Immunologic Basis of Food Protein-Induced Enterocolitis Syndrome

NCT06683521 | Recruiting DMC

• 2 other identifiers: 23-01346U01AI170836-01
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Food Protein-Induced Enterocolitis Syndrome

Keywords

Allergy; FPIES; Food protein-induced enterocolitis syndrome; Food; Diagnosis

Outcome Measures

Primary Outcomes (1)

• The proportion of individuals with active FPIES who develop FPIES symptoms during the optimal Low Dose multi-day OFC (Days 1-7)

  Up to Day 7

Secondary Outcomes (10)

• Percentage of participants who meet criteria for intravenous fluid (IVF) resuscitation among those who develop FPIES symptoms during Low Dose multi-day OFC (Days 1-7)[Time Frame: Up to Day 7]

  Up to Day 7

• Percentage of participants treated with oral ondansetron among those who develop FPIES symptoms during Low Dose multi-day OFC

  Up to Day 7

• Percentage of participants treated with parenteral ondansetron among those who develop FPIES symptoms during Low Dose multi-day OFC

  Up to Day 7

• Percentage of participants with hypotension among those who develop FPIES symptoms during Low Dose multi-day OFC (Days 1-7)

  Up to Day 7

• Percentage of participants with reactions treated in the Emergency Department (ED) among those who develop FPIES symptoms during Low Dose multi-day OFC (Days 1-7)

  Up to Day 7

Study Arms (3)

Cohort 1

EXPERIMENTAL

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.

Other: Supervised Low-Dose Oral Food Challenge (OFC); Other: At-Home Low-Dose Oral Food Challenge (OFC); Other: Supervised High-Dose Oral Food Challenge (OFC)

Cohort 2

EXPERIMENTAL

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.

Other: Supervised Low-Dose Oral Food Challenge (OFC); Other: At-Home Low-Dose Oral Food Challenge (OFC); Other: Supervised High-Dose Oral Food Challenge (OFC)

Cohort 3

EXPERIMENTAL

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.

Other: Supervised Low-Dose Oral Food Challenge (OFC); Other: At-Home Low-Dose Oral Food Challenge (OFC); Other: Supervised High-Dose Oral Food Challenge (OFC)

Interventions

Supervised Low-Dose Oral Food Challenge (OFC) OTHER

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

Cohort 1; Cohort 2; Cohort 3

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

Cohort 1; Cohort 2; Cohort 3

Supervised High-Dose Oral Food Challenge (OFC) 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 1; Cohort 2; Cohort 3

Eligibility Criteria

• Age: 1 Year - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• NYU Langone Health: lead
• National Institute of Allergy and Infectious Diseases (NIAID): collaborator

Study Sites (2)

NYU Langone Health

New York, New York, 10016, United States

RECRUITING

Jaffe Food Allergy Institute at Mount Sinai

New York, New York, 10029, United States

RECRUITING

MeSH Terms

Conditions

Hypersensitivity; Disease

Condition Hierarchy (Ancestors)

Immune System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Anna Nowak-Wegrzyn, MD, PhD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Anna Nowak-Wegrzyn, MD, PhD

CONTACT

212-263-5940; Anna.Nowak-wegrzyn@nyulangone.org

Joseline Cruz Vazquez, MPH

CONTACT

347-213-8701; Joseline.CruzVazquez@nyulangone.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2024
• First Posted: November 12, 2024
• Study Start: February 24, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): January 1, 2028
• Last Updated: June 22, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: No end date.
• Access Criteria: Upon reasonable request, the investigator who proposed to use the data will be able to access via ImmPort.

Locations

US (2)