What Treatment Guidelines Exist For Women With EoE During Pregnancy? CEGIR answers the patient's questions about having EoE and being pregnant. This period raises many questions, from how medications and diet will affect the baby to what will happen to the EoE symptoms during pregnancy. Expand Limited Studies Suggest: The risk to a mother and her baby is not increased in pregnant women on or off EoE-specific medications. EoE symptoms may improve (56%), worsen (20%), or stay the same (24%) during pregnancy. Changes in EoE symptoms revert to pre-pregnancy levels in most patients. Treatment Options During Pregnancy EoE medications considered safe in pregnancy include swallowed topical corticosteroids (budesonide) and proton pump inhibitors. Diet elimination during pregnancy is best co-managed with a dietitian with experience in EoE to ensure adequate nutrition. Management of EoE During Pregnancy Management of EoE during pregnancy should be individualised using a shared decision-making approach between the patient and her physicians. Patients with active disease at the beginning of pregnancy may continue treatment. Patients with inactive disease may continue treatment or consider stopping treatment with close monitoring. Future studies are needed to answer questions about EoE management in pregnancy. Information is provided by The Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) Learn more about CEGIRs work Donate today for a brighter tomorrow Last updated 21/06/23 ©EOS Network 2023 All rights reserved
What research is being done to prevent EGIDs? CEGIR outlines four stages of focus for Eosinophilic Gastro-Intestinal Disease EGID prevention in the future, from reducing risk factors to managing disease severity. Expand There are four stages of disease prevention: Reducing risk factors for all patients. Preventing EGIDs in healthy but susceptible patients. Identifying patients who have early signs of EGIDs without symptoms. Reducing disease severity in patients diagnosed with EGIDs. The increase in EGIDs is likely due to a combination of factors, including specific exposures, patient characteristics, and the environment. Potential early life exposures may include prematurity, caesarean section delivery, lack of breastfeeding, and acid blocker or antibiotic use in infancy. Patient factors include other allergic diseases, poor oesophageal barrier function, and increased susceptibility to inflammation and scarring. Potential environmental factors include cold, arid climates. Future studies are needed to identify cause-and-effect relationships between these factors and the development of EGIDs. Information is provided by The Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) Learn more about CEGIRs work Donate today for a brighter tomorrow Last updated 18/7/23 ©EOS Network 2023 All rights reserved
What is the role of the microbiome in EGIDs? CEGIR explains how an imbalance in the microbiome is associated with allergic diseases such as asthma and eczema and how it's linked to eosinophilic gastrointestinal diseases (EGIDs). Expand The microbiome consists of the trillions of bacteria, fungi, viruses, and other microorganisms that live on and within us. An imbalance in the microbiome, called dysbiosis, is associated with allergic diseases such as asthma and eczema. Early-life exposures affecting the microbiome are associated with eosinophilic oesophagitis (EoE), such as antibiotic use, Caesarean delivery, and preterm delivery. Previous studies of the microbiome in EGIDs have primarily focused on the bacteria present in EoE. The healthy oesophagus has a unique microbiome with increased numbers of gram-positive bacteria from the phylum Firmicutes. Studies in mice show the unique oesophageal microenvironment dictates the oesophageal microbiome. Is EoE Associated With Changes in Bacteria in Oesophagus? EoE may be associated with increased numbers of bacteria in the oesophagus. Gram-negative bacteria (e.g., Prevotella, Neisseria, Haemophilus species) may be increased in the oesophagus of patients with EoE. The stool microbiome is not a good non-invasive test for EoE because, so far, researchers have not observed any differences in the stool microbiome of patients with EoE compared to patients without EoE. Additional studies are needed to determine if changing the microbiome (e.g., probiotics) can be used to prevent or treat EGIDs. Information is provided by The Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) Learn more about CEGIRs work Donate today for a brighter tomorrow Last updated 21/06/23 ©EOS Network 2023 All rights reserved
Can EoE cause chronic fatigue even when it is in remission? CEGIR discusses chronic fatigue among EoE patients including in remission. Expand Fatigue is common in individuals with Eosinophilic Oesophagitis (EoE). The underlying mechanisms connecting fatigue and EoE are unclear; however, patients with EoE may be at risk for nutritional deficiencies, medication side effects, and sleep disruption. Patients with EoE may also experience feelings of depression, anxiety, and isolation, which can contribute to fatigue. There is limited data on fatigue in well-controlled EoE. Information is provided by The Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) Learn more about CEGIRs work Donate today for a brighter tomorrow Last updated 21/06/23 ©EOS Network 2023 All rights reserved
What treatments are in pipeline for EoE? Learn more about treatments that are going through investigations and trials for treating EoE. Answers CEGIR. Expand Eosinophilic Oesophagitis is a lifelong disease affecting the oesophagus, the tube connecting the mouth and stomach. Too many eosinophils, a type of white blood cell, in the oesophagus, causes inflammation, making swallowing difficult. Although this condition has no cure, proper therapy can help manage symptoms. However, finding the right therapy can be challenging due to the lack of licensed treatment options available. In 2018, Jorveza Budesonide oral dissolvable tablet was the first EoE treatment approved for use in Europe and has since been approved in Australia and Canada In May of 2022, the FDA approved its first USA treatment for eosinophilic esophagitis, a biological agent called Dupilumab and has since been approved in the EU. It is used to suppress the allergic arm of the immune system and treat patients with EoE that is mainly moderate to severe. Access to these treatments depends on your age, location and other factors, currently leaving many sufferers still with off-label medications that are designed for other conditions such as asthma. In this FAQ CEGIR provides information on potential EoE treatment options currently undergoing investigation. If proven to be both effective and safe, they may become viable treatment options in the future. What Treatments For EoE Are Being Investigated Multiple treatments for EoE are being investigated at different phases of the clinical trial approval process. Dupilumab (anti-IL-4r𝛼), phase III (1-11 yrs) Monoclonal antibody that binds to the IL-4 receptor 𝛼-subunit inhibiting signalling of IL-4 and IL-13, two cytokines that play a critical role in allergic inflammation. FDA-approved for atopic dermatitis (ages 6+ mths), moderate-to-severe asthma (eosinophilic or oral steroid-dependent, ages 6+ yrs), chronic rhinosinusitis (ages 18+ yrs), eosinophilic oesophagitis (EoE) (ages 12+ yrs and weighing ≥ 40 kg), prurigo nodularis (ages 18+ yrs) Cendakimab (anti-IL-13) – phase III (12-75 yrs) Monoclonal antibody that binds to and neutralizes IL-13, a cytokine that plays a key role in EoE. Tezepelumab (anti-TLSP) – phase III (12-80 yrs) Monoclonal antibody that targets thymic stromal lymphopoietin (TSLP), a cytokine released by the epithelium that plays an important role in starting allergic inflammation. FDA-approved for add-on maintenance treatment for severe asthma (ages 12+ yrs) Lirentelimab (anti-Siglec-8) – phase II/III (12-80 yrs) Monoclonal antibody that binds to the inhibitory receptor Siglec-8, causing eosinophil death and inhibiting mast cells. Etrasimod (SP1R modulator) – phase II (18-65 yrs) Small molecule that modulates the sphingosine-1-phosphate receptor and inhibits trafficking of lymphocytes from the lymph nodes into the blood. Information is provided by The Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR) Learn more about CEGIRs work Donate today for a brighter tomorrow Last updated 21/09/23 ©EOS Network 2023 All rights reserved
How does the EOS Network get involved in clinical research as a patient organisation? Expand As a patient organisation, we are often approached for specific research areas, particularly because Eosinophilic Diseases are rare. Unlike common conditions like high blood pressure or diabetes, fewer patients are available for trials in this space. As the leading organisation for Eosinophilic Diseases in the UK, we are the primary point of contact for patient involvement. However, we maintain strict confidentiality and do not allow external parties to contact our community directly. This ensures that we manage and protect all interactions. Join our Network to learn about new opportunities. The answer is provided by Amanda Cordell, CEO and Founder of EOS Network
Do you need to stop your medication to participate in a trial? Expand It depends on the trial. Some trials will examine whether the treatment being tested can be used in addition to existing treatments. You can continue taking your current treatments alongside the trial medication in these cases. Other trials will look at a new treatment meant to be used independently. You might have to stop your current therapy to participate in the trial in this scenario. Researchers typically do not stop people from taking their medications and switch them to placebos for four years, as this would be unfair and unethical. These types of trials are carefully managed and often use multi-arm, multi-stage (MAMS) designs with multiple treatment options to ensure that participants are not left on a placebo after stopping their regular treatment, which would not be ethical. Learn more about medication management before screening: EoE Guidelines The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
Are there any avenues to conduct research ourselves? Expand One way to help is by donating your time to assist in getting clinical trials started or volunteering to support their progress. You could also participate in focus groups with EOS Network, especially if you have a specific area of interest. Another option is to sponsor a trial, which can be incredibly costly as it needs to cover costs for several years of clinical visits, blood tests, and all the staff, equipment, and facilities required to conduct the trial. EOS Network receives updates about opportunities like this. Please sign up for our newsletter to stay connected and discover current trials. The answer is provided by Amanda Cordell, SEO and Founder of EOS Network
What can research change? Expand Research can bring about changes in several key areas: New ways to prevent, detect, monitor or manage the disease New treatments New ways to existing treatments How to improve quality of life for people living with chronic illness To be accepted in the medical world, research must be based on strong data, protocols, and ethical approvals. For example, the EoE Guidelines were developed by reviewing 2,500 published papers to ensure robust evidence. Thus, every piece of research you help deliver can make a significant difference. Amanda Cordell, CEO and Founder of EOS Network "It's all about improving your quality of life when you're living with a chronic illness. As an organisation, we want to ensure that people with this disease are involved in every opportunity for research. This way, we can achieve the best quality research that is truly patient-centered."
How do I take part in a trial? Expand Living with an Eosinophilic-Associated disease provides a valuable experience that can positively impact others. There are numerous ways to contribute to research beyond participating in trials, such as: Drug trials Diagnostic trials Giving a blood sample Completing a questionnaire Taking part in a focus group Patients often inspire the idea for a trial. Don't hesitate to speak to your professional if you have any concerns or questions. If many people have the same situation, it might inspire the research. You can also help to shape the future research by: Working with research funders to help prioritise patients' needs Joining project steering groups Participating in focus groups Completing surveys to inform the study At this stage, there is no actual study, but rather the research before starting the trial. Your lived experience is vital for researchers to understand what is important for patients. You can support research by organising fundraising events. These events can help us fund our work and raise awareness about EADs. There are opportunities to join the ethics committee, as well as opportunities within ethics committees, even outside of your specific disease area. If this interests you, laypeople (individuals outside healthcare professions or healthcare services researchers) are encouraged to get involved. When it comes to NICE, it is essential to consider the cost versus benefits to the NHS and to explain the patient's experience. As more treatments become available for specific diseases, there will be more opportunities to participate. EOS Network receives updates about opportunities like this. Please sign up for our newsletter to stay connected and discover current trials. The answer is provided by Amanda Cordell, SEO and Founder of EOS Network
How do researchers recruit people for clinical trials? Expand Researchers use various methods to recruit participants for their studies. They promote the trials through social media, mail drops, and websites. Sometimes, they establish partnerships with local general practitioners (GPs) to access their patient databases and identify individuals who meet specific criteria. The GPs then approach these patients to inform them about the trial. In some cases, researchers receive referrals directly from hospitals for potential participants. The process may vary if a hospital is conducting its own trial, as they directly approach their patients to invite them to participate in the study. If a hospital conducts numerous studies, they may be less inclined to refer their patients to outside researchers. As a research organisation separate from the NHS, we do not directly contact patients; instead, patients must initiate contact with us first. Identifying suitable candidates is complicated by ethical considerations and conflicting studies. For instance, if a hospital is conducting a study, they also consider other studies that may be in competition with theirs. As a researcher, I will not be able to conduct a study that competes with any other studies I am involved in. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
Do different trial stages involve different cohorts of patients? Expand In a clinical trial, each phase involves different patients. Generally, one of the criteria for eligibility is that the patient has not previously taken the drug. Participants in a new phase will typically be a new subset of patients rather than a continuation from a previous one. However, for a phase four trial, which involves repurposing an old drug, this may not always be the case. Phase 4 studies may involve collecting data from people using the drug in their everyday lives. Some of these studies focus on using an already-approved drug for new medical conditions. Participants from a phase 3 trial may be involved in the long-term usage phase 4 trial, where they continue taking the medication in their daily lives. This requires less effort from the participants as it aligns with their routines. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
If my consultant and the research team have different opinions, whose opinion takes precedence? Expand In a trial, the research team has the most information. If the consultant is not part of the trial, they will have less trial information than the research team. The researchers are required to follow the protocol and understand it thoroughly. Therefore, from a trial perspective, the research team is the primary source of information. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
What is the average time it takes from the conception of an idea to the delivery of the treatment to people? Expand The timeline for developing a new drug or vaccine can vary significantly. If you already have data, the initial stages will be shorter. In cases of emergent need or if you already know some information, such as with COVID-19 vaccines, timelines can be expedited. This doesn't compromise patient safety but rather cuts down on bureaucratic delays. Generally, it takes about ten years from conception to completion, so I'd estimate the timeframe to be 10 to 15 years. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
If a product has been repurposed from another condition, does it alter the clinical trials' timeline? Expand Yes, because it's already in an advanced trial phase. For those in phase 4, it takes around 2 to 5 years to reach people. This is mainly due to the already proven safety and focus on effectiveness. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
Can participants follow specific diets without needing to change or stop them, and do they need to maintain a stable condition throughout the study? Expand It's important for study participants to maintain stability throughout the study. The specific requirements may vary depending on the study, but researchers generally want stable participants before, during, and after the study. This means they prefer individuals who don't constantly change their diets when participating in a diet study, as it wouldn't be beneficial. For a liver study, for example, researchers wouldn't want someone who binge drinks occasionally. Consistency and stability are vital qualities that researchers look for in study participants. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
What does it mean when a study is stopped because it didn't meet its primary endpoint? Expand An endpoint in a research study aims to answer a specific question. For example, the primary endpoint of a study could be whether a particular treatment is effective at stopping symptom X. Researchers will compare the reduction in symptom X between the treatment group and the placebo group. In addition to the primary endpoint, there are also secondary outcomes which may be related to lab data. These secondary outcomes could involve comparing lab results between the treatment and placebo groups or evaluating safety data such as the number of people admitted to the hospital or the occurrence of particular side effects. These secondary outcomes also serve as endpoints in the study. When a trial fails to reach an endpoint, it usually means that it hasn't been as effective as desired. As a result, the decision is made to discontinue the trial because the drug has not shown the required effectiveness to continue forward. Another reason for stopping a trial is safety issues. If the trial uncovers a higher-than-expected side effect profile, it may be stopped for that reason as well. This can happen years down the line. This underlines the importance of such trials and why they can continue for a long time. This is also why you might hear about a drug still being in a trial even ten years later.None of this happens quickly. It can be incredibly frustrating to have a medical condition and seek treatment. However, it's important to ensure that new drugs are thoroughly tested and safe before being released to the public. Careful management is essential to guarantee the effectiveness and safety of these new treatments. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
What other factors do you consider besides money when determining the trial duration? Expand When designing a trial, the scientists involved will examine the natural progression of a condition. They will look at how quickly the condition is expected to progress, how rapidly symptoms are likely to emerge, and how soon they anticipate their drug will take effect. For instance, if a condition progresses very slowly over time, it will take longer to determine whether there's a noticeable change with the drug compared to a placebo. If you have a condition that progresses rapidly with significant symptoms, it may be necessary to evaluate the drug's effectiveness in a shorter time frame. The duration of the trial also depends on the expected safety data. Safety trials usually run for a longer period of time. Researchers need to understand the potential long-term effects that people might experience. They are trying to determine whether a treatment is both effective and safe, and this depends on the nature of the drug. It's not a straightforward process. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
What happens to the participants at the end of the trial? Do they stop taking the drug, or can they continue if it has proven effective? Expand It depends on the study. For some trials, especially phase 2 trials, the end of the study means the end of taking the drug. Some phase 3 studies will have so-called open-label extensions. In this case, after completing the initial placebo-controlled phase, the research team will allow participants to join an extension study where they will receive the actual drug. If the drug is approved, the local NHS authority and NICE guidance will take over. They will assess factors such as effectiveness, safety, and economic viability. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
Is licensing the trial's results similar in the UK and the US? Expand The Medicines and Healthcare Products Regulatory Agency (MHRA) approves trials in the UK. A similar process is happening in the US with the Food and Drug Administration (FDA). Both organisations analyse the data from the trials. Sometimes, they can visit other countries and trial sites to examine all available data before deciding and granting the drug a license. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
What happens to the drug at the end of the study, and what are the next steps for getting it to people? Expand After the trial is finished, researchers gather the data. The drug company's scientists then review the data to decide if further development is worthwhile. Some trials conclude at this stage if the drug is deemed ineffective. If the drug is effective, they will obtain a license for it. The Medicines and Healthcare Products Regulatory Agency (MHRA) approves trials in the UK, and the Food and the US Drug Administration (FDA) does the same. (EMA in Europe) This means the regulatory authority has to agree that a drug is safe and effective before it can be licensed to treat a particular condition. In the UK, the drug also needs to be reviewed by the NHS and the National Institute for Clinical Excellence (NICE) to ensure they agree with the MHRA about its safety and effectiveness. NICE also considers the cost of the medication involved, so they focus more on health economics. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
Is this process only used for drugs, or is it used for other aspects of medical research? Expand This process is primarily used for drugs, but similar procedures are also applied to dietary changes and devices. For instance, researchers can compare diets by prescribing them to a specific group while allowing another group to continue their regular diet. Similarly, with a wearable device like a blood sugar monitor, one group wearing it can be compared to another group not using it.Observational trials are also incredibly important for looking at risk factors and diagnostics. With a rare condition, diagnosis in itself can be as much of a challenge as the actual treatment, and getting that diagnostic tool right is just as important as getting the treatment when you finally get the drug. And it's not just about drugs. Observational studies can examine whether a particular type of scan is more effective than the currently available scans or whether a specific blood test can help detect a symptom before it becomes a full-blown condition. So, there are lots of different avenues for people to get involved. Another example is a trial that studies swallowing patterns to see if there's a better way to define what happens to swallowing technique in people with EoE and to measure the management of their condition. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd
Where do children fit into the process of clinical trials? Expand Paediatric studies are very important, but they are more rare.The majority of studies focus on adult populations. Conducting trials on young individuals presents unique challenges, including concerns about potential risks and side effects and the impact on their education due to appointments. When working with children, there are ethical considerations and additional complications, such as parent participation.The decision to involve children and adolescents in studies depends on the disease researchers investigate, the pharmaceutical company involved, and the likelihood of a child developing the condition. However, these studies are vital because this is the only way to understand if a drug is safe for children. The answer is provided by Karen Faulkner, Senior Research Physician at Synexus Clinical Research Ltd