This is a three-arm, 1:1:1 randomized, placebo controlled, double- blinded trial in which at least 28 subjects will receive active Anti-Thymocyte Globulin and Granulocyte colony-stimulating factor (ATG-GCSF), at least 28 subjects will receive ATG alone and at least 28 subjects will receive placebo alone within 100 days from diagnosis of Type 1 Diabetes (T1D). The primary objective of the study will be to determine the safety and ability of low dose ATG plus GCSF and low dose ATG alone to retain/enhance C-peptide production in new onset T1D patients demonstrating residual beta cell function.
The primary statistical hypothesis to be assessed in the study is whether the 2 hour area under the curve (change in baseline to 12 months) in residual beta cell function (C-peptide) will differ between those treated with ATG and GCSF or ATG alone as compared with placebo. The study will also examine the effect of the proposed treatments on surrogate markers for immunologic and metabolic outcomes.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
89
Thymoglobulin
Granulocyte colony stimulating factor (GCSF)
Normal saline administered by IV infusion to mimic ATG
Placebo prepared to mimic 6mg subcutaneous injection of GCSF
University of California - San Francisco
San Francisco, California, United States
Stanford University
Stanford, California, United States
Barbara Davis Center
Aurora, Colorado, United States
Yale University
New Haven, Connecticut, United States
University of Florida
Gainesville, Florida, United States
University of Miami
Miami, Florida, United States
University of South Florida Diabetes Center
Tampa, Florida, United States
Indiana University-Riley Hospital for Children
Indianapolis, Indiana, United States
University of Minnesota
Minneapolis, Minnesota, United States
Columbia University-Naomi Berrie Diabetes Center
New York, New York, United States
...and 3 more locations
Change in Area Under the Stimulated C-peptide Curve From Baseline to 12 Months.
The C-peptide 2 hour area under the curve (AUC) mean is calculated at baseline and 12 months and measured in nmol/L. The calculation for the concentration of c-peptide is a weighted average of the 6 timed measurements of c-peptide in nano-moles/Liter. We try to distinguish this calculation from the AUC by referring to it as the "AUC mean" and may be expressed algebraically as the AUC/(120 min.); thus, the units are the same as the y-axis").
Time frame: -10, 0 15, 30, 60, 90, and 120 minutes post-dose at baseline and 12 months
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