In short

  1. Celiac disease (CeD) remains underdiagnosed and underserved, with limited treatment options
  2. Enrollment is recognized to be difficult – trials often require changes to diet and relatively invasive procedures
  3. A sponsor approached PSI for their multi-region clinical trial in CeD, which needed to run on time to support study delivery and broader development timelines
  4. Together, the sponsor and PSI engaged potential patients through diverse recruitment strategies, including advocacy group outreach, social media campaigns, and educational initiatives
  5. A comprehensive site engagement plan, implemented across nearly 50 sites in four countries, helped ensure maximal activation and commitment from PIs
  6. The roles of KOLs were also clearly defined: using their networks and scientific engagement channels, and sharing educational materials, KOLs directly increased trial visibility and investigator engagement
  7. Ultimately, the trial achieved site activation in 3.5 months, reached FPI and LPI milestones on time
  8. Collaboration with a CRO with years of experience in the gastrointestinal therapeutic area and with global reach transformed potential challenges into manageable processes and successful enrollment

Background

Unmet needs in CeD

Celiac disease is a chronic, immune-mediated gastrointestinal condition that remains significantly underdiagnosed and underserved [1], affecting approximately 1–2% of the population in countries where wheat is a staple [2]. Diagnosis requires blood tests and an intestinal biopsy [1].

A strict, gluten-free diet, the current treatment, is difficult to follow [3]. Many patients continue to experience symptoms despite adherence, facing complications such as anemia, malnutrition, and increased autoimmune disorder risk [4], underscoring the need for new therapies.

Unique obstacles in CeD patient enrollment

Patient recruitment and site engagement are key challenges in CeD studies [5]. Patients fear dietary changes, and trials require time, commitment, and invasive evaluations.

Site engagement is complicated by diverse healthcare system requirements and logistical demands. Underestimated timelines often place pressure on study delivery and market access planning.

The challenge

A sponsor approached PSI to collaborate on delivering their CeD trial. Recognizing enrollment is only part of the challenge, they required a comprehensive strategy to bring the potentially life-changing therapy to market quickly.

Read on for an overview of PSI’s three-part strategy to ensure this celiac disease clinical trial met its milestones on time.

Enrollment

The solutions

Diverse patient recruitment methods

The bespoke, comprehensive patient engagement strategy included:

  • A gap analysis to reveal novel recruitment routes, including activating patient advocacy groups, support groups, and local CeD associations.
  • Implementing tailored patient (and ongoing) recruitment methods, including multi-channel social media campaigns to drive patient awareness of the sponsor and their study
    • One innovation was community-based educational initiatives tailored to patient lifestyles
    • Community event and health fair participation was successful across the regions, and helped boost patient support group, referral network, and outreach efforts
  • Developing FAQs and study guides and providing financial/logistical support to encourage patient retention.

Comprehensive site engagement

Multinational site support specialists initiated site-specific recruitment plans. Prescreening activities, chart reviews, community outreach events, and referral follow-ups helped to pave the way for maximal site engagement. This was supported by tailored recruitment and retention toolkits and by hosting site engagement meetings to ensure continued commitment.

Effective KOL activation

Building on the relationships with PIs and KOLs is always crucial. The sponsor and PSI strategically engaged the most effective KOLs, ensuring the trial was well-perceived and supported. A global network of KOLs means:

  • Access to recognized disease experts with high credibility in CeD and the wider gastrointestinal therapeutic area, able to motivate PIs
  • The ability to have experts actively engaged in identifying high-performing sites or advising on country feasibility
  • Documented scientific evaluation and justification of the trial protocol, improving regulator perception

Examples of important KOL activation methods employed in this trial included:

  • Referral networking
  • Individual outreach via medical conferences/congresses
  • Outreach via lunch-and-learn visits (KOLs, PCPs, and non-physician recruiters)
  • Provision of educational materials for KOLs and for MSLs
  • Community events
  • Digital advertising in target journals

The outcome

Implementing PSI’s three-part strategy meant the trial achieved its milestones on time.

This is a major accomplishment… truly a pleasure to work with such a talented and experienced group. Without each of you… we couldn’t have achieved this amazing goal.

Director of Clinical Operations, Sponsor

Key takeaways

Enrolling in CeD is challenging, but choosing a specialty CRO with expertise in chronic gastrointestinal conditions means gaining a partner who can think outside the box across recruitment strategies, site engagement, and KOL activation. Studies can be enrolled on time with reduced risk, turning a potential bottleneck into a predictable, manageable process.

Learn how PSI can support your gastrointestinal study

Learn more

Abbreviations

CeD, celiac disease;
FAQ, frequently-asked questions;
KOL, Key Opinion Leader;
MSL, Medical Science Liaison;
PCP, primary care physician;
PI, Principal Investigator.

References

1. Catassi, C., et al., Coeliac disease. Lancet, 2022. 399(10344): p. 2413–2426.
2. Myleus, A. and Catassi, C., Epidemiology of Celiac Disease. Gastrointest Endosc Clin N Am, 2025. 35(4): p. 693–706.
3. Leffler, D.A., et al., Factors that influence adherence to a gluten-free diet in adults with celiac disease. Dig Dis Sci, 2008. 53(6): p. 1573–81.
4. Lauret, E. and Rodrigo, L., Celiac disease and autoimmune-associated conditions. Biomed Res Int, 2013. 2013: p. 127589.
5. Celiac Disease Foundation. Qualify for a study or trial – Celiac disease. January 2026]; Available from: https://celiac.org/icureceliac/participate-in-research/.