Zemaira Eosinophilic Esophagitis Pilot Study

NCT05485155 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 2021-0625
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Eosinophilic Esophagitis

Outcome Measures

Primary Outcomes (2)

• Change in esophageal alpha1 anti-trypsin (A1AT) concentration

  Absolute change from baseline A1AT esophageal concentration in participants receiving Zemaira to 24 hours after the last infusion at 4 weeks

  4 weeks

• Adverse events

  The number of Adverse Events (AE), including Serious Adverse Events (SAE), related to the study drug.

  20 weeks

Secondary Outcomes (1)

• Change in serine protease activity

  4 weeks

Study Arms (1)

Active Drug

EXPERIMENTAL

Zemaira alpha-proteinase inhibitor

Drug: Alpha-proteinase inhibitor

Interventions

Alpha-proteinase inhibitor DRUG

Intravenous infusion at 120 mg/kg body weight dose/week for 4 weeks for a total of 4 infusions.

Also known as: Zemaira

Active Drug

Eligibility Criteria

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

You may qualify if:

• Participant and/or legally authorized representative must be able to understand and provide informed consent prior to study procedures being performed.
• Willing and able to comply with study visits and activities
• Age ≥ 18 to ≤ 70 years at study enrollment
• Histologically active eosinophilic esophagitis (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.
• 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.
• 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.
• Willing to maintain current dietary regimen throughout the course of the study. Diet must have been stable for 8 weeks prior to baseline endoscopy.

You may not qualify if:

• Inability or unwillingness of a participant to give written informed consent or comply with study protocol.
• 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.
• 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/microliter.
• 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.
• Diagnosed with Chronic Obstructive Pulmonary Disease (COPD).
• Known immunoglobulin A (IgA) deficiency (i.e., IgA level \< 8 mg/dL at screening).
• 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).
• Hematological disorders that prevent the blood from clotting (e.g., hemophilia, Von Willebrand disease, clotting factor deficiencies).
• Currently on anti-coagulation medications (except aspirin/Non-steroidal anti-inflammatory drugs).
• Known history of hypersensitivity following infusions of human blood or blood components (e.g., human immunoglobulins or human albumin).
• Known history of hypersensitivity or anaphylaxis to Zemaira or other A1AT products.
• 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

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• CSL Behring: collaborator
• National Institutes of Health (NIH): collaborator

Study Sites (2)

The National Institutes of Health

Bethesda, Maryland, 20892, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

MeSH Terms

Conditions

Eosinophilic Esophagitis

Interventions

alpha 1-Antitrypsin

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)

Glycoproteins; Glycoconjugates; Carbohydrates; Serpins; Peptides; Amino Acids, Peptides, and Proteins; Acute-Phase Proteins; Blood Proteins; Proteins; Alpha-Globulins; Serum Globulins; Globulins

Study Officials

• Marc Rothenberg

  Cincinnati Children's Hospital Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Regina Yearout

CONTACT

(513) 517-2108; Regina.Yearout@cchmc.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2022
• First Posted: August 3, 2022
• Study Start: May 15, 2025
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): January 1, 2027
• Last Updated: December 17, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)