Before a new drug can be approved for a disease or condition, it has to go through a clinical trial. Clinical trials are important because they give us new treatments, such as drugs, devices, or diets.
What Is a Clinical Trial?
A clinical trial is a type of research study that tests if a treatment is safe and effective in humans. A new drug or treatment has to go through phases of testing. Once a clinical trial for a new medicine has gone through 3 phases, researchers send the results to the Food and Drug Administration (FDA) for review. The FDA looks at the results of the trial to decide if they will approve the treatment.
In February 2024, Xolair® (omalizumab) was approved for food allergies. Before that time, no other medicine to treat food allergies was available. They could only be managed by completely avoiding your food allergens or through oral immunotherapy (OIT).
Xolair’s approval for food allergies was the result of the OUtMATCH clinical trial. The people who volunteered to be part of the trial were important in making this new medicine available to people with food allergies.
Nicole Rogers, Associate Project Director at Rho, a full-service contract research organization, worked on the OUtMATCH trial. She has seen firsthand how clinical trials can lead to big breakthroughs for people with conditions such as food allergies.
Kids with Food Allergies (KFA) spoke with Nicole about the trial, as well as what it’s like to take part in research that can lead to new treatment options.
KFA: Can you give a brief summary of the OUtMATCH clinical trial?
Nicole: OUtMATCH is a clinical trial that had 3 stages, each with different goals but all focused on patients as young as 1 year of age with multiple food allergies. To be eligible [take part], patients had to be determined allergic to peanut and 2 other study-specific foods, which included cashew, milk, egg, hazelnut, walnut, and wheat. This eligibility was based on results of skin prick tests (SPT), immunoglobulin E (IgE) lab values, and oral food challenges (OFC) for each of the of the foods that would be considered their patient-specific foods for the trial.
KFA: What was the experience like for patients and caregivers during the trial?
Nicole: This trial required a huge commitment from patients and their families. As mentioned above, to be eligible, patients had to complete screening visits consisting of blood draws, skin prick tests, and 4 oral food challenges. Once a patient was enrolled, they attended in-clinic injection visits every 2 to 4 weeks for up to 1.5 years through stages 1 and 2, and then completed an additional 1 and 2 years of follow-up in stage 3 depending on when the patient enrolled.
In addition to the in-clinic visits in stage 2, patients also consumed daily home doses of oral immunotherapy (OIT) powder that consisted of either the 3 patient specific allergens mixed together or of an oat placebo powder. Patients and caregivers would complete daily diaries documenting how much of the OIT dose was consumed and listing any dosing symptoms or reactions that occurred along with any medications that were taken as preventative or treatment of a suspected food allergy reaction.
KFA: Even though the study focused on peanut, milk, egg, wheat, cashew, hazelnut, and walnut, how were you able to find out that Xolair would work for all IgE food allergies?
Nicole: Based on the strong results for peanut, which is 1 of the most common and potent food allergens, along with the results for the additional foods tested, the investigators for the OUtMATCH trial were confident that Xolair would be effective against other food allergens that were not tested given that the mechanism of action [how a medicine works in your body] is the same for all foods. With that said, it is important to always consult with your doctor or allergist before taking any medication with the intent of treating or preventing symptoms related to food allergies to ensure that it is the best fit for your situation.
KFA: Why is it important for people with food allergies to participate in clinical trials?
Nicole: This trial has provided a treatment option to protect against accidental exposures to food allergens. However, hopefully, with continued research, there will one day be evidence of a treatment that will have the ability to modify the underlying disease that causes food allergies instead of patients only having protection while receiving therapy.
KFA: What advice would you give a family who is considering having their child participate in a clinical trial for food allergies?
Nicole: Don’t be afraid to ask questions. That is what the clinical site staff are there for, and they are happy to answer them for you. Also, be open about aspects of the proposed trial that will and will not work for you or your family. Enrolling in a trial and being compliant with the treatment and visits are of the utmost importance to ensure a successful trial.
KFA: If a family decides to participate in a food allergy clinical trial, what can they expect? How are concerns, such as anaphylaxis, typically handled?
Nicole: Anaphylaxis is taken very seriously by the investigators and site staff that are conducting clinical trials. The site staff for the OUtMATCH trial were all extensively trained on the signs and symptoms to watch for during treatments that could result in severe allergic reactions, and they had medicines to combat anaphylaxis such as epinephrine readily available.
KFA: What does a successful clinical trial like the OUtMATCH trial mean for the future of food allergy research and treatments?
Nicole: A trial like OUtMATCH gives patients and caregivers a new hope in knowing that there is a treatment that may prevent them from having severe reactions in the case of an accidental exposure to a food allergen. For many patients and families, this was a fear that caused limitations in how they lived their daily lives.
The success of this trial and approval of Xolair helps reduce the fear that comes with food allergies but does not take it away completely so continued research is important to hopefully one day find therapies that not only prevent or treat reactions but instead modify the underlying disease and remove the fear of life-threatening food allergies in patients’ lives completely.
About Nicole Rogers
Nicole Rogers is Associate Project Director at Rho, a full-service contract research organization. She has more than 7 years of clinical research experience with the last 3 years being focused on food allergy. Nicole is passionate about mentoring and developing clinical research skills in all members of her study teams, which she views as a key to setting up clinical studies for success.
