By Bailey Arman, associate director of communications, science & education.
Because of celiac disease, “the spontaneity in my life has been wiped out,” shared one participant with a small group of doctors, researchers, pharmaceutical representatives, and fellow patients in a gray meeting room outside of Washington D.C. The group was gathered there, in May of 2024, to discuss something potentially life-changing to the millions of people living with celiac disease: what the final stages of developing a medication for the condition should look like.
The Purpose and the People
Recognizing the swift progress of potential treatments for celiac disease, the Coalición Más allá de la Celiaquía organized the meeting to prepare for the last stages of treatment development—the goliath of a phase 3 clinical trial.
They invited representatives from the FDA and people living with celiac disease to the discussion, so that both perspectives can be represented in the design of future clinical trials.
The Coalition recently published these perspectives and takeaways in the scientific journal Alimentary Pharmacology & Therapeutics. The main points are:
- The first approved drug(s) would be used in addition to the gluten-free diet
- Phase 3 trials must track intestinal healing and symptoms
- The ideal trial participant should be specific and described at the start of the trial
The roles of the gluten challenge, trial length, and biopsy standards were also discussed. Let’s dig into the details.
What Does A Celiac Disease Treatment Do?
Not a Diet Replacement
All drugs that are currently being developed to treat celiac disease would, if approved, be used alongside a gluten-free diet to treat patients. Some attendees noted that this should be explained to the patient volunteers who generously choose to participate in clinical trials for the development of these drugs.
Drug Must Address Internal Damage and Symptoms
FDA representatives were firm that phase 3 trials for celiac disease drugs must track intestinal healing and symptoms—not one or the other. This can be tricky with a condition like celiac disease, where the symptoms don’t always match the severity of the internal damage, and vice versa.
Decreasing Patient Burden While Maintaining Scientific Standards
The Preferred Patient
Celiac disease can affect many different types of people in many different ways, from the very young to the very old, in all shapes, sizes, and ethnicities. Some have GI symptoms, some have neurological symptoms, and some have no symptoms at all. This variability makes it difficult to track whether a drug is working for all these different types of patients, which is why all parties present agreed that the criteria for who qualifies to join a trial should be as specific as possible and clearly defined at the start of the trial.
Role of Gluten Challenge
Drug developers usually need to have a gluten challenge—a controlled distribution of certain amounts of gluten—to prove their celiac disease drug works. It can make recruiting patients for trials tough, because it means they must willingly consume the thing that makes them sick. Meeting participants discussed ways to make this less burdensome by honing in on the exact dose needed for each type of trial, or even not requiring gluten consumption for certain trials.
Recruiting patients who still experience symptoms on a strict, gluten-free diet is one way that some companies have tried to avoid a gluten challenge, but the group acknowledged that because of the Hawthorne effect—a phenomenon where people change their behavior when monitored—trials may still want to give a low but consistent dose of gluten to ensure any successful results are due to the drug, and not due to patients who are more careful with their food when supervised by doctors.
Standardizing How We Track Damage
The gold standard for diagnosing celiac disease is an endoscopy: putting a camera into the small intestine and biopsying it—clipping tiny pieces for further study—to confirm the damage is because of celiac disease.
Typically, clinical trials measuring intestinal damage require two endoscopies with biopsy. This allows them to track how well someone is healing by comparing a biopsy from the start of the study with a second one at the end of the study.
The group at the meeting agreed that standardizing how these biopsies are collected—for example, ensuring all doctors collect biopsies from a similar place in the small intestine—is crucial to comparing how well a specific drug works compared to another.
A Tough Time Commitment
Any potential medication in the United States has to go through three different phases of testing. Drugs for celiac disease have made it to phase 2, but never to phase 3.
When it comes to phase 3 trials, the FDA wants celiac disease patients to participate for a full year. But instead of patients potentially undergoing a gluten challenge for a year with no drug—as a participant in the placebo group may have to—the Beyond Celiac Coalition pushed to divide this year into two sections: a 6-month period when some may receive a placebo and an additional 6 months where all patients knowingly receive the drug. This would reduce the burden on everyone and allow researchers to collect data on whether patient behavior changes (ie, whether they eat more gluten) when they know they’re taking the drug.
Looking Forward
Drug Will Be Initially Be Restricted
The group acknowledged that if a drug were approved, it would not be available to everyone at first. The two categories of excluded patients mentioned were children (people under 18 years old) and asymptomatic patients, who have internal damage but no symptoms. Both groups would require additional trials tailored to their specific needs before the drug is approved for their use.
Continuing the Conversation
The conversations around what makes clinical trials safe, effective, and worthwhile shouldn’t end with our group of a few dozen—they should expand and multiply with the wider scientific and patient community. That’s why we published these findings.
We’ve also continued the conversation by organizing a second meeting with the FDA, which took place in February of 2026. Attendees discussed what trials for children might look like, once a drug is approved in adults. We believe it’s important to provide this information to anyone who may be interested, so we hope to publish those findings as well. Subscribe to our research news feed to ensure you hear about it when it’s public!
Learn More
- Read the article published in Alimentary Pharmacology & Therapeutics
- Check out the drugs currently in development
- Get involved in clinical trials
- Learn more about the Coalición Más allá de la Celiaquía