Research at CHOP

1
Research at CHOP

• Division of Endocrinology
• Diabetes Center for Children
• Childrens Hospital of Philadelphia
• Gerre McGinley, RD,LDN,CDE

2
Program/Study Categories

• Prediction/ Prevention/ Genetics
• Early Intervention
• New Technology
• Diabetes-related Complications/ Obesity

3
Prediction/Prevention Study

• TrialNet
• Goals Understand risk factors, methods for
  prevention and treatment of T1DM
• Sponsor/Funding NIH, ADA, JDRF

4
TrialNet

• Who
• All T1DM relatives
• 1st degree 1-45 years
• 2nd degree 1-20 years
• Looking to follow participants who have positive
  diabetes antibodies to see if they develop
  diabetes

5
TrialNet

• What
• Up to five year study with three phases
• Participation and number of visits depends on
  tests results at each phase
• phase 1 one blood test
• phase 2 OGTT, blood tests
• phase 3 blood tests every six months

6
Genetics Study

• T1DGC-
  Type 1 Diabetes Genetics Consortium
• Goals
• Create repository to study gene structure and
  function
• Facilitate studies to identify genetic bases for
  T1DM
• Sponsor/Funding NIH, JDRF, NIDDK

7
T1DGC

• Who
• Any age child OR child plus two biological
  parents
• Mexican American or African American
• What
• One visit for blood draw and questionnaire

8
Early Intervention Study

• Atorvastatin (Lipitor)
• Goals
• Evaluate effect of atorvastatin on preservation
  of beta cell function (Antiinflammatory- may
  decrease autoimmune activity)
• Sponsor/Funding FDA, Phizer Inc

9
Atorvastatin

• Who
• Participants within six weeks of diagnosis of
  T1DM
• Ages 10-19 years

10
Atorvastatin

• What
• 1.5 year study with one pill daily
• 1 year treatment, six month med washout
• Double blind, random 21
• (two get drug one gets placebo)
• Visits every three months with clinic appt

11
Early Intervention Study

• ITN -Immune Tolerance Network
• ?Goals
• Evaluate effect of Antithymocyte Globulin (ATG)
  on preservation of beta cell function
  (Immunosuppressant- may prolong honeymoon period
  in T1DM)
• Sponsor/Funding NIH

12
ITN

• Who
• Participants within six weeks of diagnosis T1DM
• Ages 12-18 years

13
ITN

• What
• Two year study
• Participants blinded to treatment
• Two study teams
• ? initial treatment unblinded
• ? follow-up management blinded

14
ITN

• Onetime five day hospital admission for infusion
• Follow-up visits three within eight weeks
• then every three months with clinic appt
• Five MMTT with blood draws

15
Early Intervention Study

• TODAY Study
• Goals
• Evaluate treatment regimens and clinical courses
  of T2DM in youth
• Sponsor/Funding NIH, NIDDK

16
TODAY

• Who
• Diagnosis of T2DM less than two years
• Ages 10-17 years
• BMI gt85th percentile
• Maintain A1C lt 8 on metformin treatment alone
  during Run -In
• Presence of family support member

17
TODAY

• What
• Prospective, randomized trial for up to six years
• 13 clinical sites in US
• Three treatment arms include standard diabetes
  education plus
• metformin, metformin rosiglitazone or
  metformin an intensive lifestyle program

18
TODAY

• What
• Double blinded medical treatment arms
• Medical visits
• Every 2 months first year, then every 3 months
• Lifestyle visits
• Weekly for six months, bi-weekly for six months,
  monthly for year two, every three months rest of
  study

19
New Technology Studies

• STAR 3
• Goal
• Compare blood glucose control (A1C) using
  Real-Time device system versus Lantus/Novolog
  treatment
• Sponsor/Funding
• Medtronic Minimed

20
STAR 3

• Who
• Ages 7-18 years
• Pump naïve
• Seen in DCC gt six months
• A1C 7.5 or 9.5

21
STAR 3

• What
• 1.5 year study one year treatment plus six month
  extension
• Open label randomized (all get pump at six months
  ext)
• Weekly visits for eight weeks with intensive
  education
• Follow-up visits every three months

22
Complication/Obesity Studies

• Abdominal Adiposity
• Goal
• Evaluate waist circumference as a predictor of
  insulin resistance
• One visit for blood draw and anthropometrics

23
Obesity

• Dyslipidemia
• Goal
• Identify risk factors for obesity and T2DM
• Two year study with two visits, Baseline and two
  year follow-up
• Lean controls, Obese insulin sensitive, Obese
  insulin resistant, Obese w/ T2DM

24
Obesity

• Natural Hx/GLP1
• Goals
• Measure insulin secretion/sensitivity in T2DM and
  obese siblings
• Measure GLP-1 in T2DM and controls
• One annual visit for five years in two groups
  obese T2DM and obese siblings
• Ages puberty -17 years

25
Obesity

• Metabolic Syndrome
• Goal
• Evaluate risk factors for metabolic syndrome
• One overnight visit for sleep study in two
  groups obese insulin sensitive and obese insulin
  resistant