Neulasta in Type 1 Diabetes

Study Overview
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Brief Summary
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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
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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).

Official Title
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Short Course of Pegylated GCSF (Neulasta®) as Immunomodulatory Therapy for Type 1 Diabetes

Conditions
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Type 1 Diabetes

Intervention / Treatment
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* Drug: Neulasta (Pegylated)
* Drug: Placebo
* Dietary Supplement: Mixed Meal Tolerance Test (MMTT)
* Other: Blood test
* Other: DNA Test


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Neulasta<br>Subjects will receive Neulasta subcutaneously every 2 weeks for 12 weeks (6 doses). In addition, the following test will be done: Mixed Meal Tolerance Test (MMTT), Hemoglobin A1C (HbA1c) blood test, and Human Leukocyte Antigen (HLA) DNA Test. | Drug: Neulasta (Pegylated)<br>* 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.<br>* Other names: Pegylated granulocyte colony stimulating factor (GCSF);Dietary Supplement: Mixed Meal Tolerance Test (MMTT)<br>* 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.<br>Other: Blood test<br>* HbA1c is a blood test that measures the average blood sugar control and will be performed in both groups.<br>* Other names: Hemoglobin A1C (HbA1c);Other: DNA Test<br>* The HLA test looks at the different chromosomes (DNA) for diabetes and will be performed in both groups.<br>* Other names: Human Leukocyte Antigen (HLA);|
| Placebo Comparator: Placebo<br>Placebo injections will be given in identical volumes in identical syringes in the identical subcutaneous manner. In addition, the following test will be done: Mixed Meal Tolerance Test (MMTT), Hemoglobin A1C (HbA1c) blood test, and Human Leukocyte Antigen (HLA) DNA Test. | Drug: Placebo<br>* 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.<br>* Other names: Saline;Dietary Supplement: Mixed Meal Tolerance Test (MMTT)<br>* 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.<br>Other: Blood test<br>* HbA1c is a blood test that measures the average blood sugar control and will be performed in both groups.<br>* Other names: Hemoglobin A1C (HbA1c);Other: DNA Test<br>* The HLA test looks at the different chromosomes (DNA) for diabetes and will be performed in both groups.<br>* Other names: Human Leukocyte Antigen (HLA);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Area Under the Curve C-peptide 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. | Changed Time 0, 12 weeks, 24 weeks, 48 weeks and 96 weeks. | 
| Number of participants with adverse events |  | 96 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 |