserve the quality of the           there is a ‘wash-out’ period lasting roughly
    are not limited to, various blood values,      research data, trial participants are             28 days. This refers to a required time
    kidney and liver function, and activities      asked to provide honest and complete              period from the last day of treatment
    of daily living (ADL).                         information, to comply with the protocol          before a new treatment can begin.

2 |                                                                                        THE LUSTGARTEN FOUNDATION FOR
        KJ: Would a study participant eventually                     KJ: Are there additional considerations               KJ: Do you have any final thoughts about the
        ‘complete’ a trial? What are end-points?                     before joining a trial?                               role of clinical trials in pancreatic cancer?

        JI: Every trial participant is evaluated at                  JI: Motivation to join a clinical trial is            JI: I think it is important that no patient
        set times (ex., every six to eight weeks)                    patient-specific. Some people will remain             ever feel abandoned. Even if a patient is not
        with various tests. If it is determined that                 on clinical trials because they feel it’s             eligible for clinical trials, we can provide
        the cancer has progressed, they may not                      important to take an active role in their             standard of care treatment and/or palliative
        be continued on the trial. Instead, we will                  health care. Others feel one trial is all             support to improve quality of life. In my
        work to find another. If at that evaluation                  they can do. Many personal lifestyle                  opinion, there’s always something ‘more’ to
        point, we find that the disease either has                   factors and individual preferences need               do. New clinical trials open every day, and
        not progressed or has regressed, we will                     to be considered by patients wishing to               it never hurts to ask whether there is
        continue their participation on the trial                    determine whether a trial is the right                something ‘more.’
        until the next evaluation period. I know                     course of action. People often ask me,                      FIND CANCER CLINICAL TRIALS
        people who have been on a clinical trial                     “How do I know if I should try a clinical
                                                                                                                                    The Lustgarten Foundation
        for years (even after the trial has closed                   trial?” My answer is, “You don’t know                         Clinical Trial Matching Service
        to accrual) because they demonstrate no                      until you start investigating the trial.”                            With EmergingMed
                                                                     The key is that patients should do what                               1-800-535-1867
        disease progression. End-points refer to                                                                               
        the parameters that will be measured to                      they feel is best for them.
        evaluate the research study’s objectives.

        KJ: How are Cancer Clinical Trials funded,                    Potential Benefits & Risks of
        and are there any costs to participants?
                                                                      Participating in Cancer Clinical Trials
        JI: The federal government is the nation’s                    BENEFITS                                            RISKS
        largest sponsor of clinical trials. Cancer
        clinical trials may also be sponsored by                      ✓ Gaining early access to potential new             ✓ Possibility of unexpected side effects
                                                                        treatments for pancreatic cancer                    from the experimental drug
        drug companies, private agencies, and/or
        by institutions themselves. Each insurance                    ✓ Being an ‘informed consumer’ and                  ✓ New treatments do not necessarily
                                                                        taking an active role in your health care           offer greater benefit than the
        company is different, and patients should                                                                           standard of care
        check with their individual carriers for                      ✓ Receiving individualized attention
                                                                        and close monitoring by an expert                 ✓ Possibility of added time and costs
        specific rules and guidelines regarding clinical
                                                                        treatment team                                      (ex., travel, housing, expenses not
        trial coverage. Typically, insurance covers                                                                         covered by insurance) for patients
        SOC therapy administered throughout the                       ✓ Potential for the experimental drug
                                                                                                                            and/or caregivers
                                                                        to provide greater benefit than the
        clinical trial, and the trial sponsor covers                    standard of care
        non-standard care. Advocacy resources
                                                                      ✓ Helping to advance medical treatments
        (See Box) are available to assist patients                      for all pancreatic cancer patients
        in understanding their insurance rights.

                    ADDITIONAL RESOURCES
                                                                      Bill of Rights for Clinical Trial Volunteers
                       Scottsdale Healthcare
                        Cancer Clinical Trials                        ✓ To be told the purpose of the clinical trial       ✓ To be allowed to ask any questions about
                                                                                                        the trial before giving consent and at any
                                                                      ✓ To be told all the risks, side effects or
                                                                                                                             time during the course of the study
                    National Cancer Institute                           discomforts that might be reasonably
                        1-800-4-CANCER                                  expected                                           ✓ To be allowed ample time, without
                                                                                                     pressure, to decide whether to consent
                                                                      ✓ To be told of any benefits that can be
                                                                                                                             or not to consent to participate
                   Patient Advocate Foundation                          reasonably expected
                         1-800-532-5274                                                                                    ✓ To refuse to participate, for any reason,
                                                                      ✓ To be told what will happen in the study
                                                                                            before and after the trial has started
                                                                        and whether any procedures, drugs or
             Center for Information & Study on                          devices are different than those that are          ✓ To receive a signed and dated copy of
               Clinical Research Participation                          used as standard medical treatment                   the informed consent form
                        1-888-CISCRP3                                 ✓ To be told about options available and             ✓ To be told of any medical treatments
                                                how they may be better or worse than                 available if complications occur during
           Informed Consent:The Consumer’s Guide to the                 being in a clinical trial                            the trial
           Risks and Benefits of Volunteering for Clinical Trials.
           By Kenneth Getz & Deborah Borfitz                             Source: Getz, K., & Borfitz, D. (2002). Informed Consent. Boston, MA:Thomson CenterWatch

PANCREATIC C ANCER RESEARCH                                                                                         | 1111 Stewart Avenue • Bethpage, NY 11714 | 516-803-2304   3
Antiemetic – A drug used (or tending to)                   First-line Therapy – The first type of therapy
                                                                                                                    Performance Status – The performance status
prevent or check vomiting.                                 given for a condition or disease.                        semi-quantifies the ability of a patient to perform
                                                                                                                    daily activities.This semi-quantification is very
Cohort – A group of individuals with some                  Inclusion/Exclusion Criteria – The medical               helpful in clinical trials in assessing the state of
characteristics in common.                                 standards determining whether a person may or            health of patients under treatment.The performance
Compassionate Use – A method of providing                  may not be allowed to enter a clinical trial.These       status also plays a role in determining whether a
experimental therapeutics prior to final FDA               criteria are based on such factors as age, gender,       patient can tolerate intensive therapy.
approval for use in humans.This procedure is               the type and stage of a disease, previous treat-
used with very sick individuals who have no                                                                         Placebo – An inert or innocuous substance used
                                                           ment history, and other medical conditions. It is
other treatment options. Often, case-by-case                                                                        especially in controlled experiments testing the
                                                           important to note that inclusion and exclusion           efficacy of another drug.
approval must be obtained from the FDA for                 criteria are not used to reject people personally,
“compassionate use” of a drug or therapy.                  but rather to identify appropriate participants          Principal Investigator – The individual who has
Dose – The measured quantity of a therapeutic              and keep them safe.                                      primary responsibility for designing the protocol,
agent to be taken at one time.                                                                                      selecting the investigation team, carrying out the
                                                           Informed Consent – The process of learning               scientific investigation, and reporting the results.
Efficacy – The maximum ability of a drug or                the key facts about a clinical trial before deciding
treatment to produce a result regardless of                                                                         Protocol – A detailed plan of a scientific/medical
                                                           whether or not to participate. It is also a continuing
dosage. A drug passes efficacy trials if it is effective                                                            experiment, treatment, or procedure.
                                                           process throughout the study to provide informa-
at the dose tested and against the illness for             tion for participants.To help someone decide             Randomization – Process that reduces the
which it is prescribed. In the procedure mandated                                                                   likelihood of bias by assigning people to treatment
                                                           whether or not to participate, the doctors and
by the FDA, Phase II clinical trials gauge efficacy,                                                                groups by chance alone (randomly).When groups
and Phase III trials confirm it.                           nurses involved in the trial explain the details of
                                                           the study.                                               are created by random assignment, individual
Eligibility Criteria – The clinical and demographic                                                                 characteristics are less likely to make the results
characteristics that define which persons are              Institutional Review Board (IRB) – A committee           inaccurate.
eligible to be enrolled in a trial.                        of physicians, statisticians, researchers, community     Research Study – Investigation or experimentation
                                                           advocates, and others that ensures that a clinical       aimed at the discovery and interpretation of facts,
End Points – Overall outcome that the protocol
                                                           trial is ethical and that the rights of study partici-   revision of accepted theories or laws in the light
is designed to evaluate. Common endpoints are
toxicity, disease progression, remission, complete         pants are protected.All clinical trials in the US        of new facts, or practical application of such new
response, partial response, etc.                           must be approved by an IRB before they begin.            or revised theories or laws.
FDA Approval – In the U.S., the Food and Drug              Investigational New Drug – A new drug, antibiotic        Standard of Care – Treatment regimen or medical
Administration (FDA) must approve a substance              drug, or biological drug that is used in a clinical      management based on state-of-the-art patient care.
as a drug before it can be marketed.The approval           investigation. It also includes a biological product     Treatment Trials – Refers to trials which test
process involves several steps including pre-clinical      used in vitro for diagnostic purposes.                   new treatments, new combinations of drugs, or
laboratory and animal studies, clinical trials for                                                                  new approaches to surgery or radiation therapy.
safety and efficacy, filing of a New Drug                  Metastasis – The spread of a disease-producing
Application by the manufacturer of the drug,               agency (such as cancer cells or bacteria) from the       Trial Arm – Any of the treatment groups in a
FDA review of the application, and FDA                     initial or primary site of disease to another part       randomized trial. Most randomized trials have two
approval/rejection of application.                         of the body.                                             "arms," but some have three "arms," or even more.
Sources: and MedLinePlus

an Expert:                                                               About The        Lustgarten Foundation
contributor                                                The Lustgarten Foundation For Pancreatic
                                                           Cancer Research is a non-profit organization
                                                                                                                     In addition to its research funding activities,
                                                                                                                     The Lustgarten Foundation provides
                                                           dedicated to advancing the diagnosis,                     comprehensive patient information
                                                           treatment, cure and prevention of this                    materials at no cost to patients and the
Joyce Ingold serves as Patient Care                        disease.The Foundation was named for                      professionals who serve them.
Coordinator for TGen Clinical Research                     Marc Lustgarten,Vice Chairman of
                                                           Cablevision Systems Corporation, who                      For additional information on the work
Services (TCRS), located at Scottsdale
                                                           was diagnosed with pancreatic cancer.                     of The Lustgarten Foundation, visit
Healthcare. Joyce is an Oncology Certified                                                                 
Nurse with more than 21 years of experience.               The Lustgarten Foundation concentrates on
She is a member of the National Oncology                   stimulating the scientific community to conduct            Supported by an educational grant from
                                                           the research necessary to find a cure for
Nursing Society, Phoenix Oncology
                                                           pancreatic cancer.Today,The Lustgarten
Nursing Society, and the American                          Foundation is the nation’s largest private
Nursing Association.                                       supporter of pancreatic cancer research.

                                            1111 Stewart Avenue
ask                                         Bethpage, NY 11714

an Expert:                                  516-803-2304

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