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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-000082-16 | EudraCT Number |
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| Name | Class |
|---|---|
| FGK Clinical Research GmbH | INDUSTRY |
| FGK Representative Service B.V. | UNKNOWN |
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This is a randomized, placebo-controlled, double-blind trial to evaluate the efficacy, tolerability, and safety of ESO-101 in adult patients with active eosinophilic esophagitis (EoE). Patients will be screened at 2 visits (Visit 1 and Visit 2) during which their eligibility will be assessed based on endoscopy-independent criteria (Visit 1) and based on the histologic assessment of esophageal biopsy samples taken during the screening endoscopy (Visit 2). Eligible patients will be randomized 2:1 to once-daily treatment with ESO-101 or placebo and treated for 28 days starting on Day 0. Further clinic visits will be performed at Day 14 (Visit 4) and Day 28 (Visit 5, end of treatment) to assess the efficacy, tolerability, and safety. In addition, a safety follow-up call will be scheduled 2 weeks after the end of treatment (Day 42, Visit 6).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ESO-101 | Experimental | Oral use of 1 hard gelatin capsule (800 μg) |
|
| Placebo | Placebo Comparator | Oral use of 1 hard gelatin capsule |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ESO-101 | Drug | Daily administration in the evening at bedtime for 28 days |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute change in peak eosinophil count from Baseline to end of treatment | The processing and analysis of biopsy samples taken at Visit 2 and end of treatment (Visit 5) will be performed blinded at a central laboratory according to a laboratory manual. At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Histology results of the biopsies at Visit 2 will be considered baseline values. For the peak number of eosinophils, hematoxylin and eosin stained esophageal biopsy specimen will be assessed and the high-powered fields with the highest density of eosinophils will be counted. | From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of patients with histological remission, defined as the reduction of peak eosinophil count in all esophageal samples to <15 eosinophils/hpf at end of treatment, overall and determined differentially in each of the 3 esophageal segments | Biopsy samples will be taken at Visit 2 and end of treatment (Visit 5). At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Histology results of the biopsies at Visit 2 will be considered baseline values. For the peak number of eosinophils, hematoxylin and eosin stained esophageal biopsy specimen will be assessed and the high-powered fields (hpfs) with the highest density of eosinophils will be counted. |
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Inclusion Criteria:
Adult patients aged 18-70 years;
Confirmed clinicopathological diagnosis of EoE (eosinophilic esophagitis);
Active and symptomatic EoE, defined as:
Written informed consent;
Willingness and ability to comply with the protocol for the duration of the trial;
Negative pregnancy test at Screening (Visit 1) and Day 0 (Visit 3) in women of childbearing potential (i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy);
Women of childbearing potential must be willing to use (for a least 3 monthly cycles before the screening endoscopy [Visit 2] and until 4 weeks after the last intake of IMP) a highly effective method of contraception or birth control (failure rate less than 1% per year when used consistently and correctly). Reliable methods for this trial are:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Isabelle Racamier | EsoCap AG (Malzgasse 9, 4052 Basel, Switzerland) | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Facharztzentrum Eppendorf | Hamburg | Germany | ||||
| Universitätsklinikum Leipzig AöR |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39676687 | Derived | Lucendo AJ, Nantes-Castillejo O, Straumann A, Biedermann L, Bredenoord AJ, Guagnozzi D, Blas-Jhon L, Wiechowska-Kozlowska A, Weidlich S, von Arnim U, Santander-Vaquero C, Perello A, Perez-Martinez I, Barrio J, Vieth M, Gouya G, Dellon ES. Clinical Trial: Safety and Efficacy of a Novel Oesophageal Delivery System for Topical Corticosteroids Versus Placebo in the Treatment of Eosinophilic Oesophagitis. Aliment Pharmacol Ther. 2025 Feb;61(3):444-455. doi: 10.1111/apt.18443. Epub 2024 Dec 16. |
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The placebo will be identical to the test product in terms of appearance, constitution of inactive ingredients, packaging, labeling and administration.
| Placebo | Drug | Daily administration in the evening at bedtime for 28 days |
|
| From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Proportion of patients with a peak eosinophil count in all esophageal samples of <6 eosinophils/hpf at end of treatment, overall and determined differentially in each of the 3 esophageal segments | Biopsy samples will be taken at Visit 2 and end of treatment (Visit 5). At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Histology results of the biopsies at Visit 2 will be considered baseline values. For the peak number of eosinophils, hematoxylin and eosin stained esophageal biopsy specimen will be assessed and the high-powered fields (hpfs) with the highest density of eosinophils will be counted. | End of treatment (Visit 5 = day 28) |
| Proportion of patients with an improvement in the dysphagia severity score from Baseline to end of treatment | Patients will rate the dysphagia severity score daily on a 11-point NRS (numeric rating scale): 0 = 'no symptoms' to 10 = 'worst possible symptoms'. At Visit 3 (Day 0) and Visit 5 (end of treatment), the worst severity scores out of the 7 days preceding the respective visit will be used for the assessment. The score assessed at Visit 3 (Day 0) at the center before IMP intake will serve as baseline score. Patients will additionally assess the severity score of the actual day in the patient diary daily after IMP intake starting on Day 0. | From Baseline (Visit 3) to end of treatment (Visit 5 = 28 days after Visit 3) |
| Absolute change in mean eosinophil count from Baseline to end of treatment | The processing and analysis of biopsy samples taken at Visit 2 and end of treatment (Visit 5) will be performed blinded at a central laboratory according to a laboratory manual. At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Histology results of the biopsies at Visit 2 will be considered baseline values. For the peak number of eosinophils, hematoxylin and eosin stained esophageal biopsy specimen will be assessed and the high-powered fields with the highest density of eosinophils will be counted. | From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Relative change in mean eosinophil count from Baseline to end of treatment | The processing and analysis of biopsy samples taken at Visit 2 and end of treatment (Visit 5) will be performed blinded at a central laboratory according to a laboratory manual. At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Histology results of the biopsies at Visit 2 will be considered baseline values. For the peak number of eosinophils, hematoxylin and eosin stained esophageal biopsy specimen will be assessed and the high-powered fields with the highest density of eosinophils will be counted. | From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Proportion of patients with a relative reduction in peak eosinophil count of ≥30 percent from Baseline to end of treatment | The processing and analysis of biopsy samples taken at Visit 2 and end of treatment (Visit 5) will be performed blinded at a central laboratory according to a laboratory manual. At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Histology results of the biopsies at Visit 2 will be considered baseline values. For the peak number of eosinophils, hematoxylin and eosin stained esophageal biopsy specimen will be assessed and the high-powered fields with the highest density of eosinophils will be counted. | From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Proportion of patients with a relative reduction in peak eosinophil count of ≥50 percent from Baseline to end of treatment | The processing and analysis of biopsy samples taken at Visit 2 and end of treatment (Visit 5) will be performed blinded at a central laboratory according to a laboratory manual. At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Histology results of the biopsies at Visit 2 will be considered baseline values. For the peak number of eosinophils, hematoxylin and eosin stained esophageal biopsy specimen will be assessed and the high-powered fields with the highest density of eosinophils will be counted. | From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Proportion of patients with a relative reduction in peak eosinophil count of ≥75 percent from Baseline to end of treatment | The processing and analysis of biopsy samples taken at Visit 2 and end of treatment (Visit 5) will be performed blinded at a central laboratory according to a laboratory manual. At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Histology results of the biopsies at Visit 2 will be considered baseline values. For the peak number of eosinophils, hematoxylin and eosin stained esophageal biopsy specimen will be assessed and the high-powered fields with the highest density of eosinophils will be counted. | From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Proportion of patients with histological remission AND improvement in the dysphagia severity score from Baseline to end of treatment | The processing and analysis of biopsy samples taken at Visit 2 and end of treatment (Visit 5) will be performed blinded at a central laboratory according to a laboratory manual. At both visits, 6 biopsy samples will be taken, 2 each from the proximal, mid, and distal segment of the esophagus. Additionally, patients will rate the dysphagia and odynophagia severity score daily on a 11-point NRS (numeric rating scale): 0 = 'no symptoms' to 10 = 'worst possible symptoms'. Patients will assess the severity score of the actual day in the patient diary daily after IMP intake starting on Day 0. | From Baseline (Visit 2 + 3) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Absolute change in dysphagia and odynophagia severity scores from Baseline | Patients will rate the dysphagia and odynophagia severity score daily on a 11-point NRS (numeric rating scale): 0 = 'no symptoms' to 10 = 'worst possible symptoms'. Patients will assess the severity score of the actual day in the patient diary daily after IMP intake starting on Day 0. | From Baseline (Visit 3) to end of treatment (Visit 5 = 28 days after Visit 3) |
| Relative change in dysphagia and odynophagia severity scores from Baseline | Patients will rate the dysphagia and odynophagia severity score daily on a 11-point NRS (numeric rating scale): 0 = 'no symptoms' to 10 = 'worst possible symptoms'. Patients will assess the severity score of the actual day in the patient diary daily after IMP intake starting on Day 0. | From Baseline (Visit 3) to end of treatment (Visit 5 = 28 days after Visit 3) |
| Time to achieve symptom relief (defined as 50 percent improvement in the dysphagia or odynophagia symptoms on an NRS compared to Baseline) | Patients will rate the dysphagia and odynophagia severity score daily on a 11-point NRS (numeric rating scale): 0 = 'no symptoms' to 10 = 'worst possible symptoms'. Patients will assess the severity score of the actual day in the patient diary daily after IMP intake starting on Day 0. | From Baseline (Visit 3) to end of treatment (Visit 5 = 28 days after Visit 3) |
| Change in the EREFS from Baseline to end of treatment | The EREFS score (Eosinophilic esophagitis endoscopic reference score) is a classification and grading system for the endoscopic assessment of the esophageal features of eosinophilic esophagitis. The grading is used either to assess the severity of endoscopic findings from 0=´none´ to 2 or 3=´severe´ or to classify 0=´absent´ or 1=´present´.The total score will be calculated and compared between Baseline and end of treatment. A higher score correlates with more severe eosinophilic esophagitis symptoms. | From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2) |
| Incidence of treatment-emergent Adverse Events | All Adverse Events occurring from the first investigational medicinal product administration onwards are defined as treatment-emergent Adverse Events. | Visit 3 (Day 0) to day 42 |
| Incidence of treatment-emergent Serious Adverse Events | All Serious Adverse Events occurring from the first investigational medicinal product administration onwards are defined as treatment-emergent Serious Adverse Events. | Visit 3 (Day 0) to day 42 |
| Incidence of AESI | The following AEs are defined as AEs of special interest (AESI), if these events were not already present at Visit 2:
| Visit 2 (day -21 to -1) to Visit 6 (day 42) |
| Local tolerability | The local tolerability of the IMP administration will be evaluated daily throughout the treatment period in the patient diary. Patients should indicate whether they had any discomfort in mouth, throat or esophagus while taking IMP on a VAS (Visual analog scale) ranging from 'no discomfort' to 'the worst imaginable discomfort'. The first assessment on Day 0 recorded in the patient diary after IMP intake will be considered Baseline. | From Baseline (Visit 3) to end of treatment (Visit 5 = 28 days after Visit 3) |
| Patient-reported treatment satisfaction at end of treatment based on questions about handling, taste, and time necessary for administration | Patients will be asked to answer a questionnaire about their satisfaction with the IMP treatment at Visit 5. Questions will cover the general satisfaction with the IMP, the swallowability of the IMP, and the frequency of administration. Answers involve gradings from 0 (the best outcome) to 3 or 4 (the worst outcome) plus an indication for the preference of single or multiple administrations of the investigated product. | At end of treatment (Visit 5 = day 28) |
| Leipzig |
| Germany |
| Otto-von-Guericke-Universität Medizinische Fakultät Universitätsklinikum Magdeburg A. ö. R. | Magdeburg | Germany |
| Klinikum rechts der Isar der TUM | München | Germany |
| Amsterdam University Medical Center | Amsterdam | Netherlands |
| Albert Schweitzer Ziekenhuis | Dordrecht | Netherlands |
| Centrum Medyczne Med-GASTR Sp. z o.o. | Lodz | Poland |
| Centrum Medyczne Sonomed Sp. z o.o. | Szczecin | Poland |
| Hospital Universitario Vall d' Hebrón | Barcelona | Spain |
| Hospital Universitario de La Princesa | Madrid | Spain |
| Hospital Universitario Fundación Jiménez Díaz | Madrid | Spain |
| Hospital Universitario Central De Asturias | Oviedo | Spain |
| Hospital de Navarra | Pamplona | Spain |
| Hospital General de Tomelloso | Tomelloso | Spain |
| Hospital Universitario Rio Hortega | Valladolid | Spain |
| Hospital de Viladecans | Viladecans | Spain |
| Universitätsspital Zürich | Zurich | Switzerland |
| ID | Term |
|---|---|
| D057765 | Eosinophilic Esophagitis |
| ID | Term |
|---|---|
| D004941 | Esophagitis |
| D004935 | Esophageal Diseases |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
| D005759 | Gastroenteritis |
| D004802 | Eosinophilia |
| D007960 | Leukocyte Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D000068656 | Mometasone Furoate |
| ID | Term |
|---|---|
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
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