Reversing Type 1 Diabetes After it is Established

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
The Leona M. and Harry B. Helmsley Charitable Trust
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT01106157
First received: April 15, 2010
Last updated: January 19, 2016
Last verified: January 2016
Results First Received: October 14, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety Study;   Intervention Model: Parallel Assignment;   Masking: Single Blind (Subject);   Primary Purpose: Treatment
Condition: Diabetes Mellitus, Type 1
Interventions: Drug: Anti-Thymocyte Globin (ATG)
Drug: Placebo
Drug: Pegylated GCSF

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Anti-Thymocyte Globin Plus Pegylated GCSF

Subjects will receive an infusion of Anti-Thymocyte Globin (ATG) followed by 6 doses of pegylated GCSF every 2 weeks for 10 weeks.

Anti-Thymocyte Globin plus pegylated GCSF: Anti-Thymocyte Globin (ATG)will be given as 0.5/mg/kg on day 1 and 2mg/kg on day 2, plus 6 doses of pegylated GCSF (6mg/dose) given subcutaneously every 2 weeks beginning after the ATG infusion.

Placebo

Saline infusion will be given on both Day 1 and Day 2 followed by placebo injection given in identical volumes in identical syringes in the identical subcutaneous manner.

Placebo: Saline infusions will be given on Day 1 and Day 2 followed by placebo injections given in identical volumes in identical syringes.


Participant Flow:   Overall Study
    Anti-Thymocyte Globin Plus Pegylated GCSF     Placebo  
STARTED     17     8  
COMPLETED     16     8  
NOT COMPLETED     1     0  
Withdrawal by Subject                 1                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Anti-Thymocyte Globin Plus Pegylated GCSF

Subjects will receive an infusion of Anti-Thymocyte Globin (ATG) followed by 6 doses of pegylated GCSF every 2 weeks for 10 weeks.

Anti-Thymocyte Globin plus pegylated GCSF: Anti-Thymocyte Globin (ATG)will be given as 0.5/mg/kg on day 1 and 2mg/kg on day 2, plus 6 doses of pegylated GCSF (6mg/dose)given subcutaneously every 2 weeks beginning after the ATG infusion

Placebo

Saline infusion will be given on both Day 1 and Day 2 followed by placebo injection given in identical volumes in identical syringes in the identical subcutaneous manner

Placebo: Saline infusions will be given on Day 1 and Day 2 followed by placebo injections given in identical volumes in identical syringes

Total Total of all reporting groups

Baseline Measures
    Anti-Thymocyte Globin Plus Pegylated GCSF     Placebo     Total  
Number of Participants  
[units: participants]
  17     8     25  
Age  
[units: participants]
     
<=18 years     5     5     10  
Between 18 and 65 years     12     3     15  
>=65 years     0     0     0  
Age  
[units: years]
Mean (Standard Deviation)
  23.64  (10)     23.55  (10.6)     23.61  (10.0)  
Gender  
[units: participants]
     
Female     5     3     8  
Male     12     5     17  
Region of Enrollment  
[units: participants]
     
United States     17     8     25  



  Outcome Measures
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1.  Primary:   Change in Metabolic Function Baseline to 12 Months.   [ Time Frame: Baseline and 12 months ]

2.  Secondary:   Percent Change in Regulatory T Cells (Treg) Baseline to 12 Months   [ Time Frame: Change in Baseline to 12 months ]

3.  Secondary:   A1c   [ Time Frame: Change in baseline to 12 months ]

4.  Secondary:   Change in Insulin Requirements, Baseline to 12 Months   [ Time Frame: Change from baseline to 12 months ]

5.  Secondary:   Change in Glutamic Acid Decarboxylase Antibodies (GADA) From Baseline to 12 Months   [ Time Frame: Change from baseline to 12 months ]

6.  Secondary:   Change in Insulin Autoantibodies (IAA) From Baseline to 12 Months   [ Time Frame: Change from baseline to 12 months ]

7.  Secondary:   Change in Insulinoma Associated 2 Autoantibodies (IA-2A) From Baseline to 12 Months   [ Time Frame: Change from baseline to 12 months ]

8.  Secondary:   Change in Zinc Transporter 8 Autoantibodies (ZnT8A) From Baseline to 12 Months   [ Time Frame: Change from baseline to 12 months ]

9.  Secondary:   Percentage of Neutrophils   [ Time Frame: Change from baseline to 12 months ]

10.  Secondary:   Change in White Blood Count (WBC) From Baseline to 12 Months   [ Time Frame: Change from baseline to 12 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Michael J Haller
Organization: University of Florida
phone: 3522739264
e-mail: hallemj@peds.ufl.edu


Publications:
Publications automatically indexed to this study:

Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT01106157     History of Changes
Other Study ID Numbers: UF-ATG-GCSF001
Study First Received: April 15, 2010
Results First Received: October 14, 2015
Last Updated: January 19, 2016
Health Authority: United States: Food and Drug Administration