Neulasta in Type 1 Diabetes

NCT00662519 | Completed Phase 1 DMC

• 4 other identifiers: UF IRB 280-2006JDRF 11-2007-8251R21DK078863-01A1GCRC 683
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Type 1 Diabetes

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• 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

Study Arms (2)

Neulasta

EXPERIMENTAL

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); Dietary Supplement: Mixed Meal Tolerance Test (MMTT); Other: Blood test; Other: DNA Test

Placebo

PLACEBO COMPARATOR

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; Dietary Supplement: Mixed Meal Tolerance Test (MMTT); Other: Blood test; Other: DNA Test

Interventions

Neulasta (Pegylated) DRUG

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.

Also known as: Pegylated granulocyte colony stimulating factor (GCSF)

Neulasta

Placebo DRUG

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.

Also known as: Saline

Placebo

Mixed Meal Tolerance Test (MMTT) DIETARY_SUPPLEMENT

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.

Neulasta; Placebo

Blood test OTHER

HbA1c is a blood test that measures the average blood sugar control and will be performed in both groups.

Also known as: Hemoglobin A1C (HbA1c)

Neulasta; Placebo

DNA Test OTHER

The HLA test looks at the different chromosomes (DNA) for diabetes and will be performed in both groups.

Also known as: Human Leukocyte Antigen (HLA)

Neulasta; Placebo

Eligibility Criteria

• Age: 12 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Florida: lead
• Juvenile Diabetes Research Foundation: collaborator
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator

Study Sites (1)

University of Florida

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1

Interventions

pegfilgrastim; pegylated granulocyte colony-stimulating factor; Sodium Chloride; Hematologic Tests; Hemoglobin A; Human Papillomavirus DNA Tests; HLA Antigens

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Hemoglobins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Globins; Hemeproteins; Molecular Diagnostic Techniques; Genetic Techniques; Histocompatibility Antigens; Antigens, Surface; Antigens; Biological Factors; Isoantigens

Study Officials

• Michael J Haller, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2008
• First Posted: April 21, 2008
• Study Start: April 1, 2008
• Primary Completion: March 1, 2014
• Study Completion: March 1, 2014
• Last Updated: April 9, 2015

Record last verified: 2015-04

Locations

US (1)