This guide, which will be published as a series of articles over the next couple of weeks, is intended to help clarify the clinical trial process and to answer common questions so that you can decide whether a clinical trial is the right option for you. The guide will explain the different types of clinical trials, address common concerns, provide answers from physicians to common questions, describe patients’ clinical trial experiences, and provide resources for finding a clinical trial.

There are many issues that patients worry about when considering whether to join a clinical trial, and there are many myths surrounding those issues. This article will try to separate the myths from the facts about clinical trial participation.

Participating in a multiple myeloma clinical trial

MYTH: There are already enough myeloma patients participating in clinical trials.
FACT: Approximately 5 percent of cancer patients are currently participating in clinical trials. However, with 66,529 people currently living with myeloma in the United States and with an estimated 20,580 new cases of myeloma expected to be diagnosed this year, there is a strong need for more people to participate in research to find more treatment options.

Purpose of myeloma clinical trials

MYTH: Myeloma clinical trials only benefit doctors and corporations who are testing out new medications.
FACT: Myeloma clinical trials try to achieve many goals beneficial to the patient. These goals include:

• Discover new therapies/treatments to eradicate or slow the growth of myeloma cells
• Provide long periods of remission
• Help patients feel better if they have bone pain, fatigue, or other difficult symptoms
• Adjust and test dosages of current treatments for myeloma
• Test combinations of current treatments for myeloma
• Test combinations of a new treatment in addition to current treatments for myeloma
• Improve safety and efficacy of stem cell transplantations
• Research how myeloma progresses in different ages, races, and genders of myeloma patients
• Develop novel therapies designed to delay/prevent multiple myeloma progression in smoldering myeloma patients
• Study mechanisms for multiple myeloma progression to aid in the design of future therapies to prevent progression

Eligibility / criteria for joining clinical trials

MYTH: Only terminally ill cancer patients or patients with no other treatment options left should take part in a clinical trial.
FACT: Clinical trials are open to all types of patients, including those who have exhausted all other treatment options as well as those who are newly diagnosed with myeloma.

A myeloma patient may fulfill any of these criteria to be able to participate in a clinical trial:

• Newly diagnosed
• Did not achieve a good response to established treatments
• Relapsed after treatment
• Continuing treatment after remission (maintenance)

Eligibility is also based on:

• Type and stage of myeloma
• Age
• Overall health
• Medications you have taken in the past and how long ago (wash-out period)

Common questions on criteria/eligibility:

Is there a screening period during which doctors decide the patient’s eligibility? If so, how long does this period last?
Yes. Patients’ eligibility is determined by their physician who is monitoring their health and response to standard treatment. If a patient is relapsing, a physician may suggest trying other standard therapies or a clinical trial. In the case of trying a trial, the screening period or washout period, during which the patient is monitored and is supposed to be off any other therapy for myeloma (to be able to determine efficacy of the drug being tested in the trial and so that there is only one variable in the trial), varies from two to four weeks to up to six weeks, depending on each individual case.

Do patients need to be concerned about their disease getting worse during the washout period?
There is a possibility that the disease could worsen without any treatment for two to four weeks. If you and your physician feel that this much time without any treatment is detrimental to your health, then another option should be considered. Waiting would only be beneficial if your case is not too severe or aggressive and allows leeway of that much time.

Are patients allowed to remain on maintenance medication throughout the washout period and trial?
This depends on the protocol. Generally, maintenance drugs and vitamin supplements, when approved by your physician, are okay. However, supplements with “natural” ingredients or unknown ingredients are not allowed due to doctors not knowing what they contain (which could cause a reaction with the tested drugs).

Testing for eligibility for a myeloma clinical trial

MYTH: Most patients who participate in clinical trials are “preselected” and are a certain “type,” so there is no such thing as truly being eligible for a trial.
FACT: Testing to determine eligibility for a myeloma clinical trial is very thorough and precise – much like the tests done when a patient is diagnosed. Each patient is unique, so to best determine what trial will be most effective, different tests are conducted. Some of the tests are done before the trial, and some are done to watch and track progress during the trial.

According to Dr. William Bensinger, a myeloma specialist at the University of Washington and Fred Hutchinson Cancer Research Center in Seattle, “If a patient meets the general eligibility criteria, they must undergo a series of baseline tests that will confirm eligibility. These usually include a bone marrow aspiration, skeletal survey, serum and urine protein electrophoresis, complete blood count, and chemistry labs including kidney and liver functions tests.”

Specific age of candidates required for enrollment in a clinical trial

MYTH: Older cancer patients are ineligible for clinical trials.
FACT: Enrollment of older patients in clinical trials is low, but not because they are ineligible but due to their low participation. Older patients are encouraged to join, especially since 63 percent of all cancer patients are 65 or older.

More information on best types of candidates for myeloma trials:

Specific minorities or age groups that are candidates:

According to Dr. Ola Landgren, a clinical trial investigator at the National Cancer Institute, “Myeloma is two-fold more common in African-Americans (versus Caucasians). We have ongoing studies looking into the underlying mechanisms of these patterns. Our data suggest that African-Americans have more precursor disease, monoclonal gammopathy of undetermined significance (MGUS), while the rate of progression from MGUS to myeloma is the same for the two races.”

Also, while myeloma is still quite rare in the general population, the elderly (above 60) have higher rates of myeloma.

“There are data to suggest that family members of myeloma patients are at a two-fold higher risk of myeloma, which supports a role for genetic factors,” said Dr. Landgren.

Specific types of patients that are best candidates for clinical trials:

• Smoldering myeloma patients with certain adverse clinical features (a higher risk of developing myeloma)
• Active myeloma patients who are not responding to established treatment
• Relapsed/refractory multiple myeloma patients who are willing to try new drugs
• Newly diagnosed patients are also good candidates for studies that develop new treatment strategies.

Explanation of risks

MYTH: Researchers and doctors may not spell out all of the risks associated with a clinical trial.
FACT: All aspects of a clinical trial, including the risks, potential short and long-term side effects, financial aspects, confidentiality, and protocol, are part of the informed consent process and are carefully explained to the patient in the beginning.

Treatment of people during clinical trials

MYTH: Patients in clinical studies are human guinea pigs.
FACT: Patients participating in clinical trials may be tested more frequently than patients treated at standard cancer centers. Frequent tests are necessary to monitor the patients’ safety. Patients participating in clinical trials are treated with the same care and respect as everybody else.

Quality of care received during a clinical trial

MYTH: I will receive poorer medical treatment in a clinical trial.
FACT: Patients in clinical trials receive either the best standard treatment or a new treatment that researchers believe is at least as good as the standard treatment. Patients in clinical trials are very closely monitored.

MYTH: I will get better medical treatment in a clinical study.
FACT: Medical treatment in a clinical study is as good as standard treatment. In addition, patients are receiving constant care and are being closely monitored. Also, they receive treatments from doctors who are leaders in their fields.

Chances of getting a placebo

MYTH: Cancer patients may be given a placebo in a clinical trial and thus not receive any real treatment.
FACT: Some multiple myeloma clinical trials may use a placebo as part of the treatment regimen, but myeloma patients always receive at least the standard of care. For instance, some trials may compare the addition of a drug to standard of care. In those trials, some patients will receive the new combination while others will receive the standard of care plus placebo. Also, since maintenance therapy is still being investigated, trials testing maintenance therapy may have an arm that receives maintenance therapy and another arm that receives a placebo maintenance therapy. In trials investigating how to prevent cancer, some participants may receive a placebo only.

Success of myeloma clinical trials

MYTH: Because drugs being tested in myeloma trials are relatively new, their success rates may be lower, and patients may not respond positively.
FACT: Patient response varies from trial to trial based on the treatment being tested and also on the patients. Trials for newly diagnosed myeloma patients typically have a higher rate of success than those for relapsed/refractory myeloma. At the same time, advances in the treatment of multiple myeloma are being made, and response rates are increasing. If a patient does not respond positively to a trial, they have the option to opt out.

Safety of clinical trials

MYTH: Clinical trials are not safe.
FACT: With clinical trials, there are many unknowns, including short-term and long-term side effects of the drug and how well you will respond to the treatment.

However, new treatments are tested on humans only after they have been lab tested and there is valid scientific evidence that the treatment is likely to be effective and safe. Patient safety is a top priority in clinical trials. Patients in clinical trials are therefore monitored very closely. However, the treatment may not be effective in some patients, or patients may develop serious side effects.

“If there is any doubt the drug does not work for that individual patient and the side effects are bad – then the drug is immediately stopped,” said Dr. Landgren. “The patient always comes first - in every trial!”

More information on safety:

Other options for patients afraid of unknown side effects:
If you are worried about the safety or efficacy of a new treatment, you may want to consider a later phase trial. With each phase of testing, more is known about the safety and efficacy of the treatment. Before signing up to receive a new treatment, you can read results of previous trials that tested the drug.

Additionally, you may want to consider a clinical trial that tests a new way of using drugs that have already been approved – the trial may test new dosages of a drug or a new combination of treatments. Also, there are myeloma clinical trials that are studying new procedures for stem cell transplants.

Side effects that can be experienced during myeloma clinical trials:
(These side effects are also seen in common myeloma treatment practices and are not specific to clinical trials. Side effects vary depending on the treatments being used. It is also possible for patients to experience side effects not yet associated with myeloma treatments. Side effects often vary from patient to patient.)

• Upset stomach, nausea, and/or vomiting
• Mouth sores
• Constipation
• Extreme tiredness/exhaustion
• Infections
• Low red blood cell count (anemia)
• Low white blood cell count
• Low platelet count
• Achy feeling
• Numbness in arms, hands, legs, or feet
• Neuropathy in hands and/or feet

Finances

MYTH: My health insurance will not pay for treatment provided through a clinical trial, and it is not an affordable option.
FACT: Many people are afraid to consider a clinical trial, fearing that their health insurance may not cover treatment. However, most trials are not financially burdensome, and sometimes physicians recommend participation in clinical trials to reduce costs for the patient. In many cases, private organizations or pharmaceutical companies conduct trials at no expense to the patient (this includes covering costs for tests, treatment, etc).

In the event that the cost of treatment during the trial is not covered by the sponsoring agency, your physician/research coordinator will need to speak with your insurance company to work out the details. Generally, insurance companies do not cover experimental clinical trials, unless it can be proven that it is a necessity or that the approach is especially safe and effective. For specific coverage, a doctor would have to prove the necessity of the drug.

If your health insurance does offer a possibility of covering it, there are certain criteria that need to be met, such as a specific cost threshold (it cannot cost more than a similar drug used to treat your disease).

Beneficiaries of Medicare, TRICARE, or the Department of Veterans Affairs are encouraged to participate in clinical trials designed to diagnose or treat cancer and are often reimbursed.

For more information, visit the National Cancer Institute’s Clinical Trials and Insurance Coverage website

Ability to drop out of a clinical trial

MYTH: If I join a clinical trial, I will not be able to drop out.
FACT: You may quit a clinical study at any time.

A common concern or reason why a patient might want to drop out of a clinical trial is if they experience serious side effects or get ill from an infection during the trial. Based on the protocol, in the event of a serious side effect or infection, the drug is either stopped or the dosage is reduced. The patient is always monitored very closely, so an infection would be taken care of promptly with antibiotics, anti-viral, or anti-fungal medication.

Length of myeloma trials and how long patients are monitored for

MYTH: Myeloma trials last only as long as the sponsor can afford to pay for the drug being tested, which cannot be very long.
FACT: The length of each trial varies depending on the protocol. It depends on how many cycles of treatment the protocol includes and also on the response of the patient. Treatment may continue as long as the patient responds positively to treatment, or it will end sooner if there is no response or symptoms get worse. Some trials may last for several years.

Life after the clinical trial

MYTH: I will not be able to continue treatment after the clinical trial is over.
FACT: After treatment during the clinical trial has been completed, a patient will often be studied for a while afterward to monitor progress and to see if any other side effects or changes occur. There are many treatment options for participants during this time, whether they responded to the experimental treatment or not.

If a patient responds positively
There are several options that allow the patient to continue receiving the experimental treatment:

• If the drug is FDA approved and commercially available for another disease or therapeutic use, the patient may purchase the drug. Payment methods/insurance coverage needs to be worked out between the physician/research coordinator, the insurance company, and the drug company.
• If the drug is not commercially available, many trials offer an “extension arm” that enables the patient to continue taking the drug under an extension of the trial, sponsored by the corporation or organization that sponsored the trial. Availability of an extension arm varies with each study, and the length of the extension arm also varies. Be sure to check with your physician.
• In the case that the drug is not commercially available and there is no extension arm of the trial, physicians and research coordinators can arrange for the drug to be used under “compassionate use,” which enables the patient to continue receiving the experimental therapy after the end of the clinical trial.

If a patient does not respond
The patient has the options to go back to a treatment that had previously worked, try another established treatment plan, or opt to try another clinical trial.

To learn more about clinical trials, please read Part 1 and Part 2 and check The Myeloma Beacon in the coming weeks for the other articles in the series.