Congenital hyperinsulinism MedDRA version: 20.0 Level: PT Classification code 10061211 Term: Hyperinsulinism System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Interventions
Trade Name: Byetta
Product Name: 68-Ga-NODAGA-exendin4
Pharmaceutical Form: Radiopharmaceutical precursor, solution
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 PETICT) 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 ABCCS/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-lR 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 excfuded). 68Ga-NODAGA-exendin-4 pET ICT imaging data will be compared with autoradiography and histology performed on specimens collected during surgery.;Secondary Objective: o Comparing 68Ga-NODAGA-exendln-4 PET/CT and 18F-DOPA PET/CT in order to determine the sensitivity of the new imaging method. o Determining the lowest activity dose of 68Ga-NODAGA-exendin 4 needed for accurate imaging. o 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 PETIGT scans performed in 2 patients. o Assessing the safety (side effects) of 68Ga-NODAGA-exendin 4 as compared to 18F-DOpA o Calculating and comparing the interobserver variability of 68Ga-NODAGA-exendin 4PET/CT and 18F-DOPA PET/CT o 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. - 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