Once you have completed your consent and the trial team has confirmed you are eligible to participate, you are ready to join the trial. The actual process will be different for each person, depending on where and how the trial is being conducted and the type of treatment you will receive. But these are general guidelines on what you can expect.
What to expect…
Your first visit to a trial location is called your “baseline” visit. This is a pre-entry visit where information is collected and you are actually entered into the clinical trial. During this first visit, you are likely to meet with members of the trial team. You might undergo a number of tests to understand as much as possible about your health and your bleeding disorder before you begin to receive the treatment.
You’ll also be asked a lot of questions so your team has a detailed view of your medical history. They might also ask about medicines you’ve taken in the past or other prescriptions you are currently taking. All of this information will help the team understand how the treatment is working for you as they watch for changes throughout the trial process.
Once this information is collected, you will be assigned to a treatment group and be given a detailed review of the plan for your treatment. This will include how long the treatment will take, how often you’ll need to visit and what you’ll need to do in between visits.
At this baseline visit, you can also expect to receive lots of resources for support. Use this time to ask any questions and be sure to know your primary point of contact for questions that might come up when you are not at the trial site.
Once your baseline visit is complete, you will return for regular treatment visits. You should expect that each visit will include lots of questions to understand how you are feeling and how your disorder is responding to the treatment.
You can also expect a lot of testing. This gives your trial team information (data) about the activity of the treatment in your body, such as where is it working and for how long. All of this data helps determine if the treatment is effective or not.
Visits to the trial site is only one small part of taking part in a trial. Once you receive your treatment, you will likely be asked to carefully track how you are feeling until your next visit.
It's important to report any new symptoms you are feeling - even as mild as a stuffy nose. These are referred to as "adverse events." The research team may or may not ask you to come in to the trial site to check whether the symptom is related to the treatment you are receiving.
In addition to the data collected in the visit tests, the information you track between visits is just as important to understanding how the treatment works. This information is called patient-reported outcomes data, or “PROs.” This often includes information that is described as “quality of life” data – that might include how you are feeling each day, physically, mentally and emotionally, and how well you are able to go about your regular daily activities while receiving this treatment. This information is very important, so be sure to make time each day to track your experience with as much detail as possible.
A note about “controls” and randomization
A clinical trial needs to understand if a new treatment is better, the same, or less useful than what is considered “standard” today for a certain bleeding disorder. So when they test it in a trial, they need something to compare it to; this is called a “control.” Sometimes this process is also referred to as randomization. You are put into a group of the study based upon chance or by certain characteristics (such as age, gender, inhibitor, bleed history, etc.) to balance and compare the groups. In most cases, the control is a currently available treatment. Your trial team will explain to you that you may be assigned to the active treatment group or the control group in a trial.
In most cases, you will not know which group, nor will your trial physician. This is important because knowing which group you are in could affect how the researchers study your experience in the trial. This control helps to make sure that the differences they see between trial groups are really based on the treatment effect.
Tests you might expect in a bleeding disorder clinical trial
- Journals / e-diaries: You may be asked to keep a daily log of how you are feeling to report back to your team. This can include information, like whether you felt any different after a course of treatment, how well you slept, what you ate, how active you were, and more.
- Surveys: You might receive paper or electronic surveys requesting more information about how you are feeling with specific questions that can give the team more information about how well the treatment is working.
- Digital trackers: As part of your trial, you might receive a small digital tool called a “wearable” device. These might include bracelets called Fitbit or similar types of devices that track your physical activity.
Your role in the trial
It’s not possible to do a clinical trial without you – the participant. While your trial team is actively monitoring and testing how the treatment works for you, they are also relying on you to provide information, ask questions, share your feelings and any concerns you have. YOU are part of the team! What are the expectations of you?
- Follow the treatment schedule consistently
- Make sure to make time to track or report on how you are feeling
- Give very honest answers to every question
- Always raise questions or concerns at every step of the process
Fitting the trial into your routine
Participating in a trial might mean more than just receiving a treatment and tracking how you feel. Many people who consider trials are curious about how much they need to change their regular routines to meet the trial requirements. These considerations will vary for each person, but they might include changes to your exercise routine, changes to your diet, such as sugar or alcohol intake, changes to consumption of alcohol or other drugs, or smoking. Talk with your trial advocate or social worker to understand what changes can help you have the most positive experience possible, and don’t be afraid to ask for help in making these changes work for you.
Mental health is an important part of joining a trial. For many people, being part of testing a new treatment can come with a lot of anxiety, with hope that it works for you or concerns about the potential for side effects. For others, frequent testing and regular monitoring can feel stressful. There are many resources available, so talk with your trial team about what support they can provide you.
Community Resources to Consider
- NHF “HANDI” line: https://www.hemophilia.org/community-resources/request-information/handi-nhfs-information-resource-center
- NHF Mental Health: https://www.hemophilia.org/educational-programs/education/mental-health
- NHF’s Local Chapters: https://www.hemophilia.org/community-resources/resources-near-you/nhf-chapters
What if you notice a side effect?
Every medicine has a risk of some side effects – in trials, these are called “adverse events.” Some people have no ill effects at all, while others might have mild or moderate discomfort. On rare occasions, a treatment can carry serious risks. But it’s important to remember that treatments being tested in clinical trials have already been tested in lots of different settings to understand what effects they may cause. The trial consent information explains what risks might come with the treatment being tested in your trial.
If you do notice something you think is unusual or you don’t expect, even if it's very mild, tell your trial contact right away so they can determine whether it's related to the treatment you're receiving. In most cases, they have plans prepared for how to help you with those events. In some cases, you may decide that the side effects are too uncomfortable. Remember that you have the choice to disenroll from the trial at any time.
Key questions to ask during your trial
Highlights to Remember:
- During the trial, you can expect testing, monitoring and questions so the trial team can understand as much as possible about how the treatment is working for you. You play an important role in tracking how you are feeling and reporting that back to the team.
- If you have any concerns, side effects or other events during the trial, please contact your team right away.
- Your research team will be able to help with the logistics of being at a trial site. Ask about travel vouchers or refunds if you need to travel long distances to your location.
- Don’t forget to take care of your well being during the trial. Contact your research team for support and resources that can support you.