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Clinical Trials/NCT02236754
NCT02236754
Completed
Early Phase 1

Scan Re-Scan Pancreatic Beta Cell Imaging Using PET Imaging and 18 F-FP-DTBZ

Columbia University1 site in 1 country23 target enrollmentJuly 2, 2013

Overview

Phase
Early Phase 1
Intervention
PET Scanning
Conditions
Healthy Volunteers
Sponsor
Columbia University
Enrollment
23
Locations
1
Primary Endpoint
Mean VMAT2 Functional Binding Capacity in the β Cells to 18 F-FP-DTBZ
Status
Completed
Last Updated
last year

Overview

Brief Summary

Type 1 diabetes mellitus (T1DM) develops when there is impaired insulin production due to loss of insulin producing cells (beta cells). The amount of insulin that can be produced is imperfectly correlated with beta cell mass (BCM). The development of a reliable method to noninvasively quantify the total amount of insulin producing beta cells would be of great benefit by providing an important endpoint for the development of new treatments of diabetes. The investigators have previously identified a specific marker on islet cells called vesicular monoamine transporter 2 (VMAT2) that the investigators now propose to use in positron emission tomography (PET) scanning to determine islet beta cell mass. The PET radiopharmaceutical 18 F-fluoropropyl(FP)-dihydrotetrabenazine(DTBZ) has been used previously in human subjects without adverse effects. It has shown promise in differentiating type 1 diabetes and non-diabetes. The investigators now hypothesize that repeat PET scans will be reproducible in the same subject. Subjects with normal BCM will be recruited from among normal weight non-diabetic people with plasma insulin levels within the normal range. Subjects with predicted reduced BCM will be recruited from among patients with T1DM who have low or non-measurable insulin levels. Two PET scan measurements will be taken in each subject and the amount of VMAT2 in the pancreas will be and compared for reproducible findings. Biochemical testing will also be performed and compared to PET scans as a potential indirect marker of beta cell mass.

Detailed Description

Diabetes results when the insulin secretory capacity of the beta cell population is lost or severely compromised.Plasma insulin levels have been used as a surrogate marker of beta cell mass (BCM) but insulin levels often do not correlate well. A "gold standard of measurement" to obtain BCM would be of great value. The aim of the proposed study is to evaluate an islet imaging technique using PET scanning to directly measure BCM and thus provide valuable information for monitoring disease progress and response to therapy in people with diabetes and in people at high risk for diabetes. Type 1 diabetes (T1DM) occurs when the beta cells are selectively destroyed by a T cell mediated autoimmune process. People at high risk for developing T1DM, such as first degree relatives of patients with T1DM, can sometimes be identified before the disease develops by measuring autoantibodies to beta cells, however this test is neither sensitive nor specific. Little is known about the natural history of BCM, turnover and cell lifetime, or the course of inflammation in diabetes. This is principally because the pancreas is a highly heterogeneous organ that is difficult to biopsy without significant complications, and BCM only comprises 1 to 2% of the total volume. Accurate assessment of BCM in human diabetes is limited to autopsy studies, which usually suffer from inadequate clinical information; thus, the development of non-invasive means of BCM measurement could be important for interventional therapies of T1DM, islet regeneration/stem cell therapy and islet transplantation.

Registry
clinicaltrials.gov
Start Date
July 2, 2013
End Date
April 23, 2014
Last Updated
last year
Study Type
Interventional
Study Design
Parallel
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Patients with type 1 diabetes may be enrolled if they meet all of the following criteria:
  • Are males or females between 18 and 70 years of age, inclusive
  • Have a diagnosis of type 1 diabetes mellitus as defined by the American Diabetes Association (ADA) criteria or by diagnosed as per their endocrinologist; duration \>5 years; Insulin dose requirements \<0.8 units/kg/day
  • HbA1c level between 5% and 8.5%
  • Have fasting C-Peptide \< 0.1 ng/ml
  • Have a body mass index (BMI) between 18 and 32 kg/m2
  • Able to tolerate PET imaging
  • In the judgment of the physician, are capable of fasting 4 to 6 hours prior to screening and Day 1 imaging procedures
  • Give informed consent
  • Healthy volunteers may be enrolled if they meeting all of the following criteria:

Exclusion Criteria

  • Potential participants must not have any of the following exclusion criteria:
  • Clinically significant renal dysfunction
  • Clinically significant liver dysfunction as determined by history, physical examination, and standard liver function testing at screening (aspartate aminotransferase (AST), alanine aminotransferase (ALT), Total/Direct Bilirubin, Alkaline Phosphatase)
  • Coagulopathy
  • Use medications known to affect dopaminergic function, including monoamine oxidase (MAO) inhibitors, tetrabenazine, or levodopa
  • Recent (within 3 months) or current treatment with drugs influencing beta cell function or insulin sensitivity (e.g. glucocorticoids, reserpine) medications known to affect dopaminergic function, including MAO inhibitors, tetrabenazine, or levodopa
  • Have polycystic ovarian syndrome
  • History of movement disorder such as Parkinson's Disease, Huntington's Disease
  • Clinically significant psychiatric disease or history of psychiatric illness such as depression, bipolar disease, anxiety or schizophrenia
  • Current use (within past year) of cocaine, methamphetamine, and/or ecstasy (3,4methylenedioxymethamphetamine (MDMA))

Arms & Interventions

Patients with T1D

Pancreatic 18 F-FP-DTBZ uptake will be measured with PET scanning in patients with longstanding T1D: subjects with predicted reduced beta cell mass (subjects with established T1DM who have low or no measurable stimulated insulin and c-peptide levels).

Intervention: PET Scanning

Healthy Controls

Pancreatic 18 F-FP-DTBZ uptake will be measured with PET scanning in healthy controls: subjects with predicted normal BCM (healthy, normal weight, non-diabetic individuals who have stimulated insulin and c-peptide levels within the normal range).

Intervention: 18 F-FP-DTBZ

Healthy Controls

Pancreatic 18 F-FP-DTBZ uptake will be measured with PET scanning in healthy controls: subjects with predicted normal BCM (healthy, normal weight, non-diabetic individuals who have stimulated insulin and c-peptide levels within the normal range).

Intervention: PET Scanning

Patients with T1D

Pancreatic 18 F-FP-DTBZ uptake will be measured with PET scanning in patients with longstanding T1D: subjects with predicted reduced beta cell mass (subjects with established T1DM who have low or no measurable stimulated insulin and c-peptide levels).

Intervention: 18 F-FP-DTBZ

Outcomes

Primary Outcomes

Mean VMAT2 Functional Binding Capacity in the β Cells to 18 F-FP-DTBZ

Time Frame: Up to 2 months from enrollment

The VMAT2 functional binding capacity in the body and tail of the pancreas is calculated as BPND (VMAT2 binding potential) × PET ROI (region-of-interest) Volume. BPND is unitless, and ROI unit is mL. Higher values of the VMAT functional binding capacity indicates greater β cell mass.

Study Sites (1)

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