Trial Outcomes & Findings for Utility of [F-18] fluoroDOPA for Neonatal Hyperinsulinism (NCT NCT00674440)

NCT ID: NCT00674440

Last Updated: 2022-11-09

Results Overview

Comparison of PET scan results with outcome of surgery and histopathology results confirmed as focal or diffuse

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 106 participants
• Primary outcome timeframe: up to 1 month post surgical intervention
• Results posted on: 2022-11-09

Participant Flow

From December 2004 to December 2008, the study team recruited and enrolled 106 subjects referred to the Congenital Hyperinsulinism Center at the Children's Hospital of Philadelphia who were potential candidates for partial pancreatectomy surgery if focal lesions were suspected and were able to be localized.

The participants newly diagnosed with hypersulinism required surgery for hyperinsulinism. A subset of patients who seemed to respond to medical therapy or had continued hyperinsulinism after surgery were also included. All subjects were consented for PET scan and were eligible for surgery. 105 patients underwent F-DOPA PET with the same research procedures.

Participant milestones

Measure	All Subjects Consented There were 106 subjects consented to participate. 105 actually underwent PET scan.
Overall Study STARTED	106
Overall Study COMPLETED	95
Overall Study NOT COMPLETED	11

Reasons for withdrawal

Measure	All Subjects Consented There were 106 subjects consented to participate. 105 actually underwent PET scan.
Overall Study consented but not scanned (Withdrawn due to genetics)	1
Overall Study did not have surgery, so unevaluable	7
Overall Study had atypical histology or had second PET scan after initial surgery - not counted twice	3

Baseline Characteristics

Utility of [F-18] fluoroDOPA for Neonatal Hyperinsulinism

Baseline characteristics by cohort

Measure	Subjects Who Consented n=106 Participants These demographics include all subject recruited who consented whether they are evaluable or not
Age, Categorical <=18 years	106 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	53 Participants n=5 Participants
Sex: Female, Male Male	53 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	19 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	87 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	4 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	6 Participants n=5 Participants
Race (NIH/OMB) White	73 Participants n=5 Participants
Race (NIH/OMB) More than one race	23 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	94 Participants n=5 Participants
Region of Enrollment Canada	7 Participants n=5 Participants
Region of Enrollment Israel	1 Participants n=5 Participants
Region of Enrollment Brazil	1 Participants n=5 Participants
Region of Enrollment Curacao	1 Participants n=5 Participants
Region of Enrollment Iran	1 Participants n=5 Participants
Region of Enrollment Saudi Arabia	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to 1 month post surgical intervention

Population: An F-DOPA PET/CT imaging study was performed and reported prior to surgery. This data includes surgical histopathology confirmed as focal or diffuse. 95 of the 106 original consented participants had surgical results that were evaluable. (1 consented but never scanned, 7 did not have surgery, 3 had surgery but were excluded for atypical histology that did not fit classic focal or diffuse or had second PET scan after initial surgery therefore not counted twice).

Comparison of PET scan results with outcome of surgery and histopathology results confirmed as focal or diffuse

Outcome measures

Measure	Subjects Who Had PET and Surgery n=95 Participants To determine the sensitivity and specificity of 18F-DOPA PET/CT for the detection of a focal pancreatic lesion in infants and children with poorly controlled hyperinsulinemic hypoglycemia.
The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism True Negatives (PET diffuse/surgery diffuse)	40 Participants
The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism False negatives (PET diffuse/surgery focal):	6 Participants
The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism False positives (PET focal/surgery diffuse):	3 Participants
The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism True positives (PET focal/surgery focal):	46 Participants

SECONDARY outcome

Timeframe: 72 hours maximum or prior to pancreatic surgery

Number of Participants with Adverse Events

Outcome measures

Measure	Subjects Who Had PET and Surgery n=105 Participants To determine the sensitivity and specificity of 18F-DOPA PET/CT for the detection of a focal pancreatic lesion in infants and children with poorly controlled hyperinsulinemic hypoglycemia.
Safety of F-DOPA PET	0 Participants

Adverse Events

All Subjects Who Had PET

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

Lisa J. States, MD

Children's Hospital of Philadelphia/Perelman School of Medicine, University of Pennsylvania

Phone: 267-425-7146

Email: states@chop.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place