Clinical Trials
Is a clinical trial right for you? Use the menu below to explore what clinical trials are, how to decide if participating is right for you, and how to find and join an appropriate trial.
Clinical Trial Basics
Medical research is carried out through studies called clinical trials, which run in steps, or phases, designed to find treatments that work while keeping patients safe. The information here focuses on treatment trials.
See all six phases, from pre-clinical to Phase IV
| Phase | Goal of the Research | Who Takes Part |
|---|---|---|
| Pre-Clinical |
|
|
| Phase I |
|
|
| Phase II |
|
|
| Phase I/II (Pilot) |
|
|
| Phase III |
|
|
| Phase IV |
|
|
Are clinical trials only a last resort?
No. A Phase III trial tests whether a new treatment that already showed promise works better than an older, approved one. While Phase I trials are sometimes a last option, they can also be first-line when no effective therapy is known. Remember: every effective cancer drug was once in a Phase I trial.
What is randomization, and can I avoid it?
When a new treatment is tested against an existing one, a computer randomly assigns patients to one group or the other — you can’t choose. There’s no guarantee the new treatment works better, or even as well. Some trials let you cross over if one group is clearly doing better.
What happens if I’m in the control group?
The control group receives the standard treatment and is the benchmark a new treatment is measured against. Some rare-cancer trials use “historic” controls — comparing to earlier studies so everyone can receive the experimental treatment — though this isn’t the preferred approach, since factors like newer diagnostics can make past and present groups hard to compare.
Could I receive a placebo instead of treatment?
Sometimes a placebo is one of the randomized groups — for example when “watch and wait” is the standard, or when a new drug is added to a standard treatment and a placebo is added to the other group so neither patients nor doctors know who’s getting it. A placebo may also test a drug meant to control side effects.
How would I know if I could get a placebo?
Ask your doctor exactly what each treatment group would include, and read the informed consent document before signing up. If a placebo is part of the trial, feel free to ask why it’s included.
Why are some trials “blinded”?
Blinding means the patient and/or doctor doesn’t know which group a patient is in. It’s used when a trial measures something subjective (like how well a drug eases nausea), and for some targeted treatments where “time to progression” is the outcome. Because that can be hard to judge objectively, blinding produces more reliable results.
What is a trial protocol?
The protocol is the full plan: why the treatment should be valuable, the scientific questions, the treatment regimens, which tests and data will be collected and when, and the follow-up needed afterward. Your doctor can usually provide a copy, though it’s written in very technical language.
Government
U.S. agencies such as the National Cancer Institute fund trials, run at one or several locations.
Cooperative Groups
Consortiums of hospitals specializing in cancer research, mostly government-funded. Pooling patients reaches answers faster. SARC and the Children’s Oncology Group (COG) are examples.
Single Institution
Universities and hospitals running their own trials, sometimes banding together for multi-site studies.
Individual
Specialist physicians with a research interest and enough patients to run a trial in a reasonable time.
Companies
Companies sponsor trials to show a new treatment works better than existing options, often partnering with cooperative groups or institutions.
What Makes Sarcoma Clinical Trials Different?
Many sarcomas don’t yet have standard treatments that work well, so trials matter enormously for our community. Yet many sarcoma trials have closed without results because too few patients enrolled — often due to misconceptions about trials.
Adult participation is strikingly low
Fewer than 10% of adults with cancer enroll in trials, versus up to 90% of children — a high pediatric rate that has helped drive major gains in childhood cancer survival. Low adult enrollment so concerned the NCI and ASCO that they held a 2010 symposium on improving it.
Rare cancers often use different trial designs
Because sarcomas are rare, the pool of eligible patients can be small. Trials adapt — Phase I/II (pilot) designs speed up evaluation, and some trials use historic controls so everyone can receive the experimental treatment.
The blood-brain barrier can affect eligibility
Many sarcoma drugs are thought not to cross from the bloodstream into the brain or spinal cord. As a result, sarcoma that has spread to the central nervous system may make a patient ineligible for certain trials.
Seeing a sarcoma expert opens more doors
For sarcomas, we recommend being treated by a sarcoma expert — the physicians most likely to know which trials are available and to have recommendations for you. Find a sarcoma center near you.
Why does molecular testing matter for sarcoma?
Sarcoma is not one disease — it includes more than 50 distinct subtypes, each with its own biology. Increasingly, tumors are analyzed for specific genetic changes, and some trials look for a particular mutation rather than a particular sarcoma subtype. If you’ve had genomic or molecular testing (sometimes called tumor profiling or next-generation sequencing), those results can help your care team identify trials that target your tumor’s specific features. It’s worth asking your sarcoma specialist whether testing is appropriate for you.
What is a “basket” trial?
A basket trial enrolls patients based on a shared genetic change — regardless of where the cancer started. For a rare cancer like sarcoma, this design can open doors that subtype-specific trials can’t, because it pools patients across many cancer types who share the same molecular target. If your tumor has a known targetable alteration, ask whether a basket or “tumor-agnostic” trial might be an option.
Not sure where your care fits? Sarcoma centers are most likely to know which trials — including precision-medicine trials — are right for you.
Find a CenterMaking an Informed Decision
Deciding whether to join a trial is personal. These are the questions patients and caregivers ask most often.
What’s the benefit to me?
You’re treated with the latest therapies, your physician follows your progress more closely because trials require more data, and you have the knowledge that you helped develop better treatments for others.
Should I ask my physician about trials I might qualify for?
Yes — always ask about your options, trial or not, and whether they’re offered at your doctor’s institution. A sarcoma expert may have several recommendations. We offer a list of 20 questions to ask when weighing treatment options.
Are clinical trials as safe as standard treatments?
There’s always some potential risk, but it’s minimized by the phased process, which tests safety before effectiveness. Trials usually monitor effects and side effects more often, and rigorous regulations govern how studies on people are approved.
Will my insurance cover the cost?
The cost to you is usually comparable to standard treatment, though it depends on what the sponsor and your insurance cover. Talk to your physician about costs, and contact your insurer about coverage under the Affordable Care Act (learn more at cancer.net).
Does my doctor get paid if I take part?
Trials cost money to run, so the institution your physician works for is usually compensated for the added tests and data collection. Ask your doctor, or visit cms.gov/openpayments.
Will I have to travel to participate?
Some trials run at only a few locations; Phase III trials often run at many. Travel may add costs, but some organizations help cover it — see the list of organizations we’ve compiled. For some trials, most treatment and follow-up can be done near home.
Compare the trade-offs across seven factors
| Consideration | Pros | Cons |
|---|---|---|
| Cost | May be fully covered by the sponsor. | If not, insurance may not cover everything (rare). |
| Location | Often run by specialists, where you get the best care. | You may have to travel for part of treatment. |
| Medical Tests | You’re followed more closely than on standard treatment. | More visits and more types of tests. |
| Randomization | Built on “equipoise” — no assumption either treatment is better. | You might not get the experimental treatment. |
| Involvement | You’ve made an active choice and tend to be more aware of options. | Randomization can feel like less control. |
| Effectiveness | You get at least the standard treatment, possibly better. | The trial may not benefit you personally. |
| Helping Others | The best way to improve treatment for future patients. | No real downside — today’s treatments exist because of past participants. |
20 Questions to Ask. Our checklist helps you get the answers you need from your care team.
See the QuestionsIdentifying Trials You’re Eligible For
We have reviewed the existing search tools available for finding clinical trials. We believe the American Cancer Society’s “ACTS” program is optimal because it harnesses the incredible power of AI to identify the trials you may be eligible for and also includes real people with medical expertise to guide you.
American Cancer Society ACTS (Access to Clinical Trials & Support)
Click the link below to learn more about the ACTS program and search for trials that may benefit you:
Eight common exclusion criteria, explained
Beyond location and cost, eligibility rules can limit access. Listings always include these criteria, but it isn’t always obvious why they exist:
Prior therapy with the same agents
Excludes patients whose tumors haven’t responded; some drugs also have a maximum safe dose.
Serious side effects from prior treatment
E.g., cardiomyopathy — a new agent could make it worse.
Recent prior cancer treatment
It may be impossible to tell whether the previous or experimental treatment caused a response.
No measurable tumors
Most trials measure tumor shrinkage or growth; unmeasurable tumors make that impossible.
Central nervous system involvement
Many sarcoma drugs don’t cross the blood-brain barrier, so CNS metastases may exclude a patient.
Pregnancy or breastfeeding
Experimental treatments are never assumed safe for developing babies.
Active infections
Most agents suppress the immune system, so an active infection could be dangerous.
Other medical conditions
If your physician feels other conditions put your health at risk, you shouldn’t be enrolled.
Enrolling and Participating
If you decide to move forward, here’s what to expect — from signing up to how and when results are shared.
How do I sign up for a trial?
Every trial has an “informed consent” document to read and discuss with your doctor first. It explains why the treatment is being tested, what it involves, the risks, which tests are research-specific, possible costs, and other key details — in plain language, not jargon. Ask questions about anything you don’t understand.
Can I opt out after I’ve signed up?
Absolutely. You always have the right to leave a clinical trial at any time, for any reason.
How often are results checked?
While a trial runs, the data is checked for safety often by a monitoring committee not otherwise involved in the study. If safety problems arise — or if one group is doing much better — the trial can be stopped or changed. Otherwise, outcome data usually isn’t analyzed until the trial closes to enrollment.
How are results used?
The protocol lays out the scientific questions, and the results are analyzed statistically to determine whether the study answered them positively (the new treatment is better) or not.
Will I find out the results?
Generally yes, though it can take several years depending on the trial. There’s a strong push to return results to participants, and the NCI requires federally funded results to be published within a set time frame. Check your informed consent, and ask your doctor if you have questions.