P2390 - Patient-Reported Symptom Improvement Following Treatment With APT-1011 in Patients with Eosinophilic Esophagitis as Measured by the PROSE: Results from FLUTE, A Phase 2b Clinical Trial

Ralph Turner, PhD, MPH¹, Jeffrey M. Rohay, PhD, MSIS¹, Nicole Sparling, ¹, Joy Whitsett, ¹, Gina Eagle, MBBS, FRACP², James Nezamis, MS², karol Knoop, RN, BS, CCRA², Evan S. Dellon, MD, MPH, FACG³, Ikuo Hirano, MD⁴, Jean Paty, PhD^1
1IQVIA, New York, NY; ²Ellodi Pharmaceuticals, LP, Lawrenceville, NJ; ³University of North Carolina School of Medicine, Chapel Hill, NC; ⁴Northwestern University Feinberg School of Medicine, Chicago, IL

Introduction: Dysphagia is a key symptom of eosinophilic esophagitis (EoE) and should be evaluated during the development of therapies for EoE. Validated patient-reported outcomes (PROs) for dysphagia are needed. We assessed dysphagia symptoms as measured by the Patient Reported Outcomes for the Symptoms of Eosinophilic esophagitis (PROSE) diary in the Phase 2b FLUTE clinical trial (Fluticasone in EoE), a randomized, double-blind, placebo-controlled, dose-ranging trial examining the safety, efficacy and pharmacokinetics of APT-1011 for EoE.

Methods: The primary objective of the FLUTE trial was to evaluate histologic remission rates of APT-1011 at Week 12 in adults with EoE. In addition, the PROSE diary was evaluated as a potential instrument over the treatment period. This instrument captures episodes of dysphagia and the severity of each episode (defined by 3 parameters: pain, difficulty, and discomfort) on a daily basis. Each parameter was measured by a numerical scale (0-10) with higher scores indicating more severe symptoms. The episode severity score was the average of the individual parameter scores (mean 0-10). Patients were instructed to complete the diary on a hand-held device for each episode in real time as well as every evening, wherein they recorded episodes not entered in real-time. The daily composite severity rating was calculated by averaging all severity scores recorded for each day. Meaningful effect size differences were evaluated via the Cohen Coefficient method: 0.2 is small but important, 0.5 is moderate, 0.8 is large (Cohen J. Statistical power analysis for the behavioral sciences. Lawrence Erlbaum Assoc: Hillsdale, NJ; 1987, 24-27).

Results: 106 adults meeting selection criteria were randomized to 1 of 4 doses of APT 1011 (1.5mg HS, 1.5mg BID, 3mg HS, and 3mg BID) or placebo. At baseline the mean number of episodes was 14.10 vs 15.59 for 3mg HS vs placebo and decreased after 12 weeks of treatment by -10.55 and -6.00 respectively. Meaningful effect sizes were observed between the 3mg HS dose and placebo group for dysphagia frequency over 14 days (-0.460), as well as for the mean daily composite episode severity rating (-0.252). (Table 1).

Discussion: The PROSE diary was successfully implemented in a clinical trial to measure episodes of dysphagia. Patients reported a meaningful improvement to their dysphagia symptoms following treatment with APT-1011 3mg HS vs placebo, indicating responsiveness of this new symptom metric.



Disclosures:


Ralph Turner, PhD, MPH¹, Jeffrey M. Rohay, PhD, MSIS¹, Nicole Sparling, ¹, Joy Whitsett, ¹, Gina Eagle, MBBS, FRACP², James Nezamis, MS², karol Knoop, RN, BS, CCRA², Evan S. Dellon, MD, MPH, FACG³, Ikuo Hirano, MD⁴, Jean Paty, PhD¹. P2390 - Patient-Reported Symptom Improvement Following Treatment With APT-1011 in Patients with Eosinophilic Esophagitis as Measured by the PROSE: Results from FLUTE, A Phase 2b Clinical Trial, ACG 2021 Annual Scientific Meeting Abstracts. Las Vegas, Nevada: American College of Gastroenterology.