A Study to Investigate the Efficacy and Tolerability of ESO-101 in Patients With Eosinophilic Esophagitis

NCT04849390 | Completed Phase 2

• 2 other identifiers: ACESO2020-000082-16
• Study Type: interventional
• Target: 43
• Locations: 5 countries
• Sites: 17

Brief Summary

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).

Conditions

Eosinophilic Esophagitis

Outcome Measures

Primary Outcomes (1)

• 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)

Secondary Outcomes (18)

• 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

  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

  End of treatment (Visit 5 = day 28)

• Proportion of patients with an improvement in the dysphagia severity score from Baseline to end of treatment

  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

  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

  From Baseline (Visit 2) to end of treatment (Visit 5 = 4-7 weeks after Visit 2)

Study Arms (2)

ESO-101

EXPERIMENTAL

Oral use of 1 hard gelatin capsule (800 μg)

Drug: ESO-101

Placebo

PLACEBO COMPARATOR

Oral use of 1 hard gelatin capsule

Drug: Placebo

Interventions

ESO-101 DRUG

Daily administration in the evening at bedtime for 28 days

Also known as: Mometasone furoate (800 μg)

ESO-101

Placebo DRUG

Daily administration in the evening at bedtime for 28 days

Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult patients aged 18-70 years;
• Confirmed clinicopathological diagnosis of EoE (eosinophilic esophagitis);
• Active and symptomatic EoE, defined as:
• peak eosinophil count ≥15 eosinophils/high-powered field (hpf) at 2 levels of the esophagus at the screening endoscopy (Visit 2) as measured in a total of 6 hpfs derived from 6 biopsies, 2 each from the proximal, mid, and distal segment of the esophagus;
• either a dysphagia or odynophagia severity sore of ≥4 on a 11-point numeric rating scale for ≥1 day during the 7 days before Screening (Visit 1);
• 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:
• combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal);
• progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable);
• intrauterine device or intrauterine hormone-releasing system;
• bilateral tubal occlusion;
• a vasectomized sexual partner;
• sexual abstinence (only accepted as true abstinence when this is in line with the preferred and usual lifestyle of the patient; periodic abstinence \[e.g. calendar, ovulation, symptothermal, post-ovulation methods, and withdrawal\] is not an acceptable method of contraception).

You may not qualify if:

• Women who are pregnant, lactating, possibly pregnant or planning a pregnancy during the trial period;
• Current or past (within the last 3 months) alcohol or drug abuse;
• Initiation of a diet-modifying food restriction within 4 weeks before the screening endoscopy (Visit 2) until EOT (end of treatment);
• Use of systemic corticosteroids or biologic immunomodulators within 3 months before the screening endoscopy (Visit 2) until the EOT;
• History of non-response to treatment of EoE with topical corticosteroid drugs (defined as no improvement of clinical symptoms of EoE after a minimum of 4 weeks corticosteroid therapy used at appropriate doses according to the investigator's judgment) or requirement of cessation of corticosteroid therapy for EoE treatment due to oral candidiasis or systemic corticosteroid side effects;
• Use of corticosteroids for treatment of EoE within 4 weeks before the screening endoscopy (Visit 2) until the EOT;
• Use of inhalable (pulmonary or nasal) corticosteroids within 4 weeks before the screening endoscopy (Visit 2) until the EOT;
• Asthma requiring corticosteroid therapy in the seasonal allergy period according to the investigator's judgment based on anamnesis until the EOT;
• Change in proton pump inhibitor (PPI) dosing regimen within 4 weeks before the screening endoscopy (Visit 2) until the EOT;
• Use of systemic leukotriene receptor antagonists, immunosuppressant therapy, or chronic oral or systemic anticoagulants (such as coumarin derivates, novel oral and subcutaneous anticoagulants) within 2 weeks before Screening (Visit 1) until the EOT;
• Unable to swallow a test tablet of about the size of the IMP capsule used in the trial;
• History of diabetes mellitus;
• Other severe comorbid condition, concurrent medication, or other issue that renders the patient unsuitable to participate in the trial in the judgment of the investigator, including but not limited to: comorbid condition with an estimated life expectancy of ≤12 months, dialysis, severe pulmonary (requiring home oxygen, uncontrolled chronic obstructive pulmonary disease Gold III/IV) or cardiovascular conditions (heart failure New York Heart Association III and IV, uncontrolled hypertension systolic blood pressure by repeated measurement \>180mmHg);
• History of cancer (except non-melanoma skin cancer, or carcinoma in situ of cervix) or treatment with anticancer therapy (chemotherapy, immunotherapy, radiotherapy, hormone therapy for cancer treatment, targeted therapy or gene therapy) within 12 months before Screening (Visit 1) until the EOT;
• Known intolerability or hypersensitivity to mometasone furoate or any of the IMP excipients (e.g. bovine gelatin, polyvinyl alcohol, polyvinyl acetate, glycerol, sorbitol);

Sponsors & Collaborators

• EsoCap AG: lead
• FGK Clinical Research GmbH: collaborator
• FGK Representative Service B.V.: collaborator

Study Sites (17)

Facharztzentrum Eppendorf

Hamburg, Germany

Location

Universitätsklinikum Leipzig AöR

Leipzig, Germany

Location

Otto-von-Guericke-Universität Medizinische Fakultät Universitätsklinikum Magdeburg A. ö. R.

Magdeburg, Germany

Location

Klinikum rechts der Isar der TUM

München, Germany

Location

Amsterdam University Medical Center

Amsterdam, Netherlands

Location

Albert Schweitzer Ziekenhuis

Dordrecht, Netherlands

Location

Centrum Medyczne Med-GASTR Sp. z o.o.

Lodz, Poland

Location

Centrum Medyczne Sonomed Sp. z o.o.

Szczecin, Poland

Location

Hospital Universitario Vall d' Hebrón

Barcelona, Spain

Location

Hospital Universitario de La Princesa

Madrid, Spain

Location

Hospital Universitario Fundación Jiménez Díaz

Madrid, Spain

Location

Hospital Universitario Central De Asturias

Oviedo, Spain

Location

Hospital de Navarra

Pamplona, Spain

Location

Hospital General de Tomelloso

Tomelloso, Spain

Location

Hospital Universitario Rio Hortega

Valladolid, Spain

Location

Hospital de Viladecans

Viladecans, Spain

Location

Universitätsspital Zürich

Zurich, Switzerland

Location

Related Publications (1)

• 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.

  PMID: 39676687 DERIVED

MeSH Terms

Conditions

Eosinophilic Esophagitis

Interventions

Mometasone Furoate

Condition Hierarchy (Ancestors)

Esophagitis; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Gastroenteritis; Eosinophilia; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Isabelle Racamier

  EsoCap AG (Malzgasse 9, 4052 Basel, Switzerland)

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The placebo will be identical to the test product in terms of appearance, constitution of inactive ingredients, packaging, labeling and administration.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2021
• First Posted: April 19, 2021
• Study Start: June 29, 2021
• Primary Completion: October 9, 2023
• Study Completion: October 9, 2023
• Last Updated: November 14, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

NL (2); ES (8); CH (1); DE (4); PL (2)