Trial Outcomes & Findings for Novel Blood Test to Predict Safe Foods for Infants and Toddlers With Food Protein-induced Enterocolitis Syndrome (FPIES) (NCT NCT04644783)

Last Updated: 2024-05-16

Results Overview

A Receiver operating characteristic (ROC) curve was built to estimate the NPV. A random-effects logit model was used to model the binary outcome (safe or trigger food) as a function of the 9 biomarker measurements in the assay. (Expression Value = Relative fold change of 9-gene expression panel in response to food treatment, divided by fold change in response to LPS treatment, multiplied by 1000). The random effect in the logit model took into consideration the correlated data measured within the same subject. A cluster ROC curve analysis was used to assess the precision of the assay. Specifically, the area was computed under the cluster ROC curve (AUC). A threshold to obtain the NPV was selected based on inspection of the ROC curve. At the initial visit, participants had their blood drawn, which was assayed. On average, participants came in up to 4 weeks later after the test results were ready. Participants were then asked to trial a new food each week for up to 7 weeks.

Recruitment status

COMPLETED

Study phase

Target enrollment

10 participants

Primary outcome timeframe

Up to 7 weeks from the first blood trial, on average 11 weeks

Results posted on

2024-05-16

Participant Flow

Participant milestones

Participant milestones
Measure	Blood Test With Assays 10 participants with FPIES exhibiting reactions to more than 2 foods were recruited. Blood test assay: Participants had their blood drawn and evaluated with a new blood assay that screened a large number of foods (more than 20) in a culture plate. Participants were asked to eat the foods identified as safe by the blood assay.
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Blood Test With Assays n=10 Participants 10 participants with FPIES exhibiting reactions to more than 2 foods were recruited. Blood test assay: Participants had their blood drawn and evaluated with a new blood assay that screened a large number of foods (more than 20) in a culture plate. Participants were asked to eat the foods identified as safe by the the blood assay.
Age, Continuous	0.75 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	10 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 7 weeks from the first blood trial, on average 11 weeks

Outcome measures

Outcome measures
Measure	Blood Test With Assays n=10 Participants 10 participants with FPIES exhibiting reactions to more than 2 foods were recruited. Blood test assay: Participants had their blood drawn and evaluated with a new blood assay that screened a large number of foods (more than 20) in a culture plate. Participants were asked to eat the foods identified as safe by the the blood assay.
Negative Predictive Value (NPV), Defined as the Percentage of Test-predicted Safe Foods That Are Actually Safe Foods. NPV <29.24 Threshold	98.5 percentage
Negative Predictive Value (NPV), Defined as the Percentage of Test-predicted Safe Foods That Are Actually Safe Foods. Area Under the ROC Curve	73.16 percentage

SECONDARY outcome

Timeframe: Up to 7 weeks from the first blood trial, on average 11 weeks

The same ROC curve described for the primary outcome was used to derive the PPV. At the initial visit, participants had their blood drawn, which was assayed. On average, participants came in up to 4 weeks later after the test results were ready. Participants were then asked to trial a new food each week for up to 7 weeks.

Outcome measures

Outcome measures
Measure	Blood Test With Assays n=10 Participants 10 participants with FPIES exhibiting reactions to more than 2 foods were recruited. Blood test assay: Participants had their blood drawn and evaluated with a new blood assay that screened a large number of foods (more than 20) in a culture plate. Participants were asked to eat the foods identified as safe by the the blood assay.
Positive Predictive Value (PPV), Defined as the Percentage of Test-predicted Unsafe Foods That Are Actually Unsafe Foods.	31.1 percentage

Adverse Events

Blood Test With Assays

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Mohamad El Zaatari

University of Michigan

Phone: 734-647-2954

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place