Novel Blood Test to Predict Safe Foods for Infants and Toddlers With Food Protein-induced Enterocolitis Syndrome (FPIES)

A Novel Blood Test to Predict Safe (Non-trigger) Foods for Infants and Toddlers With Food Protein-induced Enterocolitis Syndrome (FPIES)

The aim of this study is to validate a blood test that can identify safe foods for food protein-induced enterocolitis syndrome (FPIES). This study proposes a solution to the problems of FPIES by developing a new blood assay that screens a large number of foods (more than 20) in a culture plate. If this blood test is successful it may be able to identify safe foods more quickly.

The study will recruit ten participants that will have 2-3 trigger foods as well as ten participants with 4 or more trigger foods.

Study Overview

• Status: Enrolling by invitation
• Conditions: Pediatric Disorder; Allergies; Food Protein-Induced Enterocolitis Syndrome
• Intervention / Treatment: Diagnostic test: Blood test assay

Detailed Description

Participants will complete surveys and have blood draws during the study. Additionally, participants will be asked to keep track of their diet as well as introduce safe foods identified by the researchers.

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 7 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of FPIES
• Have to have documented reactions to 2-3 trigger foods with recurrent delayed vomiting or documented reactions to 4 or more trigger foods with recurrent delayed vomiting.

Exclusion Criteria:

• Patients who are currently on medications that suppress the immune system
• Patients who do not have at least 2 trigger foods identified.
• Patients who have a history of an organic Gastrointestinal (GI) disease (e.g., inflammatory bowel disease, celiac disease, biliary disorders, bowel resection), cardiac, pulmonary, neurologic, renal, endocrine, or gynecological pathology
• Lack of parental or guardian informed consent.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: DIAGNOSTIC
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Blood test with assays; Ten patients with FPIES exhibiting reactions to 2-3 foods, and up to 10 exhibiting FPIES reactions to 4 or more foods will be recruited.	Diagnostic test: Blood test assay; Participants will have their blood drawn and be evaluated with a new blood assay that screens a large number of foods (more than 20) in a culture plate. Participants will be asked to eat the identified safe foods by the the blood assay.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Negative predictive value (NPV), defined as the proportion of test-predicted safe foods that are actually safe foods.	The study team will build a Receiver operating characteristic (ROC) curve to estimate the NPV. A random-effects logit model will be used to model the binary outcome (safe or trigger food) as a function of the 9 biomarker measurements in the assay. The random effect in the logit model will take into consideration of the correlated data measured within the same subject. A cluster Receiver operating characteristic (ROC) curve analysis will be used to assess the precision of the assay. Specifically, the study team will compute the area under the cluster ROC curve (AUC), along with a 95% confidence interval (CI). The assay is predictive if the lower limit of the 95% CI is above 0.5, which is the null value indicating no predictive ability. Given the assay is predictive, the team will select a threshold to obtain the NPV.	Up to 10 weeks (from first food trial)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Positive predictive value (PPV), defined as the proportion of test-predicted unsafe foods that are actually unsafe foods.	The study team will use the same ROC curve described for the primary outcome to derive the PPV.	Up to 10 weeks (from first food trial)

Collaborators and Investigators

• Sponsor: University of Michigan
• Investigators: Principal Investigator: Mohamad El Zaatari, PhD, University of Michigan

Study record dates

Study Major Dates

• Study Start (ACTUAL): October 8, 2019
• Primary Completion (ANTICIPATED): December 1, 2023
• Study Completion (ANTICIPATED): December 1, 2023

Study Registration Dates

• First Submitted: November 19, 2020
• First Submitted That Met QC Criteria: November 19, 2020
• First Posted (ACTUAL): November 25, 2020

Study Record Updates

• Last Update Posted (ACTUAL): November 9, 2022
• Last Update Submitted That Met QC Criteria: November 7, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Keywords: Vomiting; Trigger foods; Safe foods
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Disease; Gastrointestinal Diseases; Gastroenteritis; Intestinal Diseases; Syndrome; Enterocolitis
• Other Study ID Numbers: HUM00156027

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No