Type 1 Diabetes
Conditions
Brief summary
The primary purpose of this study is to find out if giving a 12 week course of Neulasta (Pegylated granulocyte colony stimulating factor (GCSF)) to people with type 1 diabetes (T1D) is safe. The secondary purpose of this research study is to determin if giving GCSF to patients with T1D changes the immune system or preserves insulin production.
Detailed description
As a participant in this study the following will happen: Blood test, Mixed meal Tolerance Test (MMT) to find out how much C-peptide is being produced, Human Leukocyte Antigen (HLA) test, Hemoglobin A1C (HbA1c) test for overall blood sugar control, and randomized to either receive the 12 week course of Neulasta (Pegylated granulocyte colony stimulating factor (GCSF)) or a placebo (no medication).
Interventions
DRUGNeulasta (Pegylated)
Subjects will receive 6mg/dose given every 2 weeks for 12 weeks. Subjects less than 45kg will receive 100mcg/kg/dose every 2 weeks for 12 weeks.
DRUGPlacebo
Placebo injections will be given in identical volumes in identical syringes in the identical subcutaneous manner. 6mg/dose given every 2 weeks for 12 weeks. Subjects less than 45kg will receive 100mcg/kg/dose every 2 weeks for 12 weeks.
DIETARY_SUPPLEMENTMixed Meal Tolerance Test (MMTT)
This is a 2 hour test to find out how much C-peptide is being produced. C-peptide is used to measure the insulin being made by the cells of the pancreas. Both groups will have this test performed.
OTHERBlood test
HbA1c is a blood test that measures the average blood sugar control and will be performed in both groups.
OTHERDNA Test
The HLA test looks at the different chromosomes (DNA) for diabetes and will be performed in both groups.
Sponsors
Juvenile Diabetes Research Foundation
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
University of Florida
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
* Must be \> 12 years old and have a confirmed diagnosis of recent onset T1D as defined by: * T1D for \< 6 months, * Stimulated C-peptide ≥ 0.2 pmol/ml * Presence of at least one diabetes-related autoantibody (e.g., GAD, IA-2, IAA, ICA) * Normal screening values for CBC * Willing to comply with intensive diabetes management * No history of allergy to GCSF * Females having reproductive potential must be willing to avoid pregnancy and have a negative pregnancy test
Exclusion criteria
* Known hypersensitivity to E.Coli-derived proteins, pegfilgrastim, Filgrastim, or any other component of the product * Chronic use of steroids or other immunosuppressive agents * Active infection * Inability to maintain intensive diabetes management * Pregnancy * History of malignancy * Currently participating in another type 1 diabetes treatment study. * Use of non-insulin pharmaceuticals that affect glycemic control
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Area Under the Curve C-peptide Changed Time 0, 12 weeks, 24 weeks, 48 weeks and 96 weeks. | Changed Time 0, 12 weeks, 24 weeks, 48 weeks and 96 weeks. | The target population distribution of change in the Area Under the Curve in residual beta cell function (C-peptide levels) is the same for treatment and placebo. |
| Number of participants with adverse events | 96 Weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Hemoglobin A1c (HbA1c) levels have the same target distribution for treatment and placebo | 96 weeks |
| Increase T regulatory cells (Treg) from the bone marrow | 12 weeks |
Countries
United States
Outcome results
None listed