Clinical trials are research studies conducted to test how well new medical approaches work in cancer patients. Each study answers scientific questions and tries to find better ways to prevent, screen for, diagnose, or treat a disease. Patients who take part in cancer clinical trials have an opportunity to contribute to expanding the knowledge and progress of cancer treatments.
Before a new treatment is tried with patients, it is carefully studied in the laboratory. This research points out the new methods most likely to succeed and, as closely as possible, shows how to use them safely and effectively. But this early research cannot predict exactly how a new treatment will work with patients.
With any new treatment, there are possible risks and benefits. There may also be some risks that are not yet known. During a clinical trial, additional information is gained about a new treatment, its risks, and how well it works.
Advances in medicine and science result from new ideas and approaches developed through research. New cancer treatments must prove to be safe and effective in scientific studies with a certain number of patients before they can be made widely available. Through trials, researchers learn which approaches are more effective than others. A number of today’s standard treatments were first shown to be effective in clinical trials.
What are the Benefits of Taking Part in a Clinical Trial?
Benefits of participating in a clinical trial include:
Participants have access to promising new approaches that are often not available outside the clinical trial setting.
The approach being studied may be more effective than the standard approach.
Participants receive regular and careful medical attention from a research team that includes doctors and other health professionals, thus patients receive multiple expert opinions and the combined knowledge and care of several health care providers.
Participants may be the first to benefit from the new method under study.
Results from the study may help other cancer patients in the future.
What are the Possible Risks of Taking Part in a Clinical Trial?
Advances in medicine and science result from new ideas and approaches developed through research. New cancer treatments must prove to be safe and effective in scientific studies with a certain number of patients before they can be made widely available. Through trials, researchers learn which approaches are more effective than others. A number of today’s standard treatments were first shown to be effective in clinical trials.
Possible risks of participating in a clinical trial may include:
New drugs or procedures under study are not always better than the standard care to which they are being compared.
New treatments may have side effects or risks that doctors do not expect or that are worse than those resulting from standard care.
Participants in randomized trials will not be able to choose the approach they receive.
Health insurance and managed care providers may not cover all patient care costs in a study.
Each study’s protocol has guidelines for who can or cannot participate in the study. These guidelines, called eligibility criteria, describe characteristics that must be shared by all participants. The criteria differ from study to study. They may include age, gender, medical history, and current health status. Eligibility criteria for clinical trials also require that patients have a particular type and stage of cancer. Enrolling participants with similar characteristics helps to ensure that the results of the trial will be due to what is under study and not other factors. In this way, eligibility criteria help researchers achieve accurate and meaningful results. These criteria also minimize the risk of a person’s condition becoming worse by participating in the study.
This is a big question. Finding answers and making decisions are often hard for a cancer patient. The diagnosis of cancer and deciding what to do about it can be overwhelming. It is important to discuss your opinions with medical experts and your family. Your doctors can counsel you about your choices for standard treatment or clinical trials, as they often know what new medical technologies are being developed and how those new trial methods may benefit you.
Informed consent means that as a patient you are given information so you can understand what is involved in a trial, including its potential benefits and risks, and then decide freely to take part in the trial or not. You are given an informed consent form to read and consider carefully. Ask any questions you may have. Then, if you agree to take part, you can sign the form.
What to Expect as a Participant in a Clinical Trial
Patients who participate in a clinical trial work with a research team. Team members may include doctors, nurses, social workers, dietitians, and other health professionals. The health care team provides care, monitors participants’ health, and offers specific instructions about the study. For the trial results to be as reliable as possible, it is important for participants to follow the research team’s instructions. This may include keeping logs or answering questionnaires.
Patients in a clinical trial receive their care in the same places that standard treatments are given – at cancer centers, hospitals or doctors’ offices. If you join a research study, you will be watched closely and data on your case will be carefully recorded. You may receive more examinations and tests than are usually given. During the course of a study, if it is clear that a treatment is not in your best interest, you will be removed from the study and you can discuss other options with your physician.
The same ethical and legal codes that govern standard medical treatment also apply to clinical trials. Every clinical trial has a protocol, or action plan, for conducting the trial. The plan describes what will be done in the study, how it will be conducted, and why each part of the study is necessary. The same protocol is used by every doctor or research center taking part in the trial. All clinical trials that are federally funded or that evaluate a new drug or medical device subject to FDA regulation must be reviewed and approved by an Institutional Review Board (IRB). The IRB, which includes doctors, researchers, community leaders, and other members of the community, review the protocol to make sure the study is conducted fairly and participants are not likely to be harmed. The IRB also decides how often to review the trial once it has begun. Based on this information, the IRB decides whether the clinical trial should continue as initially planned and, if not, what changes should be made.
There are many kinds of cancer clinical trials. They range from studies of ways to prevent, detect and diagnose, or control and treat cancer, to studies of the psychological impact of the disease and ways to improve the patient’s comfort and quality of life (including pain control).
Prevention trials test new approaches such as medications, vitamins, or other supplements that doctors believe may lower the risk of developing a certain type of cancer.
Screening trials study ways to detect cancer earlier.
Diagnostic trials study tests or procedures that could be used to identify cancer more accurately.
Treatment trials are conducted with people who have cancer.
Supportive care trials explore ways to improve the comfort and quality of life of cancer patients and cancer survivors.
Genetics studies are sometimes part of another cancer clinical trial. The genetics components of the trial may focus on how genetic makeup can affect detection, diagnosis, or response to cancer treatment.
Government agencies such as the National Cancer Institute (NCI) and other parts of the National Institutes of Health (NIH), the Department of Defense and the Department of Veterans Affairs, sponsor and conduct clinical trials. In addition, organizations or individuals such as physicians, medical institutions, foundations, volunteer groups, and pharmaceutical companies also sponsor clinical trials.
Health insurance and managed care providers often do not cover the patient care costs associated with a clinical trial. What they cover varies by health plan and by study. Check with your provider for further information.
Clinical trials are usually conducted in a series of steps called phases. Treatment clinical trials listed in PDQ, the NCI’s comprehensive cancer information database, are always assigned a phase. However, screening, prevention, diagnostic, supportive care, and genetic studies do not always have a phase.
Phase I trials are the first step in testing a new approach in people. Researchers evaluate what dose is safe, how a new agent should be administered, and how often. Phase I trials usually enroll a small number of patients and take place at a few locations. The dose of the new therapy or technique is increased a little at a time. The highest dose with an acceptable level of side effects is determined to be appropriate for further testing.
Phase II trials study the safety and effectiveness of an agent or intervention and evaluate how it affects the human body. Phase II studies usually focus on a particular type of cancer, and include fewer than 100 patients.
Phase III trials compare a new agent or intervention with the current standard therapy. Participants are randomly assigned to the standard group or the new group by computer selection. This method, called randomization, helps to avoid bias and ensures that human choices or other factors do not affect the study’s results. Phase III trials often include large numbers of people across the country.
Phase IV trials are conducted to further evaluate the long-term safety and effectiveness of a treatment. They usually take place after the treatment has been approved for standard use. These trials may involve thousands of people across the country.
After a clinical trial is completed, the researchers look carefully at the data collected during the trial before making decisions about the meaning of the findings and further testing. After a Phase I or II trial, the researchers decide whether to move on to the next phase, or to stop testing the agent or intervention because it was not safe or effective. When a Phase III trial is completed, the researchers determine whether the results have medical importance. The results of clinical trials are often published in peer-reviewed scientific journals. Once a new approach has been proven safe and effective in a clinical trial, it may become a standard practice.
The following clinical trials are currently open to accrual at the University of Tennessee Cancer Institute.
BREAST
Neoadjuvant –
Neoadjuvant Weekly Nab-Paclitaxel (Abraxane®) Plus Carboplatin Followed By Doxorubicin Plus Cyclophosphamide with Bevacizumab Added Concurrently To Chemotherapy for Palpable and Operable Triple Negative Invasive Breast Cancer.
A Randomized Phase III Trial of Neoadjuvant Therapy in Patients with Palpable and Operable Breast Cancer Evaluating the Effect on Pathologic Complete Response (pCR) of Adding Capecitabine or Gemcitabine to Docetaxel when Administered Before AC with or without Bevacizumab and Correlative Science Studies Attempting to Identify Predictors of High Likelihood for pCR with Each of the Regimens.
Adjuvant –
Program for the Assessment of Clinical Cancer Tests (PACCT-1): Trial Assigning Individualized Options for Treatment: The TAILORx Trial (node negative ER/PR positive).
A Clinical Trial to Determine the Efficacy of Five Years of Letrozole Compared to Placebo in Patients Completing Five Years of Hormonal Therapy Consisting of an Aromatase Inhibitor (AI) or Tamoxifen Followed by an AI in Prolonging Disease-Free Survival in Postmenopausal Women with Hormone Receptor-Positive Breast Cancer.
A Multicenter Phase III Randomized Trial of Adjuvant Therapy for Patients with HER2-Positive Node-Positive or High Risk Node-Negative Breast Cancer Comparing Chemotherapy Plus Trastuzumab with Chemotherapy Plus Trastuzumab Plus Bevacizumab
Phase III Trial of Continuous Schedule AC + G Vs. Q 2 Week Schedule AC, Followed by Paclitaxel Given Either Every 2 Weeks or Weekly for 12 Weeks as Post-Operative Adjuvant Therapy in Node-Positive or High Risk Node-Negative Breast Cancer.
A Double-Blind, Phase III Trial of Doxorubicin and Cyclophosphamide followed by Paclitaxel with Bevacizumab or Placebo in Patients with Lymph Node Positive and High Risk Lymph Node Negative Breast Cancer
Randomized Phase III Trial of Bisphosphonates as Adjuvant Therapy for Primary Breast Cancer; treating women who have undergone surgery for stage I, stage II, or stage III breast cancer.
Phase III Trial of LHRH Analog Administration during Chemotherapy to Reduce Ovarian Failure Following Standard Adjuvant Chemotherapy in Early Stage, Hormone Receptor Negative Breast Cancer.
Metastatic –
Phase III, randomized, open-label study of Neratinib versus Lapatinib plus Capecitabine for the treatment of ErbB-2-positive locally advanced or metastatic breast cancer.
Endocrine Therapy With or Without Inhibition of EGF and HER2 Growth Factor Receptors: A Randomized, Double-Blind, Placebo-Controlled Phase III Trial of Fulvestrant with or Without Lapatinib (GW572016) For Postmenopausal Women with Estrogen-Receptor Positive Advanced Breast Cancer.
A Randomized Phase III Double-Blind, Placebo-Controlled Trial of First-Line Chemotherapy and Trastuzumab with or without Bevacizumab for Patients with Her-2/NEU Over-Expressing Metastatic Breast Cancer.
A multi-center, open-label, exploratory study of Bcr-Abl kinetics in adult patients on nilotinib with Philadelphia chromosome positive (Ph+) chronic myelogenous leukemia in chronic phase (CML-CP) and a suboptimal molecular response to imatinib
A Clinical Trial Comparing Preoperative Radiation Therapy and Capecitabine with or without Oxaliplatin with Preoperative Radiation Therapy and Continuous Intravenous Infusion of 5-Fluorouracil with or without Oxaliplatin in the Treatment of Patients with Operable Carcinoma of the Rectum
Adjuvant -
1st Line Metastatic -
A Phase III Trial of Irinotecan/5-FU/Leucovorin or Oxaliplatin/5-FU/Leucovorin with Bevacizumab, or Cetuximab (C225), or with the Combination of Bevacizumab and Cetuximab for Patients with Untreated Metastatic Adenocarcinoma of the Colon or Rectum
Multicenter Randomized Phase II Study of Erlotinib, Cisplatin and Radiotherapy versus Cisplatin and Radiotherapy in Patients with Stage III and IV Squamous Cell Carcinoma of the Head and Neck
Metastatic –
A Randomized, Open-Label, Controlled, Phase II Trial of Combination Chemotherapy with or without Panitumumab as First-line Treatment of Subjects with Metastatic or Recurrent Head and Neck Cancer, and Cross-over Second-line Panitumumab Monotherapy of Subjects who Fail the Combination Chemotherapy Only Arm.
Cetuximab at either 500 or 750 mg/m2 every other week for Platinum Refractory Cancer of the Head and Neck.
Phase III Chemoprevention Trial of Selenium Supplementation in Persons with Resected Stage I Non-Small Cell Lung Cancer.
A Phase III Randomized Trial of Adjuvant Chemotherapy with or Without Bevacizumab for Patients with Completely Resected Stage IB (> 4 cm) - IIIA Non-Small Cell Lung Cancer.
A Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Single-Agent Tarceva ® (erlotinib) Following Complete Tumor Resection with or without Adjuvant Chemotherapy in Patients with Stage IB-IIIA Non-Small Cell Lung Carcinoma who have EGFR-positive Tumors.
Locally Advanced -
A Pilot Trial of Cisplatin/Etoposide/Radiotherapy Followed by Consolidation Docetaxel and the Addition of Bevacizumab (NSC-704865) in Three Cohorts of Patients with Inoperable Locally Advanced Stage III Non-Small Cell Lung Cancer.
Metastatic –
A Randomized Phase III Study of Docetaxel or Pemetrexed with or without Cetuximab in Patients with Recurrent or Progressive Non-Small Cell Lung Cancer after Platinum-Based Therapy
An Open-Label, Randomized, Phase 2 Study of R-CHOP Plus Enzastaurin Versus R-CHOP in the First-Line Treatment of Patients with Intermediate and High-Risk Diffuse Large B-Cell Lymphoma.
Randomized Phase III Trial of Consolidation Therapy with Bortezomib (Velcade®) - Lenalidomide (Revlimid®) - Dexamethasone (VRD) versus Bortezomib (Velcade®) - Dexamethasone (VD) for Patients with Multiple Myeloma Who have Completed a Dexamethasone Based Induction Regimen
Previously untreated –
A Randomized Phase III Trial of CC-5013 (Lenalidomide, NSC-703813) and Low Dose Dexamethasone (LLD) Versus Bortezomib (PS-341, NSC-681239), Lenalidomide and Low Dose Dexamethasone (BLLD) for Induction in Patients with Previously Untreated Multiple Myeloma Without an Intent for Immediate Autologous Stem Cell Transplant
Phase III, Randomized, Open-Label, 3-Arm Study to Determine the Efficacy and Safety of Lenalidomide (Revlimid®) Plus Low-Dose Dexamethasone When Given Until Progressive Disease or for 18 Four-Week Cycles Versus the Combination of Melphalan, Prednisone and Thalidomide Given for 12 Six-Week Cycles in Patients with Previously Untreated Multiple Myeloma Who are Either 65 Years of age or Older or Not Candidates for Stem Cell Transplantation
A Randomized, Double-blind Phase III Trial of Adjuvant Sunitib Versus Sorafenib Versus Placebo with Resected Renal Cell Carcinoma
A Randomized Phase II Study of VEGF, RAF kinase, and mTOR Combination Targeted Therapy (CTT) with Bevacizumab, Sorafenib and Temsirolimus in Advanced Renal Cell Carcinoma
A Double-blind, randomized, placebo-controlled Phase III study to assess the efficacy of recMAGE-A3 + AS15 ASCI for patients with MAGE-A3 positive resected stage III melanoma.
GLOSSARY OF TERMS
Adjuvant Chemotherapy: One or more anticancer drugs used in combination with surgery or radiation therapy as part of the treatment of cancer.
Antibody: A protein produced by a plasma cell in the lymphatic system or bone marrow. An antibody binds to the specific antigen that has stimulated the immune system. Once bound, the antigen can be destroyed by other cells of the immune system.
Antigen: A substance, foreign to the body, that stimulates the production of antibodies by the immune system. Antigens include foreign proteins, bacteria, viruses, pollen and other materials.
Biological Therapy: Use of biologicals (substances produced by our own cells) or biological response modifiers (substances that affect the patient’s defense systems) in the treatment of cancer.
Blood Count: Measurement of the number of red cells, white cells, and platelets in the sample of blood.
Bone Marrow: The inner, spongy core of the bone that produces blood cells.
Cancer: A generalized term for more than 100 diseases characterized by abnormal and uncontrolled growth of cells. The resulting mass or tumor can invade and destroy surrounding normal tissue. Cancer cells from the tumor can spread throughout the blood or lymph to start new cancers in other parts of the body.
Chemotherapy: Treatment with anticancer drugs.
Clinical Trial: The systematic investigation of the effects of materials or methods according to a formal study plan in a human population with a particular disease. In cancer research, a clinical trial generally refers to the evaluation of treatment methods such as surgery, drugs, or radiation techniques, although methods of prevention, detection, or diagnosis also may be the subject of such studies.
Combination Chemotherapy: Use of two or more anticancer drugs.
Combination Therapy: The use of two or more modes of treatment – surgery, radiotherapy, chemotherapy, immunotherapy – in combination, alternatively, or together, to achieve optimum results against cancer.
Control Group: A group of patients that receives the standard treatment, a treatment or intervention currently being used and considered to be of proven effectiveness on the basis of past studies. Results in patients receiving newly developed treatments may then be compared to the control group. In cases where no standard treatment yet exists for a particular condition, the control group would receive no treatment. No patient is placed in a control group without treatment if there is any beneficial treatment known for that patient.
Double-blind: Characteristic of a controlled experiment in which neither the patient nor the attending physician knows whether the patient is getting one or another drug or dose. In single-blind studies, patients do not know which of several treatments they are receiving, thus preventing personal bias from influencing their reactions and study results. In either case, the treatment can be quickly identified by a special code.
Hormone: Chemical product of the endocrine glands of the body which when secreted into body fluids has a specific effect on other organs.
Immune System: A complex network of organs, cells, and specialized substances distributed throughout the body that defends it from foreign invaders that cause infection or disease.
Immunotherapy: A form of biological therapy. A method of treating cancer using substances which stimulate the body’s immune defense system.
Informed Consent: The process in which a patient learns about and understands the purpose and aspects of a clinical trial and then agrees to participate.
Interferon: A protein substance produced by white blood cells and other types of cells that have been exposed to certain viruses.
Investigational New Drug: A drug allowed by the FDA to be used in clinical trials but not approved by the FDA for commercial marketing.
Investigator: An investigator is the experienced clinical researcher who prepares a protocol or treatment plan and implements it with patients.
Metastasis: The transfer of disease from one part of the body to another.
Multimodality Therapy: The combined use of more than one method of treatment, for example surgery and chemotherapy.
Oncologist: A physician who is a cancer specialist.
PDQ: A computerized database supported by NCI that is available to physicians nationwide. It offers the latest information on standard treatments and ongoing clinical trails for each type and stage of cancer.
Placebo: An inactive substance resembling a medication given for psychological effect or as a control in evaluating a medicine believed to be active.
Protocol: The outline or plan for use of an experimental procedure or experimental treatment.
Radiation Therapy: Treatment using X-rays, radium, neutrons, or other types of cell-destroying radiation (also called radiotherapy).
Radiosensitizers: Drugs being studied to try to boost the effect of radiation therapy.
Randomized Clinical Trials: A study in which patients with similar traits, such as extent of disease, are chosen or selected by chance to be placed in separate groups that are comparing different treatments.
Remission: The decrease or disappearance of evidence of disease. Also, the period during which
this occurs.
Risk/Benefit Ratio: The relation between the risks and benefits of a given treatment or procedure.
Side Effect: A secondary and usually adverse effect from a drug or other treatment.
Single Blind: A controlled experiment in which the patient does not know what treatment they are receiving, but their attending physician is aware of the type of treatment they are receiving. This is used to prevent personal bias from influencing participant reactions and study results.
Staging: Methods used to establish the extent of a patient’s disease.
Standard Treatment: A treatment or other intervention currently being used and considered to be of proved effectiveness on the basis of past studies.
