Immunologic Basis of Food Protein-Induced Enterocolitis Syndrome

NCT ID: NCT06683521

Last Updated: 2025-06-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

72 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-02-24

Study Completion Date

2028-01-31

Brief Summary

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This is a phase I multicenter clinical trial that aims to find the optimal dose for conducting a novel low-dose, multi-day oral food challenge (OFC) protocol for diagnosing food protein-induced enterocolitis syndrome (FPIES). Individuals ages 1-60 years with a history of suspected or confirmed FPIES will be eligible for enrollment. Recruitment is expected to occur over 3 years.

Detailed Description

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At the time of enrollment, participants will undergo screening (Visit 0) and complete a baseline gastrointestinal symptom diary.

At Visit 1 (Day 1), participants will undergo a supervised Low Dose OFC to their FPIES trigger. Those who react will be considered low-threshold reactors and recommended to continue strict avoidance of their FPIES trigger.

For participants who do not react at the Day 1 Low Dose OFC, continued participation involves a daily home challenge on Days 2-7. Participants will be provided with a diary to monitor symptoms. If objective or persistent subjective symptoms develop, participants will be advised to contact the study team before consuming any further doses.

Participants will return for Visit 2 on Day 8. Those who reported symptoms that led to discontinuation of the home challenge will undergo biospecimen collection. Those who tolerated the Day 1 supervised and Days 2-7 home challenges will undergo a High Dose OFC to their suspected FPIES trigger. Those who tolerate this challenge will be considered to have outgrown their FPIES and instructed to introduce the food regularly at home. Those who react will be considered high-threshold reactors and advised to continue strict avoidance of their FPIES trigger and continue routine allergy care.

Conditions

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Food Protein-Induced Enterocolitis Syndrome

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

DIAGNOSTIC

Blinding Strategy

NONE

Study Groups

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Cohort 1

Participants will be sequentially enrolled into 3 cohorts of 24 each.

The first cohort will undergo Low Dose OFC to 300 mg food protein. Following interim analysis of Low Dose OFC outcomes in cohort 1, the Low Dose OFC serving size may be amended or continued at 300 mg food protein for cohort 2 \& 3 based on pre-specified criteria.

Group Type EXPERIMENTAL

Supervised Low-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

All participants will undergo a supervised Low Dose OFC (300 mg food protein or amended) on Day 1.

At-Home Low-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

Participants who tolerate the Day 1 Low Dose OFC will undergo a daily Low-Dose OFC (300 mg food protein or amended) at home on Days 2-7.

Supervised High-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

Participants who tolerate the Days 1-7 Low Dose OFC will undergo a supervised High Dose OFC (maximum 3000 mg food protein) on Day 8.

Cohort 2

Participants will be sequentially enrolled into 3 cohorts of 24 each.

Following interim analysis of Low Dose OFC outcomes in cohort 1, Low-Dose OFC may be continued at 300 mg food protein or be amended for cohort 2 and 3 based on pre-specified criteria.

Group Type EXPERIMENTAL

Supervised Low-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

All participants will undergo a supervised Low Dose OFC (300 mg food protein or amended) on Day 1.

At-Home Low-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

Participants who tolerate the Day 1 Low Dose OFC will undergo a daily Low-Dose OFC (300 mg food protein or amended) at home on Days 2-7.

Supervised High-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

Participants who tolerate the Days 1-7 Low Dose OFC will undergo a supervised High Dose OFC (maximum 3000 mg food protein) on Day 8.

Cohort 3

Participants will be sequentially enrolled into 3 cohorts of 24 each.

Following interim analysis of Low Dose OFC outcomes in cohort 1, Low-Dose OFC may be continued at 300 mg food protein or be amended for cohort 2 and 3 based on pre-specified criteria.

Group Type EXPERIMENTAL

Supervised Low-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

All participants will undergo a supervised Low Dose OFC (300 mg food protein or amended) on Day 1.

At-Home Low-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

Participants who tolerate the Day 1 Low Dose OFC will undergo a daily Low-Dose OFC (300 mg food protein or amended) at home on Days 2-7.

Supervised High-Dose Oral Food Challenge (OFC)

Intervention Type OTHER

Participants who tolerate the Days 1-7 Low Dose OFC will undergo a supervised High Dose OFC (maximum 3000 mg food protein) on Day 8.

Interventions

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Supervised Low-Dose Oral Food Challenge (OFC)

All participants will undergo a supervised Low Dose OFC (300 mg food protein or amended) on Day 1.

Intervention Type OTHER

At-Home Low-Dose Oral Food Challenge (OFC)

Participants who tolerate the Day 1 Low Dose OFC will undergo a daily Low-Dose OFC (300 mg food protein or amended) at home on Days 2-7.

Intervention Type OTHER

Supervised High-Dose Oral Food Challenge (OFC)

Participants who tolerate the Days 1-7 Low Dose OFC will undergo a supervised High Dose OFC (maximum 3000 mg food protein) on Day 8.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Provision of appropriate consent and/or assent
* Age 1-60 years
* Suspected or confirmed FPIES diagnosis
* Reported convincing FPIES reaction (per criteria in 2017 FPIES guidelines) within: (Children \<18 years of age: The past 6-36 months) (Adults age \>18 years: The past 6 months-10 years)
* Individuals of childbearing potential practicing sexual abstinence or using effective methods of contraception during study participation
* English-speaking

Exclusion Criteria

* Past severe FPIES defined as hospitalization due to an acute FPIES reaction with neurological compromise or requiring life support
* Acute FPIES reaction in the past 6 months
* Frequent gastrointestinal symptoms: nausea, abdominal pain, reflux, heartburn, emesis, diarrhea, constipation per participant or guardian report or as evidenced by FPIES Symptoms Score (FPIES-SS)
* Current active eosinophilic gastrointestinal disorders, inflammatory bowel disease, gastroesophageal reflux disease, or any other chronic gastrointestinal condition
* Poorly controlled atopic dermatitis at screening per PI discretion
* Poorly controlled or severe asthma/wheezing at screening, defined by at least one of the following criteria: 1: History of two or more systemic corticosteroid courses within six months of screening or one course of systemic corticosteroids within three months of screening to treat asthma/wheezing; 2: Prior intubation/mechanical ventilation for asthma/wheezing; 3: One hospitalization or ED visit for asthma/wheezing within six months of screening; and 4: Inhaled corticosteroid (ICS) dosing of \>500 mcg daily fluticasone (or equivalent ICS based on National Heart, Lung, and Blood Institute (NHLBI) dosing chart).
* IgE-mediated food allergies where the trigger has not been identified
* Inability to discontinue prohibited medications for 7 days prior to the screening visit and lasting for the duration of study participation unless indicated for use as rescue medication
* Personal or family history of prolonged QT syndrome
* Personal history of arrhythmia
* Current diagnosis of arterial hypertension
* Current diagnosis of cardiovascular disease
* Current diagnosis of any chronic autoimmune disease
* Current diagnosis of liver disease
* Primary or secondary immunodeficiency
* Phenylketonuria (PKU) (ondansetron tablets may contain phenylalanine)
* Use of systemic steroids within 30 days of screening
* Chemotherapy, radiotherapy, or any systemic immunosuppressive drugs in the past 12 months
* Use of biologic drugs or allergen-specific immunotherapy by any route in the past 12 months
* Inability to defer routine immunizations or passive immunization with immune globulin for the duration of participation in the study
* Allergy to any of the following medications: ondansetron (Zofran), dolasetron (Azemet), granisetron (Kytril), or palonosetron (Aloxi)
* Pregnancy or breastfeeding
* Current or past medical problems or findings from the physical examination or screening evaluation not listed above, which, in the opinion of the investigator(s), may pose additional risks from study participation, interfere with the participant's ability to comply with study requirements, and/or impact the quality or interpretation of the data obtained from the study
Minimum Eligible Age

1 Year

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Allergy and Infectious Diseases (NIAID)

NIH

Sponsor Role collaborator

NYU Langone Health

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Anna Nowak-Wegrzyn, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

NYU Langone Health

Locations

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NYU Langone Health

New York, New York, United States

Site Status RECRUITING

Jaffe Food Allergy Institute at Mount Sinai

New York, New York, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Anna Nowak-Wegrzyn, MD, PhD

Role: CONTACT

212-263-5940

Joseline Cruz Vazquez, MPH

Role: CONTACT

347-213-8701

Other Identifiers

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U01AI170836-01

Identifier Type: NIH

Identifier Source: secondary_id

View Link

23-01346

Identifier Type: -

Identifier Source: org_study_id

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