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Standardizing Randomized Controlled Trials in Celiac Disease: An International Multidisciplinary Appropriateness
Benjamin Lebwohl1, Christopher Ma2, Stephen M Lagana3
1Celiac Disease Center, Department of Medicine, Columbia University College of Physicians and Surgeons, New York, New York; Department of Epidemiology, Mailman School of Public Health, Columbia University, New York, New York.
Gastroenterology
|September 13, 2023
Summary
Developing new celiac disease (CeD) treatments requires standardized clinical trial designs. This study provides consensus recommendations for CeD randomized controlled trials (RCTs), focusing on eligibility and outcome measures.
Area of Science:
- Gastroenterology
- Clinical Trial Design
- Celiac Disease Research
Background:
- Safe and effective pharmacologic treatments for celiac disease (CeD) are needed.
- A lack of consensus exists regarding the optimal design of randomized controlled trials (RCTs) for CeD.
- Standardized approaches are crucial for advancing CeD therapeutic development.
Purpose of the Study:
- To establish consensus on the appropriate design and configuration of RCTs for celiac disease.
- To provide standardized recommendations for key aspects of CeD clinical trials.
- To guide future research in pharmacologic treatments for CeD.
Main Methods:
- A 2-round modified Research and Development/University of California Los Angeles Appropriateness Method study was employed.
- Eighteen expert gastroenterologists and gastrointestinal pathologists voted on statements regarding CeD RCTs.
- Two distinct RCT designs were evaluated: those incorporating a gluten challenge and those assessing reversal of histologic changes.
Main Results:
- Recommendations were established for participant eligibility criteria, including dietary adherence and baseline histology.
- Specific guidance was provided for conducting gluten challenges (e.g., FODMAPs-free) and selecting outcome measures.
- Both histologic and patient-reported outcomes were considered appropriate for evaluating treatment efficacy in CeD trials.
Conclusions:
- Standardized recommendations for CeD clinical trial design, eligibility, and outcome measures have been developed.
- These recommendations aim to improve the consistency and rigor of future RCTs in celiac disease.
- The consensus provides a framework for evaluating novel pharmacologic interventions for CeD.
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