Zemaira Eosinophilic Esophagitis Pilot Study

• Status: Not yet recruiting
• Trial end date: 2024-09-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a prospective, open-label drug study that will examine the effects of Zemaira (alpha-1 trypsin inhibitor) in patients with Eosinophilic Esophagitis.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborators: CSL Behring; National Institutes of Health (NIH)
• Treatments: Protease Inhibitors

Criteria

Inclusion Criteria:

1. Participant and/or legally authorized representative must be able to understand and provide informed consent prior to study procedures being performed.

2. Willing and able to comply with study visits and activities

3. Age ≥ 18 to ≤ 70 years at study enrollment

4. Histologically active EoE at time of screening or within 12 weeks prior to enrollment, with a peak count of ≥ 15 eosinophils (eos)/high powered field (hpf) in any region of the esophagus, with no other known cause for esophageal eosinophilia; involvement of eosinophilic inflammation in other gastrointestinal segments will be allowed but not required or sufficient.

5. History of moderate to severe symptoms of abdominal/chest pain or dysphagia an average of ≥ 2 episodes per week during the 2 weeks prior to screening.

6. History of approximately 8 week standard of care (SOC) treatment (e.g., proton pump inhibitors (PPI's), topical corticosteroids) that did not adequately control or treat the EoE or documentation that such treatment was not tolerated. Participant may re-screen if this is not met.

7. Stable medical management of EoE (and other eosinophilic disorders, if applicable) including stable dosage of medications in the 8 weeks prior to study enrollment, if applicable. Participants may be on baseline anti-EoE therapy (such as elimination diet, elemental diet, proton pump inhibitors (PPI), topical or systemic glucocorticoids (≤10 milligrams (mg) daily), immunosuppressive agents, cromolyn, and H1 and H2 anti-histamines) as long as there is agreement not to change their dosage.

8. Willing to maintain current dietary regimen throughout the course of the study. Diet must have been stable for 8 weeks prior to baseline endoscopy.

Exclusion Criteria:

1. Inability or unwillingness of a participant to give written informed consent or comply with study protocol.

2. Current active H. pylori infection. A history of H. pylori infection needs to have medical documentation of one of the three acceptable eradication tests: antigen, breath or histology. Participants with current active H. pylori infections can be re-screened for study participation in the future if they are treated and have medical documentation of one of the three acceptable eradication tests: antigen, breath or histology.

3. Another disorder that causes esophageal eosinophilia (e.g., hypereosinophilic syndrome*, Churg Strauss vasculitis, eosinophilic granuloma or a parasitic infection).

*Hypereosinophilic syndrome defined by multiple organ involvement (with the exception of atopic disease or eosinophilic gastrointestinal disorder (EGID)) and persistent blood absolute eosinophil count ≥1500/mcL.

4. Systemic gastrointestinal disorders such as Crohn's disease, inflammatory bowel disease, or Celiac disease not including chronic gastritis, chronic duodenitis, mucosal eosinophilia or other EGID's.

5. Diagnosed with Chronic Obstructive Pulmonary Disease (COPD).

6. Known immunoglobulin A (IgA) deficiency (i.e., IgA level < 8 mg/dL at screening).

7. Current coronavirus disease of 2019 (COVID-19) infection (i.e., detection of the presence of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) before the screening endoscopy using testing done at the clinical site).

8. Hematological disorders that prevent the blood from clotting (e.g., hemophilia, Von Willebrand disease, clotting factor deficiencies).

9. Currently on anti-coagulation medications (except aspirin/NSAIDS).

10. Known history of hypersensitivity following infusions of human blood or blood components (e.g., human immunoglobulins or human albumin).

11. Known history of hypersensitivity or anaphylaxis to Zemaira or other A1AT products.

12. Uncontrolled, or poorly controlled, comorbid conditions including, but not limited to, cardiovascular diseases, hypertension and diabetes as defined by the following criteria:

• A myocardial infarction within the last 6 months.

• Blood pressure > 179/99 mmHg

• Diabetics with a hemoglobin A1C (HbA1C) > 7% and that are deemed to have uncontrolled diabetes as defined by the physician

13. History of cancer: Individuals who have had basal cell carcinoma, localized squamous cell carcinoma of the skin, or in situ carcinoma of the cervix are eligible provided that the participant is in remission and curative therapy was completed at least 12 months prior to the date informed consent was obtained. Individuals who have had other malignancies are eligible provided that the individual is in remission and curative therapy was completed at least 5 years prior to the date informed consent was obtained.

14. Current or expected treatment with sublingual immunotherapy (SLIT) or oral immunotherapy (OIT).

15. Current or recent use of any investigational drug (within 3 months or 5 half-lives, whichever is longer, prior to screening).