Frequently Asked Questions
Below are some frequently asked questions about the myOpportunITy clinical research studies.
It is a good idea to talk to your doctor if you are considering taking part in a clinical research study.
What is myOpportunITy?
myOpportunITy is comprised of three clinical studies that are evaluating a potential new subcutaneous (under the skin) infusion called rozanolixizumab for adults living with immune thrombocytopenia (ITP).
The first two of these studies, called myOpportunITy 1 and myOpportunITy 2, will take place over up to 34 weeks to assess rozanolixizumab against a placebo (an infusion which contains no medicinally active ingredients). These two studies are identical and will run at the same time at different study sites (hospitals where the study is conducted), so each individual can only participate in one of them.
In each of these two studies, participants will be randomly assigned to one of two treatment groups. One group (2/3 of participants) will receive rozanolixizumab, and the other group will receive a placebo. This step is important because it allows us to see whether rozanolixizumab actually works in people living with ITP.
The myOpportunITy 1 and myOpportunITy 2 studies will consist of a screening period, a treatment period and a safety follow-up period. During the treatment period, you would receive infusions of either rozanolixizumab or placebo every two weeks.
During myOpportunITy 1 or myOpportunITy 2, you will have a total of approximately 30 visits, some of which can be home visits.
Home visits are where a nurse visits you in your home and performs the tests and assessments there without you having to travel to the study site. If you prefer, you can go to the site for these visits instead. Your study doctor will also help to decide if home visits are appropriate for you.
If you complete the myOpportunITy 1 or myOpportunITy 2 study and meet the entry criteria, you will have the option to enroll in the extended 60-week myOpportunITy 3 study. This will evaluate rozanolixizumab for longer-term management of ITP without the use of placebo. This means that all participants in myOpportunITy 3 (also those previously treated with the placebo) will receive rozanolixizumab.
Why are the myOpportunITy 1 and myOpportunITy 2 studies identical?
In order to be given the approval to make a new treatment available to the public, health authorities have to evaluate the data gathered in all the different clinical studies. These authorities have certain requirements that need to be met in order to grant what's called market approval.
One such requirement is that information from clinical studies is verifiable and reproducible. Conducting two identical studies that are independent of each other will ensure that this requirement is met. The two studies will not be conducted in the same exact set of countries, to ensure a variety of different data sources and to reduce bias.
What is a clinical research study?
Clinical research studies can help advance the understanding of a disease and are the most important way for researchers to find out if potential new treatments are well tolerated and effective. Studies like these are needed to be able to make new treatments available to patients.
Why might I want to participate in a clinical study?
People decide to take part in clinical studies for a variety of reasons. Some want to contribute to medical research and to potentially help others with the same condition. Some may wish to try a different approach to treatment. Clinical studies are completely voluntary, and participants can leave at any time.
Who is UCB?
UCB, a global pharmaceutical company based in Belgium, is developing rozanolixizumab and is sponsoring the myOpportunITy studies.
UCB is a global biopharmaceutical company focused on creating value for people living with severe diseases in immunology and neurology. The company was founded in 1928 and by the 1960s, UCB had begun visionary research into biotechnology, which led to a number of lifechanging medicines through the 80s and 90s. Over the last decade, UCB has become a leading global biopharmaceutical company. UCB is now building on a strong heritage in research and development to discover new treatments that focus on the needs of patients – both today and in the future.
UCB have listened and will keep listening to the evolving needs of participants throughout the myOpportunITy studies. UCB is engaging with organizations that support people with ITP to better understand their needs.
Who can take part in myOpportunITy?
To be eligible for the 34-week myOpportunITy 1 or myOpprtunITy 2 study, you must at the time of entering the study:
- Be 18 years of age or older
- Be diagnosed with persistent or chronic primary immune thrombocytopenia (ITP)(i.e. have a diagnosis of primary ITP for at least three months)
- Have a platelet count below 30 (<30x109/L)
- Have received at least one previous treatment for ITP
Exclusion criteria are things that you must NOT have at the start of the study. The main exclusion criteria for the myOpportunITy studies are as follows:
- Strong bleeding that needs immediate treatment to balance your platelet levels
- Known hypersensitivity (e.g. being allergic) to any components of the study drug
- Previously or currently diagnosed with a condition called hyperprolinemia
- Relevant active infection, or a serious infection within the last six weeks prior to the first infusion of the study drug
- Known tuberculosis (TB) infection or high risk of getting TB
Women that can bear a child must not be breastfeeding, pregnant or planning to get pregnant and must use effective contraception and for three months after the last infusion. Male participants must use a highly effective method of contraception during the study and for three months after their last infusion. You can talk to your study team about what methods of contraception are considered highly effective.
There are additional criteria that you will need to meet to participate in the study — the study team will assess and discuss these with you during a screening process. A study team typically consists of nurses and doctors, and can also include study coordinators or other staff involved in the study, but this can vary at each study site. During the study screening, members of the study team will be asking you questions and doing some tests to make sure the study is right for you. This discussion will also address whether you can remain on your current treatment.
You can also ask your own doctor to explain the entry criteria (also called eligibility criteria) to see if you may be able to participate in the study.
Entry criteria are there to make sure the group of people included have similar disease characteristics, and to exclude people for whom the study drug might have more risks than benefits.
Where are the myOpportunITy studies taking place?
The myOpportunITy studies are taking place at various study site locations around the world. You can use the site finder to see the closest study site location to you.
Can there be side effects from the study treatment?
Every medication can have side effects. For rozanolixizumab, earlier studies showed that rozanolixizumab is well tolerated and the side effects are generally mild to moderate in severity and can be well managed. Before you enter the study, you will receive more detailed information on which side effects of rozanolixizumab treatment are already known from previous studies, and you are encouraged to talk about potential side effects with your study doctor.
How long will the myOpportunITy studies take?
The myOpportunITy 1 and myOpportunITy 2 studies take place over up to 34 weeks. For participants who have had their spleen removed, the study could be up to 44 weeks in total to assess during the screening period if vaccinations are up to date and potentially refresh some vaccinations. If you complete one of these studies (i.e. you don't leave the study early), there is an extended 60-week study, myOpportunITy 3, in which everyone who meets the entry criteria will be treated with rozanolixizumab to understand its safety and effectiveness over a longer period of time.
myOpportunITy 1 and myOpportunITy 2 studies
STEP 1: SCREENING
- Confirms you can join the study
- There will be blood/urine tests, physical exams, and health questionnaires (you will be given more detailed information about these tests and assessments when you join the study)
- If your spleen has been removed, you will be tested to see if your vaccinations are up to date and potentially receive a refresher of some vaccines
STEP 2: STUDY TREATMENT PERIOD
- If the screening shows you are suitable for the study, you will be randomly assigned to receive rozanolixizumab or a placebo (an infusion which contains no medicinally active ingredients)
- Comparing rozanolixizumab with a placebo is important because it will allow us to see whether it actually works
- Twice as many people will receive rozanolixizumab than placebo, so your chances of receiving this potential new treatment are two in three
- You will be given subcutaneous (under the skin) infusions every two weeks throughout the treatment period, each of which will take about 30 minutes
- You will need to stay at the study site for four hours after the first three infusions and two hours after subsequent infusions to monitor your health and take blood tests
- On the weeks that you don't have an infusion, you will still have an appointment with the study team, and some of these can be arranged to be at your home, if possible. This is done to closely monitor you
- We will monitor your health and wellbeing with standard tests, procedures, and reviews of your ITP symptoms (you will be given more detailed information about these tests and assessments when you join the study)
STEP 3: SAFETY FOLLOW-UP PERIOD
- Two weeks after your last infusion, you will begin a six-week period where you will be observed for any ongoing effects of the infusions
- There are only two visits during this time, but you can contact your study team at any time during the whole study
- You will not be given any subcutaneous rozanolixizumab or placebo infusions during these visits
- The safety follow-up period only happens if you do not participate in the myOpportunITy 3 extension study; if you do participate in myOpportunITy 3, you go straight from step 2 to step 4 below
myOpportunITy 3 study
STEP 4: OPEN-LABEL EXTENSION STUDY
- All participants who complete the treatment period of myOpportunITy 1 or myOpportunITy 2 (i.e. do not leave the study early) and meet the entry criteria will be invited to take part in an extended 60-week study
- If you agree to take part, you will have regular study visits where your health will be monitored, and you will receive the study drug for optional at-home administration. In this extended study, all participants will receive rozanolixizumab
- The same tests as before will continue to be done to monitor your health and wellbeing
Can I keep my current doctor?
We encourage you to continue seeing your usual doctor, and update them on how you are progressing in the study (provided you consented to this).
Will I receive compensation for my participation?
Due to ethical and legal reasons, we cannot offer you monetary payment for your participation in the study. However, costs for travel to and from the study site can be reimbursed. Please ask your study team to provide you with more information around travel reimbursement.
The study drug will be provided free of charge by UCB during your participation in this study. You will not be charged for any procedure performed for the purpose of this study. Please note that UCB will not be paying for your usual ongoing medical care. This includes procedures and/or non-study medications that are required during this study as part of your usual medical care.
What are my options if I am not eligible for this study?
Clinical studies use inclusion and exclusion criteria to make sure that the potential benefits of the study drug outweigh any risk to participants and that the group of people in the study have similar disease characteristics. If you are not currently eligible for this study, please continue to talk to your doctor about other treatment options or clinical studies that you might be able to join.
What is ITP?
Immune thrombocytopenia (ITP) is a rare autoimmune disorder. The immune system is our body’s defense system against infections. Antibodies are the defenders which give us this protection. In the case of an autoimmune disorder like primary ITP, your antibodies cannot tell the difference between foreign proteins that should be eliminated (like bacteria or viruses or other substances) and the proteins on the body’s own cells. As a result, they end up targeting and destroying certain healthy or normal cells such as platelets. In primary ITP, the antibodies target and destroy the platelets and the cells producing them, leading to an overall reduction in the number of platelets in the body. When the number of platelets in your blood falls to a certain level, you develop a tendency to bleed or bruise easily.
What is rozanolixizumab?
Rozanolixizumab is an antibody made to work as a medicine that targets the process which allows defective antibodies enter into your blood and destroy your platelets. As a result, the defective antibodies are reduced in your blood which may allow the number of your platelets (also called platelet count) to increase.
How would my safety be protected during the study?
We want every participant to feel comfortable and safe. An essential part of any clinical study is keeping track of any side-effects that participants experience.
Tracking side-effects helps researchers analyze whether the treatment is well tolerated. If specific and/or severe side-effects occur while you are participating in the myOpportunITy studies, your dedicated study team will take action.
In addition, the myOpportunITy studies will be regularly reviewed by an independent data and safety monitoring board in order to identify and address any concerns that may arise.
Are there any potential side effects of rozanolixizumab that I should be aware of?
All medicines have side-effects. An important aim of the myOpportunITy studies is to make sure that the health benefits of rozanolixizumab outweigh any risk or inconvenience from side-effects.
Earlier studies showed that rozanolixizumab is well tolerated and the side effects are generally mild to moderate in severity and can be well managed. The myOpportunITy studies are being done to confirm these findings.
Before you enter the study, you will receive more detailed information on which side effects of rozanolixizumab treatment are already known from previous studies, and you are encouraged to talk about potential side effects with your doctor.
Throughout the studies, your health and wellbeing will be closely monitored. If you have any specific concerns or think you might be experiencing a side-effect, you can talk to the study team at any time.
What happens if I am assigned to placebo?
In the myOpportunITy 1 and myOpportunITy 2 studies, neither you nor the study team will know whether you have been given rozanolixizumab or placebo. You will be closely monitored throughout the study to ensure your health does not deteriorate. Rescue therapy is allowed at any time if you need it, will be free of charge, and you may continue with certain medications that you are already taking. As a general rule, you can continue on the study if you receive rescue therapy. All participants in the myOpportunITy 3 extension study will receive rozanolixizumab.
What happens if my platelet count is low during the study?
During the first part of the study treatment period in myOpportunITy 1 or myOpportunITy 2 the dose of the study medication can be adapted depending on the observed levels of platelets in your blood. The aim is to use a dose of the study medication that keeps your platelet count between 50 (≥50x109/L) and 200 (≤200x109/L). The dose can be adjusted to reach a stable platelet count throughout the whole treatment period.
Platelet counts are measured at every visit in the myOpportunITy 1 and myOpportunITy 2 studies.
If study treatment does not lead to an increase in platelet count to above 30 (>30x109/L) or if you have active bleeding, you can be given rescue therapy at any time if you and your doctor agree this is needed. Rescue therapy may be intravenous immunoglobulin (IVIg), high dose corticosteroids and pulse steroids, platelet transfusions, or other medications. As a general rule, you can continue on the study if you receive rescue therapy.
How will I know if the study drug is working?
It is possible that your ITP symptoms may change throughout the study. During the appointments you will have throughout the study, a member of the study team will take blood tests and ask you to fill in questionnaires about how you are feeling and what’s happening with your ITP. This close monitoring is to ensure that any changes to your condition are thoroughly documented. In addition to these tests and questionnaires, you are encouraged to speak openly with the study team about any changes you notice in your health.
What to expect after the study
What happens when the myOpportunITy 1 and myOpportunITy 2 studies are over?
Participants who complete the myOpportunITy 1 or myOpportunITy 2 study (i.e. do not leave the study early) will be invited to take part in a 60-week extension study, myOpportunITy 3, provided they meet the entry criteria. The myOpportunITy 3 study starts directly after myOpportunITy 1 or myOpportunITy 2 without any gap. During this extension study, all participants will receive rozanolixizumab for optional at-home administration. These types of extension studies allow researchers to track the effectiveness and safety of treatments over a longer period of time than the initial study.
Following these studies, how long will it take for rozanolixizumab to become available to the wider public?
It can take some time, usually years, before a new treatment is made available after a clinical study. That’s because there are rigorous processes to ensure the study really does show a benefit, and for health authorities to check and assess the effectiveness and safety of the potential treatment. You can certainly ask your study team and your own doctor about how this process is going.
How can I find out about the results of the study?
In order to analyze the results of the study, UCB has to wait until all patients in all countries have finished the study. For a lot of studies, this can take up to several years, depending on the duration of the study.
UCB will make the results of the study available in the applicable public study registry databases such as clinicaltrials.gov or the EU Clinical Trials Register. UCB also makes study result summaries in lay language (lay summaries) available on their website, and you can ask your study doctor about the availability of the study results after you have left the study. Results for the myOpportunITy studies are not expected to be available before the second half of 2022, but could also become available later than that.
We’re looking for people to join the studies—and help us try to improve the ups and downs of ITP.
Would you or someone you care about consider taking part?
myOpportunITy will be conducted at study sites around the world.