What is a Clinical Trial?

A clinical trial is a well-planned research study that tests a drug or treatment to see how well it works on people. In the United States, clinical trials are overseen by the Food and Drug Administration. They are typically carried out in a cancer center, institute, clinic, or other medical facility under the control of a medical team. Clinical trials may involve a variety of different approaches in dealing with cancer. These approaches can include:

  • using new drugs
  • using new combinations of existing drugs
  • using new protocols for administering drugs (for example: different dosing and/or cycle lengths)
  • new combinations of chemotherapy and radiation therapy
  • employing new methods for dealing with cancer (for example: investigating one of the two main immunotherapy approaches, antibody therapy and vaccine therapy)

Clinical trials involve rigorous testing, reporting and adherence to specific guidelines. Not all patients are eligible to be involved in a clinical trial, even if it includes the specific cancer they have. There are constraints that might make you ineligible to participate in a specific clinical trial, e.g., your age, your previous chemotherapy regimens, and whether or not your cancer has metastasized.

Clinical Trials for Sarcomas

Sarcomas are an uncommon type of cancer. Over the past forty years, sarcomas have been treated with chemotherapy. Standard chemotherapy unfortunately has not been effective for all patients. New treatment options are needed. Sarcoma clinical trials are one way to identify new therapies. Sarcoma clinical trials provide an opportunity to have access to new drugs which have the potential to improve outcomes and hopefully cure sarcoma.

The words "clinical trial" may evoke uneasiness for some people. The thought of having a new drug that is still in the process of being studied may be concerning. Understanding the different phases of clinical trials and having strategies to navigate the clinical trial system may help alleviate concerns.

Video: Clinical Trials for Solid Tumors

The Four Phases of Clinical Trials

Phase 1 trials are the earliest stage of drug development. A phase 1 trial attempts to determine the dosing of a particular drug and the side effects through rigorous monitoring of the patients and frequent testing of blood at timed intervals. Phase 1 clinical trials are generally not specific to any one type of cancer. Often small numbers of volunteers are enrolled in these types of trials.

Video: Phase 1 and 2 Clinical Trials

Phase 2 trials include more volunteers who have a certain type of cancer that the researchers believe may be helped by the new treatment. In phase 2 trials, researchers seek to gather further safety data (information about side effects) and evidence of potential beneficial effects. If the drug proves to be effective and the side effects acceptable, a drug may then be studied in a Phase 3 clinical trial.

Phase 3 trials study a drug in a large number of volunteers with a specific cancer. This phase further tests the drug’s effectiveness and in some cases compares a new drug to a standard therapy to see if one is better than the other. Phase 2 and Phase 3 clinical trials can sometimes involve a "control" which means one person will get the new drug and another will get the standard therapy or placebo. The process is usually determined by randomization which is like a "flip of a coin".

Video: The Benefits of Phase III Clinical Trials

Phase 4 trials are conducted after a product is already approved and on the market to find out more about the treatment’s long-term benefits and risks.

Many drugs used in treatment today, were initially evaluated in phase 1, 2 or 3 clinical trials prior to approval by the FDA. Clinical trials have rigorous regulations and guidelines to ensure the safety of all volunteers.

What Are the Different Types of Clinical Trials?

There are several types of clinical trials that may be available to patients throughout the journey with sarcoma. There are trials that research better ways to prevent disease or recurrence; trials designed to improve detection, screening or diagnosis of disease; trials that study new treatment approaches or combinations of drugs, and trials that seek to improve comfort or quality of life.

When to Consider Clinical Trials

Clinical trials may be worth considering at different times when dealing with sarcoma. Dr. Karen Albritton once commented:

"I think it’s important to understand when to consider what phase of a clinical trial might be appropriate for you. Sometimes untreated patients enter Phase III clinical trials. Indeed, at initial diagnosis of Ewing’s sarcoma, you would only want to consider a Phase III clinical trial. 

At first relapse or progression of the disease, you would want to consider Phase II trials that are specific to Ewing's sarcoma. Third line, you would want to consider phase II trials perhaps for all types of sarcoma or solid tumors. Fourth line, you would want to consider Phase I trials. 

If you have tried these other routes and have moved more to a mode of realizing there is unlikely to be a drug that will cure the disease, but still want to be getting a drug, and contributing to advancement of Ewing's treatments, this may be time for becoming involved in a Phase I trial."

In the end, you will contrast participation in the clinical trial with your other treatment options.

  • Do the possible outcomes and risks seem acceptable to you and your cancer given the currently known results about the chemotherapy agents involved?
  • How will your participation affect your ability to participate in future clinical trials involving the same or other drugs?
  • Will there be some other, more appropriate or more interesting clinical trials that might be opening up soon?

None of these are easy questions to answer. You will want to seek the advice of your oncologist and your family. If you do decide to enter a clinical trial, be prepared to complete additional forms and paperwork and to establish a specific schedule of visits for treatments and tests, so be sure to allow enough time and to bring your calendar with you to do this. We urge you to document your experience in as much detail as possible so that it can ultimately be of benefit to others.

Considering Clinical Trials

Choosing a Clinical Trial

Video: Cooperative Group Clinical Trials

There are a number of sources where you can learn about candidate clinical trials: from a member of your medical team, from the professional and lay literature, and from searching appropriate websites.

When considering if a clinical trial is "worth" your time (in contrast to only "aiding science" by your participation in it), you probably want answers to a number of questions. You can get help in obtaining answers to your questions and in understanding the issues involved by establishing a relationship with the Research Assistant or Research Nurse associated with the clinical trial. Remember to do your research before applying for a clinical trial. Some sample questions for the Research Assistant or the Research Nurse are:

  • Are there currently or have there been patients with my cancer on the trial? Known results?
  • Are there currently or have there been similar trials elsewhere in the US or abroad? What was learned about dosing and types of cancer the drugs have been successful with in these trials?
  • Is this a multi-center trial? Are there any results known from using the drugs in other centers?

See Participating in a Trial: Questions to Ask Your Doctor from the National Cancer Institute.

Visiting the Clincial Trial Physician or Team

Suppose you are going to visit with the team conducting the clinical trial to see if you are a candidate for it. Here are some suggestions about what you should bring to your first appointment with the clinical trial doctors.

  • Your medical history. The more of your medical history the doctor has, the "better" the appointment might go. The medical history is used to determine if you can participate in the trial or not. Details of the chemotherapy drugs that you have had (which drugs, their frequency, dosing, etc), pathology reports, CT scans, MRI scans, and X-Rays can often be very useful to aid the medical staff in making this determination. Fill out as much of the paper work ahead of time as possible. Have your insurance company approve your participation and provide the cancer center with the appropriate approval information before your visit.
  • Your questions about the clinical trial itself. What can you expect? What will be done, how often, when, and for how long? Under what set of circumstances would you not be allowed to continue on the clinical trial after you have started it? Since clinical trials involve the collection of research data for the oncologists and research staff, there are frequent blood tests (called "PK" studies, where PK = pharmacokinetic) which are collected over the first 2-3 cycles of the treatment. These PK studies normally have to be done at the cancer center where the clinical trial is being conducted
  • Your questions about the drugs that will be used, their frequency, dosing, side effects, precautions, known results, etc.
  • Your questions about the tests you’ll need and where they can be done. You may also have to undergo baseline and post treatment tests (e.g., CT scans, MRIs, PET scans, and/or X-Rays) as appropriate. These tests may or may not be able to be done elsewhere (e.g., near your home). Where these tests are done may be a consideration if the site conducting the clinical trial is relatively far from your home. You may need to plan to stay overnight near the cancer center depending on the location of the cancer center, how long the treatment takes, how long you are to be observed after the treatment, and when and where any after-treatment tests are scheduled. There are programs that might be able to help family with travel expenses associated with getting to and from cancer treatment centers. Make sure when you call and make the appointment to get detailed directions to the cancer center facility, particularly if it is another city or state.

Beyond Clinical Trials

Video: When is a clinical trial not right for me?

Even if you can participate in a clinical trial, it might not be your best course of action. For example, participation at the beginning of a Phase I trial where the amount of chemotherapy received is very small, might have the effect of reducing your eligibility to be considered as a candidate in a future clinical trial. If a Phase I trial is the only current clinical trial option, it might be better to wait for another trial, or to try a chemotherapy agent off-study or in a "compassionate use" context.


After a successful clinical trial, the FDA approves a drug for use for one or more cancers. "Off-study" refers to using an FDA approved drug for a cancer other than those for which it was approved.

Compassionate Use

A patient with advanced disease or with no approved treatment or clinical trial options can attempt to get access to a new, unapproved drug outside of participating in a clinical trial. Access to a drug outside of a clinical trial prior to FDA approval is commonly referred to as "compassionate use. See the following resources:

Clinical Trials from a Participant's Perspective:
Peaks, Potholes & Paralyses

Living inside a clinical trial is a journey of peaks and potholes. Peaks are easy. The highest is waking up alive. Potholes litter the path too, and they are usually annoyances or inconveniences. When they occur, I try to remember what G. K. Chesterton said, "An inconvenience is an adventure wrongly considered."

The busy ER provided one such recent adventure. Sick babies, strokes, motor vehicle accidents, and traumas from a nearby tornado-hit to a motor-home park filled the nooks and crannies of a small town in southwestern Missouri in January. When CODE BLUE was announced overhead, my first impulse was to rush to Room 214 and save the day. Then I remembered I hadn’t saved anyone’s day for eighteen months when metastatic leiomyosarcoma caused me to leave my beloved internal medicine practice. I was in the ER, not as a physician, but as a cancer patient whose oncologist had referred for evaluation of a suspected deep vein thrombosis (DVT). My port used for chemo was inflamed and tender. After twelve hours of waiting for the true emergencies to be seen, I was told the Doppler was negative and released.

The deepest pothole in my journey came with the news that, without chemo, I had less than a year to live. My options included several unappealing traditional chemo regimen and ET-743, a promising drug available only via clinical trial in limited supply in only a few sites across the country. Through the intervention of my oncology team, I was referred to another center for consideration of an expanded-access clinical trial. Five months into my clinical trial, I am filled with hope. My CT scans have demonstrated no progression of metastatic disease. Stable disease is considered a response.

Gratitude continues to be a constant companion that I have been given this chance to participate in this drug trial. Every day I live breeds hope that I will survive to benefit from advances in research that may bring more definitive treatment. In between the peaks of gratitude and hope exists the reality of traveling six hundred miles round trip for a 24-hour infusion of chemo every twenty-one days. Although my physical side effects are limited to annoying potholes of nausea, gastroesophageal reflux, constipation, anorexia, and fatigue, my mind and soul suffer more limiting blows. A life-suffocating fatigue breeds a mental paralysis that dulls my ability to experience pleasure, animation, or hope.

Fortunately, this paralyzing inertia lasts for only a week or so of the cycle. This shadow-self is transient and precedes a rebirth of desire, joy, and hope. During these peaks I have enjoyed traveling to the Rocky Mountains, Greece, Grand Cayman, and spending more time with my family. There is nothing like the loss of vitality to make its return a cause for uninhibited celebration. Life is a gift. It is precious beyond description. I will be forever grateful for those who continue to do cancer research and champion the cause of those like me who depend on the kindness of strangers for our very life.

Searching for Trials

The search for a clinical trial usually involves several steps. To get started, simply search for trials that involve a specific type of sarcoma. Your search will yield targeted clinical trials for that particular disease. Most of these studies will be in Phase II or III.

Click on a term below to view open trials for a specific type of sarcoma at ClinicalTrials.gov. Results will include clinical trials in all locations. To limit your search to a specific country or state, click "refine search" and scroll down to choose locations.

Broadening your search

Video: Clinical Trials for Solid Tumors

Some clinical trial descriptions do not include specific types of sarcoma; they use more general terms instead. In order to explore all trial options, you will need to expand to an advanced search that includes broad terms that apply to your situation, like:

Additional Resources


ClinicalTrials.gov is a registry of federally and privately supported clinical trials conducted in the United States and around the world. ClinicalTrials.gov gives you information about a trial's purpose, who may participate, locations, and phone numbers for more details. This information should be used in conjunction with advice from health care professionals.

Coalition of Cancer Cooperative Groups - Trial Check

TrialCheck®, winner of 2008 Consumer Health World Award "Best in Show," is the country’s most up-to-date source of cancer clinical trial information. The Coalition’s copyrighted cancer clinical trials screening questionnaire will identify trials appropriate for you, your loved ones or your patients. TrialCheck® contains more than 4,000 cancer clinical trials from Cooperative Groups, National Cancer Institute (NCI), academic centers, and pharmaceutical and biotech companies. TrialCheck is updated on a daily basis with new information.

SARC's Clinical Trials

SARC is a non-profit organization dedicated to the development and support of clinical trial research for the prevention, treatment and cure of sarcomas - a cancer of the bone and connective tissue of the body.

Southwest Oncology Group

Southwest Oncology Group is one of the largest cancer clinical trials cooperative groups in the United States. Funded largely by research grants from the National Cancer Institute, part of the National Institutes of Health, the Group conducts clinical trials to prevent and treat cancer in adults, and to improve the quality of life for cancer survivors.

Sarcoma Trials Listed at Center Watch

Founded in 1994, CenterWatch is a trusted source and global destination for clinical trials information for both professionals and patients. CenterWatch, a Boston-based publishing and information services company focusing on the clinical research industry, is a business of Jobson Medical Information, LLC. We provide proprietary data and information analysis on clinical trials through a variety of newsletters, books, databases, and information services used by pharmaceutical and biotechnology companies, CROs, SMOs, and investigative sites involved in the management and conduct of clinical trials. As a pioneer in publishing clinical trials information, CenterWatch was the first Internet site to publish detailed information about active clinical trials that could be accessed by patients and their advocates. Today, we have one of the largest databases of clinical trials actively seeking patients on the Internet.

Clinical Trials Information from the National Cancer Institute

The National Cancer Institute maintains a list of clinical trials and clinical trials results, as well as educational materials about clinical trials.

American Cancer Society Clinical Trials Matching Service

The American Cancer Society Clinical Trials Matching Service is a free, confidential program that helps patients, their families, and health care workers find cancer clinical trials most appropriate to a patient's medical and personal situation. Through a partnership with the Coalition of Cancer Cooperative Groups, we can help you find research studies that are testing new drugs or methods to prevent, detect or treat cancer.

EORTC European Organization for Research and Treatment of Cancer

EORTC mission is to "conduct, develop, coordinate, and stimulate laboratory and clinical research in Europe to improve the management of cancer and related problems by increasing survival but also patients' quality of life." The ultimate goal of EORTC is to "improve the standard of cancer treatment in Europe, through the development of new drugs and other innovative approaches, and to test more effective therapeutic strategies, using drugs which are already commercially available, or surgery and radiotherapy." There is a history of nearly three decades of coordinated cancer treatment research between the EORTC and the National Cancer Institute (NCI), which is the leading agency for cancer research and treatment in the United States and is part of the National Institutes of Health (NIH).

Medical Research Council UK

MRC’s Cancer Division Clinical Trials Unit (CTU) is the largest cancer trials office in the United Kingdom (UK), and is active in all aspects of clinical trials. It’s objectives are to, "Initiate, design, conduct, analyse and publish clinical trials of national and international importance; Develop all aspects of clinical trial and meta-analysis methodology; Advance the design, conduct, analysis and reporting of clinical trials research; and Investigate cancer treatments of unproven effectiveness through the conduct of individual patient data meta-analyse." In particular CTU’s website states, "The CTU conducts several trials jointly, or in parallel, with other large trial organisations both within and outside the UK. In addition, many CTU trials are open to participation from individual clinicians world-wide."

Musella Foundation

The Musella Foundation for Brain Tumor Research Information provides this site on clinical trials and noteworthy treatments for brain treatments. You can browse for treatments by tumor type, treatment type, geographic area, or you can search by keyword or use the advanced search to narrow down the treatments, or search by doctor or hospital. The site also contains a brain tumor guide, and you can subscribe to the Foundation’s newsletter. All brain tumor patients are also welcomed to participate in the site’s "Brain Tumor Virtual Trial," a new concept in collecting and analyzing outcomes data for brain tumor patients. This program collects information from brain tumor patients, over the internet, and the Foundation records and analyzes the outcomes of the treatments with the idea to enable the Foundation to identify which treatments or combinations of treatments look the most promising.

Memorial Sloan Kettering Cancer Center

You can find descriptions of some of the newest clinical trials at Memorial Sloan-Kettering Cancer Center at this website. Clinical trials can be found by searching all trials or pediatric trials by disease category. Search results provide the full name of the trial, the trial’s purpose, eligibility, and the contact investigator.

National Center for Complementary & Alternative Medicine

The National Center for Complementary and Alternative Medicine (NCCAM) is one of the centers that make up the National Institutes of Health (NIH). The NCCAM is dedicated to exploring complementary and alternative healing practices in the context of rigorous science, training complementary and alternative medicine (CAM) researchers, and disseminating authoritative information to the public and professionals. On the NCCAM Health Information subweb, you can find general information about understanding complementary and alternative treatments, more specific information about the various complementary and alternative treatments available, as well as alerts and advisories, and information about dietary supplements. One can also search for complementary and alternative medicine clinical trials by treatment or therapy or by disease or condition.

City of Hope: Clinical Trials On-Line
Duke Comprehensive Cancer Center 
Fred Hutchinson Cancer Research Center 
Johns Hopkins (Sidney Kimmel Comprehensive Cancer Center) 
Ohio State (Arthur James Cancer Hospital) 
Stanford Comprehensive Cancer Center 
St. Jude Children's Hospital 
University of California at San Francisco (UCSF) Comprehensive Cancer Center

Abigail Alliance

The Abigail Alliance is dedicated to helping create wider access to developmental cancer drugs and other drugs for life threatening illnesses. The Alliance is promoting creative ways of increasing expanded access and compassionate use programs, and promoting creative ideas to get promising new drugs to the market sooner. The Abigail Alliance is also dedicated to help inform cancer patients and their doctors about the www.clinicaltrials.gov and www.cancer.gov clinical trial websites and to make sure all trials are listed and kept up to date.

Health Insurance and Clinical Trials

Clinical Trials and Insurance Coverage
State Laws Regarding Insurance Coverage

Housing Assistance

Amschwand Foundation Housing Program (Houston, TX) 
Believe in Tomorrow Housing 
Hope Lodge (A Home Away from Home) 
Joe's House - A Guide for Cancer Patients 
Miracle House 
National Association of Hospital Hospitality Houses 
Ronald McDonald House Charities

Travel Assistance

Air Care Alliance
Angel Flight America
Colgon Air, Inc. 
Corporate Angel Network 
Lifeline Pilots 
Mercy Medical Airlift 
Miles for Kids in Need (AA) 
National Patient Air Transport Helpline 
Patient AirLift Services (East Coast)