Overview

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).