Eosinophilic EsophagitisPhase 2ZEEPSNCT05485155
Zemaira Eosinophilic Esophagitis Pilot Study
A Phase 2 study testing Alpha-proteinase inhibitor in people with Eosinophilic Esophagitis, run by Children's Hospital Medical Center, Cincinnati at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Eosinophilic Esophagitis
- What is being tested?
- Alpha-proteinase inhibitor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesUnited States
- Study dates
- 15 May 2025to Jan 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
Show all 47Show fewer
- 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
- Diabetics with a hemoglobin A1C (HbA1C) \> 7% and that are deemed to have uncontrolled diabetes as defined by the physician
- 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.
- Current or recent use of any investigational drug (within 3 months or 5 half-lives, whichever is longer, prior to screening).
- Currently participating or planning to participate in another clinical study involving an investigational drug during the course of this study.
- Presence of steroid-responsive diseases (e.g., asthma) with a recent (within the last 6 months) history of disease exacerbations requiring steroid treatment.
- Current use of systemic steroids with daily dose \> 10 mg for any reason or steroid burst for \> 3 days within 1 month of screening.
- Current pregnancy or breastfeeding.
- Any esophageal stricture unable to be passed with a standard, diagnostic upper endoscope.
- Esophageal varices or interventional treatment for esophageal varices that, in the opinion of the investigator, would put the participant at undue risk for significant complications from an endoscopy procedure.
- History of alcohol or drug abuse within 6 months prior to screening.
- Participant or his/her immediate family is a member of the investigational team.
- Presence of clinically significant laboratory abnormalities at the screening that meets one or more of the following criteria:
- Serum alanine aminotransferase (ALT) ≥ 3 times upper limit of normal (ULN)
- Serum total bilirubin ≥2 times ULN
- Absolute neutrophil count (ANC) ≤ 1000 cells/mm3
- Hemoglobin (Hgb) ≤ 10.0 g/dL
- Platelet count ≤ 100,000/mm3
- Glomerular Filtration Rate (GFR) ≤ 44 mL/min/1.73 m2
- Planned or anticipated major surgical procedure during the study.
- Known or suspected immunodeficiency disorder, including human immunodeficiency disorder (HIV) or a history of invasive opportunistic infections (e.g., tuberculosis, non-tuberculous mycobacterial infections, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystosis, aspergillosis, or an infectious etiology of esophagitis (e.g. cytomegalovirus or Candida) despite infection resolution or otherwise recurrent infections of abnormal frequency or prolonged infections suggesting an immune-compromised status as judged by the investigator.
- Planned or anticipated use of any prohibited medications and procedures during the study.
- Currently on Zemaira for other indications.
- Initiation, discontinuation or change in the dosage regimen of the following medications within 8 weeks prior to the baseline endoscopy:
- Proton pump inhibitors Leukotriene inhibitors Nasal and/or inhaled corticosteroids Participants on a stable dose of these medications for at least 8 weeks prior to the baseline endoscopy may be included in the study. PPI and leukotriene inhibitor dosing must not change during the study, but nasal and/or inhaled corticosteroids can be increased if there is a deterioration in the condition for which the medications are prescribed.
- Presence of the following esophageal disorders: achalasia cardia, Barrett's esophagus or precancerous lesions noted on the endoscopy.
- Women of childbearing potential, or male participants with female partners of childbearing potential, who are unwilling to practice highly effective contraception prior to the initial dose/start of the first treatment, during the study, and for at least 12 weeks after the last dose. Highly effective contraceptive measures include:
- Stable use of combined (estrogen and progestogen containing) hormonal contraception (oral, intravaginal, transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) for one month prior to screening
- Intrauterine device; intrauterine hormone-releasing system
- Bilateral tubal ligation
- Vasectomized partner
- And/or sexual abstinence
- Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
The National Institutes of Health
Bethesda, Maryland, 20892
Perla Adames-Castillo, RN, perla.adamescastillo@nih.gov
Gregory Constantine, MD
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Regina Yearout, 513-517-2108, Regina.Yearout@cchmc.org
Marc E Rothenberg, MD, PhD
About this study
Official title: An Open-Label Study of Zemaira (Alpha 1-Trypsin Inhibitor) in Subjects With Eosinophilic Esophagitis
This is a prospective, open-label drug study that will examine the effects of Zemaira (alpha-1 trypsin inhibitor) in patients with Eosinophilic Esophagitis.
Read the detailed description
This is a phase II, open-label trial of Zemaira in participants diagnosed with eosinophilic esophagitis. Potential participants will be screened during a 12-week screening period. Participants will be enrolled based on the presence of active disease and their ability to meet the study inclusion and exclusion criteria. Qualifying participants will receive weekly intravenous infusions of 120 mg/kg body weight dose/week for 4 weeks (for a total of 4 infusions). During the treatment period, participants will be monitored for adverse events/reactions and will complete patient reported outcome metrics to track their symptoms and general wellbeing. Final assessments will be performed 24 hours after the last dose of the study drug. All participants will be followed for an additional 12 weeks after the last dose of study drug.
- Conditions
- Eosinophilic Esophagitis
- Being tested
- Alpha-proteinase inhibitor
- Medical subject headings
- Eosinophilic Esophagitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alpha-proteinase inhibitorDrug
Also called Zemaira
Intravenous infusion at 120 mg/kg body weight dose/week for 4 weeks for a total of 4 infusions.
| Study group | Role | What they receive |
|---|---|---|
| Active Drug | Experimental | Zemaira alpha-proteinase inhibitor Receives: Drug: Alpha-proteinase inhibitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 4 weeks
Adverse events
The number of Adverse Events (AE), including Serious Adverse Events (SAE), related to the study drug.
Measured: 20 weeks
Secondary outcomes (1)
Change in serine protease activity
Absolute change from baseline serine protease activity in participants receiving Zemaira to the end of treatment visit at 4 weeks as determined by protease activity testing.
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2022
- Study start15 May 2025
- Primary completionOct 2026 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Eosinophilic EsophagitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- CSL Behring, National Institutes of Health (NIH)
- Responsible party
- Sponsor
- Investigators
- Marc Rothenberg, Principal investigator, Cincinnati Children's Hospital Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05485155
- Sponsor's protocol ID
- 2021-0625
- Acronym
- ZEEPS
- Status last verified
- May 2025
- First submitted
- 1 Aug 2022
- Last updated
- 17 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study of Zemaira (Alpha 1-Trypsin Inhibitor) in Subjects With Eosinophilic Esophagitis
- ClinicalTrials.gov ID
- NCT05485155
- Acronym / sponsor ID
- ZEEPS / 2021-0625
- Conditions
- Eosinophilic Esophagitis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- CSL Behring, National Institutes of Health (NIH)
- Study start
- 15 May 2025 (actual)
- Primary completion
- Oct 2026 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 3 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a prospective, open-label drug study that will examine the effects of Zemaira (alpha-1 trypsin inhibitor) in patients with Eosinophilic Esophagitis.
Detailed description
This is a phase II, open-label trial of Zemaira in participants diagnosed with eosinophilic esophagitis. Potential participants will be screened during a 12-week screening period. Participants will be enrolled based on the presence of active disease and their ability to meet the study inclusion and exclusion criteria. Qualifying participants will receive weekly intravenous infusions of 120 mg/kg body weight dose/week for 4 weeks (for a total of 4 infusions). During the treatment period, participants will be monitored for adverse events/reactions and will complete patient reported outcome metrics to track their symptoms and general wellbeing. Final assessments will be performed 24 hours after the last dose of the study drug. All participants will be followed for an additional 12 weeks after the last dose of study drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Active Drug | Experimental | Drug: Alpha-proteinase inhibitorZemaira alpha-proteinase inhibitor |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (47 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Alpha-proteinase inhibitorAlso: ZemairaIntravenous infusion at 120 mg/kg body weight dose/week for 4 weeks for a total of 4 infusions. | Drug | Active Drug |
Primary outcome
Change in esophageal alpha1 anti-trypsin (A1AT) concentration
Time frame: 4 weeks
Absolute change from baseline A1AT esophageal concentration in participants receiving Zemaira to 24 hours after the last infusion at 4 weeks
Adverse events
Time frame: 20 weeks
The number of Adverse Events (AE), including Serious Adverse Events (SAE), related to the study drug.
1 secondary outcome
Change in serine protease activity
Time frame: 4 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Marc Rothenberg principal investigator
Cincinnati Children's Hospital Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Alpha-proteinase inhibitor in people with Eosinophilic Esophagitis, run by Children's Hospital Medical Center, Cincinnati at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 47 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Regina Yearout ((513) 517-2108, Regina.Yearout@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in esophageal alpha1 anti-trypsin (A1AT) concentration (time frame: 4 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Dec 2025.