Congenital hyperinsulinism MedDRA version: 18.0 Level: PT Classification code 10061211 Term: Hyperinsulinism System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Sponsors
European Commission
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Biochemically and clinically proven endogenous congenital hyperinsulinism: - Unresponsive to medical treatment (diazoxide) - Indication for 18F-DOPA PET/CT based on mutation analysis • Standard imaging (18F-DOPA PET/CT) not older than 8 weeks • =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Genetically proven diffuse CHI (presenting with a homozygous or compound heterozygous ABCC8/KCNJ11 mutation) • Calculated creatinine clearance below 40 ml/min • Evidence of other malignancy than insulin producing tumors in conventional imaging (suspicious liver, bone and lung lesions based on CT) • Age > 16 years • No signed informed consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main study aim is the in vivo and ex vivo investigation of the expression and distribution of the GLP-1R in the pancreas of children (< 16 years) with proven endogenous congenital hyperinsulinism who qualify for surgery based on response to medical treatment and genetic analysis (only patients with genetically proven diffuse CHI will be excluded). 68Ga-NODAGA-exendin-4 PET/CT imaging data will be compared with autoradiography and histology performed on specimens collected during surgery. ;Secondary Objective: • Comparing 68Ga-NODAGA-exendin-4 PET/CT and 18F-DOPA PET/CT in order to determine the sensitivity of the new imaging method. • Analyzing kinetics of radiotracer uptake in the pancreas of CHI patients by dynamic PET scans in 5 patients. • Determining the lowest activity dose of 68Ga-NODAGA-exendin 4 needed for accurate imaging. • Performing dosimetric studies to determine the radiation dose received by children injected with this calculated minimum dose of 68Ga-NODAGA-exendin 4 based on whole-body PET/CT scans performed in 2 patients. • Assessing the safety (side effects) of 68Ga-NODAGA-exendin 4 as compared to 18F-DOPA • Calculating and comparing the interobserver variability of 68Ga-NODAGA-exendin 4 PET/CT and 18F-DOPA PET/CT • Evaluating the clinical outcome parameters (laboratory parameters (glucose), dosage of medical treatment) after surgery ;Primary end point(s): The expression and distribution of the GLP-1R in the pancreas of children (<16 years) with proven endogenous congenital hyperinsulinism by comparison of 68Ga-NODAGA-exendin 4 PET/CT imaging data with autoradiography and histology performed on specimens collected during surgery;Timepoint(s) of evaluation of this end point: After collection of data from each patient and at the end of the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Comparison of the sensitivity of 68Ga-NODAGA-exendin 4 PET/Ct and 18F-DOPA PET/CT for the pre-operative localization of focal CHI and the discrimination between focal and diffuse CHI. • Analysis of the kinetics of radiotracer uptake in the pancreas of CHI patients. • Determination of the minimal injected dose of 68Ga-NODAGA-exendin 4 needed for accurate imaging. • Determination of the effective radiation dose received by children injected with the calculated minimum dose of 68Ga-NODAGA-exendin 4. • Assessment of the safety (side effects) of 68Ga-NODAGA-exendin 4 as compared to 18F-DOPA. • Calculation and comparison of the interobserver variability of 68Ga-NODAGA-exendin 4 PET/CT and 18F-DOPA PET/CT • Evaluation of the clinical outcome parameters (laboratory parameters (glucose) and dosage of medical treatment) after surgery;Timepoint(s) of evaluation of this end point: After collection of data from all patients | — |
Countries
Netherlands, Switzerland
Contacts
Public ContactMartin Gotthardt
RadboudUMC
Outcome results
None listed