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An in depth look at Clinical Trials with Be The Match

Medical advancements take time and research. There are several types of studies and trials that are conducted during the medical research process, and in general, clinical research includes two types: observational studies and clinical trials. Both help researchers by involving people and are commonly used for identifying new treatments and therapies for rare diseases, including histiocytosis. It can be overwhelming when deciding if a clinical trial or study is appropriate for you or a loved one, so I sought out the incredible team at Be The Match – Jason Carter Clinical Trials Search & Support Program (JCCTSS) for today’s conversation. I am hoping this can help provide some guidance for making an informed decision, and can shine some light on the amazing support that JCCTSS can offer!  

You can also explore www.histio.org/research/clinical-trials/. Here you will find clinical trials by diagnosis, educational videos, registries, genetic test information, and other helpful resources.

So first, let us briefly define the two types of clinical research: observational studies and clinical trials. Observational studies observe individuals in a normal setting, looking for changes over time to specific characteristics. Natural history studies are a type of observational study that help researchers to understand more about the disease, from timelines to demographics, genetic factors to possible environmental factors. Observational studies may involve tests, questionnaires, or medical exams and can help identify possibilities for clinical trials.  

Clinical trials are research studies aimed at evaluating interventions [behavioral, medical, or surgical]. They are a way to gather information about how well a new treatment or test works, to compare new treatments with current or older treatments, to understand side effects and how to manage them, and to learn more about quality of life during/after treatment. There are four phases that clinical trials go through, which hopefully leads to approval by the FDA. 

Phase I	Tests an experimental treatment on a small group (20-80 people); often these are conducted with healthy volunteers to assess safety, however in rare diseases, they can be conducted with those who have the disease.
Phase II	Enroll a larger group (100-300) and main emphasis is on effectiveness, while continuing to monitor safety. Some trials have participants take a placebo. Can last several years. In rare disease studies, it is common to combine Phase I and II or Phase II and III to speed up development timelines.
Phase III	Investigators will look to learn about reactions to the therapy and focus on safety and effectiveness, studying different populations and varied dosages. This phase enrolls anywhere from 300+ to 3000 individuals. If Food and Drug Administration deems results as positive, the experimental treatment will be approved for use in the affected population.
Phase IV	Long term monitoring of safety and effectiveness in large, diverse populations. Side effects may become more apparent over time.

I spent some time with Brenna O’Brien, Clinical Trials Navigator with Be The Match’s Jason Carter Clinical Trials Search & Support program (JCCTSS). The program was created to help people with blood cancers or blood disorders and their families find and join clinical trials. They take the time to review active clinical trials from clinicaltrials.gov and modify the information into easy-to-understand content that is accessible through an easy-to-use online search tool. There are educational resources available and a patient support center to help along the way. 

Brenna and her team partner with patients and their families/friends to offer support in understanding clinical trials, accessing information on open opportunities, and gaining access to financial grants to help with clinical trial travel costs. The support is one-on-one, allowing you to get customized answers to all questions. I inquired about the most frequent questions Clinical Trials Navigators are asked so that we can share some of those answers with you.  

• What does Be The Match do?
  • Be The Match® is a global leader in bone marrow transplantation. We conduct research to improve transplant outcomes provide support and resources for patients, and partner with a global network. They host a registry to help connect patients with the donors they need for life saving treatment. Be The Match also has other programs and services including support groups and workshops, educational books, DVDs, and fact sheets, caregiver support, telephone counseling, and help finding clinical trials for blood cancers and blood disorders. 
• How can someone join / enroll in a clinical trial?
  • Before joining any clinical trial, you will sit down with the doctor who is managing the study; this is called an informed consent meeting, where you will learn the risks and benefits of the trial before you agree to participate. Be sure to ask questions so you can understand the process, learn which treatments are standard versus what is new or unproven. The study coordinator will also go over what other treatment options are available, your rights as a patient, and what the purpose of the study is. Only after you have signed an agreement to participate, can you enroll in a study. 
• Can you share some other facts about clinical trials?
  • Most trials (especially for cancer) do not include a placebo. Many times, patients receive heightened care and attention. Everyone will get treatment either with a standard of care or with the studied treatment. 
  • Trials are not only used as a “last resort”. There is a range of risk associated with clinical trials and the patient can choose the level of risk they are most comfortable with. Risk is inherent in all cancer treatment. 
  • You can leave a trial at any time without any repercussions to your relationships or care at your center. 
  • Before joining a trial, you will learn what costs are billed to your insurance and which ones are covered by the trial. 
• What do I need to know about payment and insurance coverage?
  • Most insurance companies are required legally to pay for the costs related to your patient care, including regular doctor visits, hospital stays, X-rays, and lab tests. The specific costs related to the clinical trial may not be covered, however you can discuss with your insurance in advance to understand what the burden might be and how to access financial grants to cover costs. You should also talk to the institution and doctor managing the trial to see what programs they offer for financial assistance. 
• If the trial is not close to my home, how will I manage travel costs?
  • JCCTSS’s Clinical Trials Navigators can help you learn if you qualify for financial assistance from the Drs. Jeffrey and Isabel Chell Clinical Trials Travel Grant. You can also look into housing support from the Ronald McDonald house or other similar programs. Contact the Histiocytosis Association if you need help locating resources in other areas.
• I do not live in the United States; can I still participate in a trial?
  • Some clinical trials do accept international patients. Be The Match has a broader Patient Support Center that can assist with international inquiries about transplants and clinical trials in the states. There are options available for patients, whether international or not, some of which will offer partial or full coverage of costs associated with the trial. At times, there are options available through the National Institutes of Health (NIH) for both United States and international patients. The Be The Match patient support center can assist in understanding what trials are available; they can be reached toll-free at 1-888-999-6743 or patientinfo@nmdp.org. 
• What other resources does your team provide?
  • JCCTSS is a part of the larger Patient Support Center at Be The Match; this includes services such as the Peer Connect Program, where you can speak with patients or parents who have gone through the transplantation or cellular therapy. The Patient Support Center also offers financial assistance for transplantation as well as sessions of one-on-one counseling with Licensed Clinical Social Workers. If anyone in the histio community has gone through transplantation or cell therapy services and is interested in joining the Patient Connect Program, please have them reach out to our team! patientinfo@nmdp.org