This is a 24-week randomized, double-blind, placebo-controlled induction study of APT-1011 in adults (≥18 years old) with eosinophilic esophagitis (EoE) followed by a single-arm, open-label extension. This study will evaluate the efficacy and safety of APT-1011 3 mg administered HS (hora somni, at bedtime) for the induction of response to treatment (symptomatic and histologic) over 24 weeks. The open-label extension will continue to evaluate long-term safety in subjects who consent to continue on open-label treatment with APT-1011.
The efficacy and safety of APT-1011 3 mg administered at bedtime will be evaluated for the induction of response (histologic and symptomatic) after 24 weeks of treatment. After completing 24 weeks of double-blind study treatment, subjects may consent to participate in the open-label extension, otherwise they will complete study drug treatment and enter a 2-week off treatment safety follow-up. The duration of the double-blind portion of the study, screening through follow-up visit for subjects completing study drug at Week 24, will be up to 32 weeks long, i.e., 6-week screening period (the includes a 4-week run-in phase) followed by 24 weeks induction phase and 2 weeks off-treatment follow-up. For subjects consenting to participate in the open-label extension, the duration of the study will be determined by the Sponsor.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
218
APT-1011 is an orally disintegrating tablet that includes fluticasone propionate as its active ingredient. Other Names: fluticasone propionate
Placebo orally disintegrating tablet. Other Names: PBO
Esophagogastroduodenoscopy (EGD) is a test that involves an endoscope, a lighted camera on the end of a tube, that is passed down a subject's throat to visualize their esophagus
Histological Remission (Co-Primary Outcome Measure)
To evaluate the percentage of subjects with histological remission (defined ≤ 6 peak eosinophils \[eos\]/high power field \[HPF\] on esophageal mucosal biopsies at Week 24). HPF will be defined as a standard area of 235 square microns in a microscope with 40x lens (0.3 mm\^2) and 22 mm ocular
Time frame: Week 24
Complete Symptomatic Response (Co-Primary Outcome Measure)
To evaluate the percentage of subjects with complete symptomatic response at Week 24 (defined as zero dysphagia episodes in the 14 consecutive days prior to Week 24)
Time frame: Week 24
Clinicopathologic Responder Rate
To compare the percentage of clinicopathologic responders, defined as having complete symptomatic AND histological response at Week 24 (defined as zero dysphagia episodes in the 14 consecutive days prior to Week 24 AND ≤ 6 peak eos/HPF on esophageal mucosal biopsies)
Time frame: Week 24
Percentage of Subjects with ≥70% Reduction in Dysphagia Frequency
To evaluate the percentage of subjects with ≥70% reduction in dysphagia frequency at Week 24 as compared to baseline (as measured over the 14 consecutive days prior to each visit)
Time frame: Week 24
Mean Change in Dysphagia Frequency
To compare the mean change from baseline to Week 24 in dysphagia frequency (as measured over the 14 consecutive days prior to each visit)
Time frame: Week 24
Mean Change in PROSE Difficulty Swallowing
To compare the mean change from baseline to Week 24 in difficulty swallowing using the Patient Reported Outcomes Symptoms of Eosinophilic Esophagitis (PROSE)
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Digestive Health Specialists
Dothan, Alabama, United States
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Mobile, Alabama, United States
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Scottsdale, Arizona, United States
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Tucson, Arizona, United States
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Little Rock, Arkansas, United States
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North Little Rock, Arkansas, United States
Om Research LLC
Camarillo, California, United States
Providence Facey Medical Foundation
Mission Hills, California, United States
United Medical Doctors
Murrieta, California, United States
TriWest Research Associates, LLC
San Diego, California, United States
...and 50 more locations
Time frame: Week 24
Mean Change in PROSE Pain with Swallowing
To compare the mean change from baseline to Week 24 in pain with swallowing using the PROSE
Time frame: Week 24
Mean Number of Dysphagia-Free Days
To compare the mean number of dysphagia-free days from baseline to Week 24
Time frame: Week 24
Percentage of Responders (Strictures and ≥Grade 2 rings)
To compare the percentage of responders, defined as no longer having strictures and/or ≥Grade 2 rings which were present at baseline, at Week 24
Time frame: Week 24
Percentage of Responders (Strictures)
To compare the percentage of responders, defined as no longer having strictures which were present at baseline, at Week 24
Time frame: Week 24
Percentage of Responders (≥Grade 2 rings)
To compare the percentage of responders, defined as no longer having ≥Grade 2 rings which were present at baseline, at Week 24
Time frame: Week 24
Mean Change in EREFs
To compare endoscopic appearance evaluated by the mean change from baseline to Week 24 in Eosinophilic Esophagitis Endoscopic Reference Score (EREFs)
Time frame: Week 24
Time to First Complete Symptom Response
Time to first complete symptom response (defined as zero dysphagia episodes in a 14-consecutive-day period)
Time frame: Week 24