Understanding Clinical Trials in Myelodysplastic Syndromes (MDS)
*Please note: This slide show is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always consult your doctor about any questions you may have regarding a medical condition.
What is a clinical trial?
A clinical trial is a research study in which patients volunteer to receive a new drug or treatment before it is approved.
Therapeutic clinical trials help find new ways to treat diseases, including
myelodysplastic syndromes, or
MDS.
MDS clinical trials are important
New tests and treatments are needed for MDS.
While MDS can be treated, the only cure is a stem cell transplant, also called a bone marrow transplant. This can be a challenging medical option for multiple medical and social reasons.
MDS clinical trials are important
One in 3 people with MDS have a type that gives them a higher risk of developing a disease called acute myeloid leukemia (AML).1
How are clinical trials done?
Clinical trials are done in 4 phases.2 Each phase has a different potential goal or outcome.
In Phase 1, a small group of volunteers tests a treatment for safety and side effects (also called toxicity).
In Phase 2, a larger group tests the treatment's effectiveness (or how well it treats the disease).
How are clinical trials done?
In Phase 3, many volunteers are treated to confirm effectiveness and compare the treatment with currently available treatments that may be considered standard of care.
A treatment is approved by the regulatory authorities once it is proven safe for patients and effective as a disease treatment. Then doctors can prescribe it with wider access for the public.
In Phase 4 clinical trials, researchers collect information on the newly approved treatment for longer term outcomes.2
Are clinical trials supervised?
Before starting, clinical trials must be approved by a group called an Institutional Review Board.3
During the trial, study monitors check that the research is done correctly for safety and privacy.3
The trial can be stopped if prespecified or unanticipated problems are found.
What research is being done on MDS?
Clinical trials for MDS are investigating many different approaches to treating MDS:
Everything from targeted therapies, which work on a specific part of MDS biologic development, through sample banking and patient reported outcomes.
Who can join an MDS clinical trial?
You may join an MDS clinical trial to try a new treatment or to help others. Specific eligibility criteria are used to ensure each patient is appropriate for the chosen trial.
Why are clinical trial volunteers needed?
Clinical trial volunteers are needed because new treatments must be tested by humans before being prescribed. But only 6 in every 100 people with cancer joins a clinical trial.4
Many people may not know that trials are available or how to find one.
How do I decide to join a trial or not?
You can learn about trials before joining. Once you find a trial that interests you, contact your healthcare team to learn if you qualify. If so, you will go through a process called informed consent.
This involves meeting with a study team member to review the research plan, risks, and benefits. If you want to join, you sign a statement agreeing to participate and then work closely with the trial team and your doctor.3
Can I change my mind about being in a clinical trial?
You can leave a clinical trial at any time if you change your mind.5
You will still receive standard treatments and care if you choose to leave the trial.6
Will I get a placebo, or "sugar pill", in an MDS clinical trial?
A placebo, or "sugar pill," contains no active ingredients.7
Cancer clinical trials do not often use placebos. Instead of a placebo, the trial can compare the new treatment to the standard treatment.8
Will I benefit from being in a clinical trial?
You may benefit personally from being in a clinical trial, and you will help others with MDS in the future. The trial results will help doctors learn more about MDS.5
Are there risks to being in a clinical trial?
Like every treatment, clinical trial treatments do have risks. It is important to learn about the possible risks before you decide to join a trial. You may always ask questions.
The risks are often like the risks of standard cancer treatment.5
How do I find MDS clinical trials?
If you are interested in joining a trial, speak with your medical team. They can help you find a trial right for you.
You can also search for clinical trials by visiting
ClinicalTrials.gov or the MDS Foundation's clinical trials finder at
mds-foundation.org.
References
- MDS Foundation. What is MDS?
https://www.mds-foundation.org/what-is-mds
- American Cancer Society. Types and phrases of clinical trials. Last revised: August 18, 2020.
https://www.cancer.org/treatment/treatments-and-side-effects/clinical-trials/what-you-need-to-know/phases-of-clinical-trials.html
- U.S. National Institutes of Health. The Basics. NIH clinical research trials and you. Last revised: October 3, 2022.
https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics
- Unger JM, Fleury M. Nationally representative estimates of the participation of cancer patients in clinical research studies according to the commission on cancer. J Clin Oncol. 2021;39(28) Suppl 74.
https://ascopubs.org/doi/abs/10.1200/JCO.2020.39.28_suppl.74
- Memorial Sloan Kettering Cancer Center. Frequently asked questions about clinical trials.
https://www.mskcc.org/cancer-care/clinical-trials/frequently-asked-questions
- Cancer.Net. Patient safety in clinical trials. Last revised: September 2019.
https://www.cancer.net/research-and-advocacy/clinical-trials/patient-safety-clinical-trials
- National Cancer Institute. How Do Clinical Trials Work? Use of placebos in clinical trials. Last revised: August 31, 2023.
https://www.cancer.gov/research/participate/clinical-trials/how-trials-work#use-of-placebos-in-clinical-trials
This slide show explains what
clinical trials are, how they are conducted, and why they are important for patients with
myelodysplastic syndromes (
MDS).
Clinical trials are research studies in which people volunteer to help test
new treatments,
procedures, or ways to
diagnose diseases, including MDS. Clinical trials are important to better understand the biology of MDS and find more treatments. Click through to learn about the
phases of clinical trials,
safeguards,
eligibility criteria,
informed consent, the potential
benefits and
risks of participation, and how to find MDS clinical trials. Be sure to ask your doctor about joining a clinical trial. You can ask anytime before or during treatment.
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