Neulasta in Type 1 Diabetes

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

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.

Study Overview

• Status: Completed
• Conditions: Type 1 Diabetes
• Intervention / Treatment: Drug: Neulasta (Pegylated); Dietary supplement: Mixed Meal Tolerance Test (MMTT); Other: Blood test; Other: DNA Test; Drug: Placebo

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).

• Study Type: Interventional
• Enrollment (Actual): 21
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 45 years (Child, Adult)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Neulasta; 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); 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.; Other Names: Pegylated granulocyte colony stimulating factor (GCSF); Dietary supplement: Mixed 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.; Other: Blood test; HbA1c is a blood test that measures the average blood sugar control and will be performed in both groups.; Other Names: Hemoglobin A1C (HbA1c); Other: DNA Test; The HLA test looks at the different chromosomes (DNA) for diabetes and will be performed in both groups.; Other Names: Human Leukocyte Antigen (HLA)
Placebo Comparator: Placebo; 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.	Dietary supplement: Mixed 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.; Other: Blood test; HbA1c is a blood test that measures the average blood sugar control and will be performed in both groups.; Other Names: Hemoglobin A1C (HbA1c); Other: DNA Test; The HLA test looks at the different chromosomes (DNA) for diabetes and will be performed in both groups.; Other Names: Human Leukocyte Antigen (HLA); Drug: Placebo; 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.; Other Names: Saline

What is the study measuring?

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	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

Collaborators and Investigators

• Sponsor: University of Florida
• Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Juvenile Diabetes Research Foundation

Study record dates

Study Major Dates

• Study Start: April 1, 2008
• Primary Completion (Actual): March 1, 2014
• Study Completion (Actual): March 1, 2014

Study Registration Dates

• First Submitted: April 16, 2008
• First Submitted That Met QC Criteria: April 16, 2008
• First Posted (Estimate): April 21, 2008

Study Record Updates

• Last Update Posted (Estimate): April 9, 2015
• Last Update Submitted That Met QC Criteria: April 8, 2015
• Last Verified: April 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Immune System Diseases; Autoimmune Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 1; Physiological Effects of Drugs; Immunologic Factors; Adjuvants, Immunologic; Lenograstim
• Other Study ID Numbers: UF IRB 280-2006; JDRF 11-2007-825 (Other Grant/Funding Number: JDRF); 1R21DK078863-01A1 (U.S. NIH Grant/Contract); GCRC 683 (Other Grant/Funding Number: GCRC)