New treatments for Waldenstrom macroglobulinemia (WM) cannot be offered to people with WM until research is done to prove their safety and effectiveness in human volunteers. These research studies, called clinical trials, must be conducted according to strict guidelines set by the U.S. Food and Drug Administration (FDA). If a new test or treatment is found to be safe and more effective than those currently being used, it may become the new standard of care in the management of people with WM.
What are clinical trials?
Clinical trials are research studies that test how well new medical approaches work in people. Before an experimental treatment can be tested on human volunteers in a clinical trial, it must have shown benefit in laboratory testing or animal research studies. The most promising treatments are then moved into clinical trials, with the goal of identifying new ways to safely and effectively prevent, screen for, diagnose, or treat a disease like WM. Clinical trials can study many things, such as new:
- combinations of treatments
- tests to find and track disease
- drugs not yet approved by the FDA
- procedures to relieve symptoms
What are the phases of a clinical trial?
Clinical trials are conducted in a series of steps, called phases. Each phase of a clinical trial has a different purpose. A clinical trial is only done when there’s a good reason to believe that the treatment or test being studied may be better than the one being used now (the standard treatment).
- Phase I: Researchers test a drug in a small group of people (15-30) to evaluate its safety, dosage range, and side effects. They carefully watch for harmful side effects as the dose of the drug is slowly increased. Researchers determine what dose confers the most benefit with the fewest side effects.
- Phase II: The drug is given to a larger group of people to further evaluate its safety and study its effectiveness. While the emphasis in Phase I is on safety, the emphasis in Phase II is on effectiveness. Participants are closely monitored to see if the new treatment works. At this point, the new drug is rarely compared to the current drug that is used. If the new drug is found to work, it can be tested in a Phase lll trial.
- Phase III: The new drug is compared to a current standard-of-care drug. These trials assess the effectiveness and side effects of each drug in large groups of people (from 100 to several thousand people) to see which one works better.
Participants are randomly assigned to either the control group, which receives the current standard-of-care treatment, or to the new treatment group. Randomization means that participants are assigned to a group by chance; this is usually accomplished by using a computer program.
Randomization is done to make sure that the people in each group are alike. Researchers can then be confident that the results of the trial are due to the treatment and not differences between the groups. Neither you nor your doctor can request that you be assigned to a particular group. You will not know which group you’re in until the study is completed.
If the new therapy works well in a Phase III trial, researchers can submit an application to the FDA asking permission to make the treatment available to the general public.
- Phase IV: This phase occurs after a treatment has been approved and is being marketed. The drug’s maker may study it further to evaluate the side effects, risks, and benefits over a longer period of time and in a larger number of people than in a Phase III clinical trial.
Should I consider participating in a clinical trial?
People take part in clinical trials for many reasons. When you volunteer to participate in a clinical trial you help researchers learn more about curing, preventing, and treating WM and its complications. Your contribution to medical research has the potential to help improve the quality of life for people with WM. In addition to helping others, you get extra care and monitoring from the clinical trials staff and may receive the newest treatments for your disease before they are widely available.
To join a clinical trial you must meet specific requirements, called eligibility criteria, in order to participate in the study. Patients in a trial are usually alike in key ways such as age, current health status, history of prior treatments, and stage of their disease. This is to make sure that any benefits documented during the study are due to the treatment being tested and not to medical differences between patients.
Whether one should participate in a clinical trial is a personal decision. Consult with your doctor to help you weigh the potential benefits and risks of participating, and then decide what is right for you.
What questions should I ask my doctor about clinical trials?
If you’re considering participating in a treatment clinical trial, “Questions to ask your Doctor about your Treatment(link is external),” originally published by the National Cancer Institute, is a good resource. It’s a comprehensive list of questions that you can bring with you to your doctor’s office as a guide for discussion. For your ease of reference, the questions are listed below.
Questions about the Trial
- What is the purpose of the trial?
- Why do the researchers believe that the treatment being studied may be better than the one being used now? Why may it not be better?
- How long will I be in the trial?
- What kinds of tests and treatments are involved?
- How will the doctor know if the treatment is working?
- How will I be told about the trial’s results?
- How long do I have to make up my mind about joining this trial?
- Who can I speak with about questions I have during and after the trial?
- Who will be in charge of my care?
- Is there someone I can talk to who has been in the trial?
Questions about Risks and Benefits
- What are the possible side effectsor risks of the new treatment?
- What are the possible benefits?
- How do the possible risks and benefits of this trial compare to those of the standard treatment?
Questions about Your Rights
- How will my health information be kept private?
- What happens if I decide to leave the trial?
Questions about Costs
- Will I have to pay for any of the treatments or tests?
- What costs will my health insurance cover?
- Who pays if I’m injured in the trial?
- Who can help answer any questions from my insurance company?
Questions about Daily Life
- How could the trial affect my daily life?
- How often will I have to come to the hospital or clinic?
- Will I have to stay in the hospital during the clinical trial? If so, how often and for how long?
- Will I have to travel long distances?
- Will I have check-ups after the trial?
Questions about Comparing Choices
- What are my other treatment choices, including standard treatments?
- How does the treatment I would receive in this trial compare with the other treatment choices?
- What will happen to my cancer without treatment?
When speaking with your doctor, it’s a good idea to bring a family member or friend who can take notes. You might also consider recording the meeting so you can review the conversation later as you consider your options. If you decide to move forward, you’ll be referred to the clinical trials research team to start the informed consent process.
What is informed consent?
During the informed consent process, a member of the research team explains the details of the study and answers your questions and concerns. You’ll then be given a written consent form that outlines these details, which typically includes information about:
- the purpose of the study
- who is eligible to take part
- the treatment(s) being studied
- possible risks and benefits
- tests used to monitor your progress during the study
- who pays for costs associated with the study (tests, doctor visits, etc.)
The informed consent process continues for the duration of the clinical trial. If new benefits, risks, or side effects are discovered during the study, researchers must notify you. Signing the written consent form does not mean you are obligated to remain in the study for its duration. Taking part in a clinical trial is always voluntary. You have the right to leave at any time, for any reason. The clinical trial team may ask to follow you for a certain period of time after you leave the study to make sure you’re safe and there are no unexpected problems.
Before signing the consent form, ask a lot of questions to make sure you fully understand what’s involved in participating. You should not feel rushed or pressured. You can take the consent form home and discuss the matter further with family members and friends before making a final decision.
Who pays for the cost of clinical trials?
The trial sponsor, which can be a government entity, a research institution, and/or a business like a pharmaceutical company, usually pays for:
- the cost of the treatment being studied
- tests done specifically for research purposes
- extra doctor visits that may be required
Routine patient care costs – considered the usual costs of your medical care, such as doctor visits, hospital stays and laboratory tests that you’d have whether or not you were participating in a clinical trial – may not be covered by your health insurance while you’re in the study.
In the U.S., certain states now have laws that require insurers to pay for the costs of routine care that occurs doing the course of a clinical trial. If you have Medicare, you may be reimbursed for some of the costs related to participating in trials of new ways to diagnose and treat cancer. Contact your health insurer or the clinical trial research coordinator for assistance in determining what costs will be covered and what your financial responsibility will be.
What if there are no appropriate clinical trials where I live?
If there is no trial appropriate for you in your local area, you might consider whether you’re willing and able to travel out-of-town to participate in a study. Unfortunately, travel expenses are not typically covered by the trial sponsor; however, the following organizations may help:
- Angel Flight: This name is used by a number of groups (Angel Flight Northeast, Angel Flight Central, etc.) whose members provide free air transportation for people needing medical treatment far from home.
- Corporate Angel Network(link is external): A non-profit organization that arranges free air travel for cancer patients to treatment centers by using empty seats on private and corporate planes.
- American Cancer Society: Provides free, home-like housing for cancer patients and their caregivers, called Hope Lodges(link is external), in over 30 locations in the U.S. and Puerto Rico. If there is no Hope Lodge in the area, call the American Cancer Society at 1-800-227-2345 for more information about lodging assistance. Free or reduced-cost housing may be available through the local cancer facility.
How do I find clinical trials for Waldenstrom macroglobulinemia?
Finding an appropriate clinical trial can be complicated and overwhelming. To help simplify the process for patients and their families, the Leukemia & Lymphoma Society (LLS) offers personalized assistance through their Clinical Trial Support Center(link is external). The Center is staffed by registered nurses with expertise in blood cancers who work with you one-on-one to navigate the clinical trial process.
Your LLS Clinical Trial Nurse Navigator will:
- Speak with you to discuss your goals and help you decide if a trial might be right for you.
- Help you understand the clinical trial process, including your rights and obligations as a participant.
- Ask for details about your diagnosis, including your genetic profile, past treatments and responses, as well as gather information about your past medical history and current physical condition that might impact your eligibility for certain clinical trials.
- Understand how your financial situation, insurance coverage, support network, and ability and willingness to travel far distances might impact your choice of clinical trials.
- Provide you with a list of appropriate clinical trials to discuss with your physician.
- Guide you in your efforts to enroll in a clinical trial, including connecting you with trial sites.
- Be available for support throughout your clinical trial experience.
For more information, contact the LLS Clinical Trial Support Center by:
- Phone: 1-800-955-4572 (Monday-Friday, 9:00 a.m. to 9:00 p.m. ET)
- Email: CTSC@LLS.org
If you want to research clinical trials on your own, you can visit the U.S. National Library of Medicine Clinical Trials Database(link is external), a comprehensive list of publicly and privately funded clinical studies being conducted in the U.S. and around the world.
To conduct a general search for studies in WM, do the following
- In the “Status” box, select either those trials that are “recruiting or net yet recruiting” participants, or “all studies,” which can include studies that are active but not recruiting, closed, or completed.
- In the “Condition/Disease” search box, type Waldenstrom and then choose Waldenstrom macroglobulinemia from the dropdown menu.
To narrow your search, you can:
- Choose United States from the dropdown menu in the “Country” search box; this allows you to further narrow your search by state, city, and distance from that city.
- Use the “Other Terms” search box to view trials of particular drugs or view an individual trial by its assigned NCT number (an identification code given to each study, also called the ClinicalTrials.gov identifier; format is NCT followed by an 8-digit number).
To learn more about using this website, visit this page(link is external) written for patients and their families.