The purpose of this study is to assess the long term safety and efficacy in subjects with Type 1 Diabetes Mellitus who completed the Protege Study (CP-MGA031-01).
The primary objective of the extension study is to assess long-term safety, with particular focus on the development of serious adverse events (SAEs), adverse events of special interest (AESIs) including opportunistic infections and lymphoproliferative disease, and other immediately reportable events (IREs), in subjects with recent-onset T1DM who complete CP-MGA031-01. The secondary objectives of the extension study are to: 1) assess long-term efficacy; 2) evaluate immunological effects(North America only); 3) measure anti-teplizumab antibody levels;4) assess Health Related Quality of Life Questionnaires.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
QUADRUPLE
Enrollment
219
serum chemistry, hematology, infection screen, thyroid function, Insulin, hemoglobin A1c,and autoantibodies
EQ-5D, Peds QL, Low blood sugar survey, and hospitalization information.
CD3, CD4, CD8, CD19, CD3+ CD16+ CD56+ subsets; CD3-CD16+ CD56+ subsets; CD4+CD25+, CD8+CD25+, CD4+CD69+, CD8+CD69+, CD4+CD40+ subsets CD4+ and CD8+CD25+FoxP3+Treg Subsets
Atlanta Diabetes Associates
Atlanta, Georgia, United States
The Number of Participants Who Experience an Adverse Event, Serious Adverse Event or Adverse Event of Special Interest.
Time frame: Duration of study participation up to 15 months
Proportion of Subjects in Segment 2 With Both a Total Daily Insulin Dose of Less Than 0.5 U/kg/Day and Hemoglobin A1c (HbA1c) Level of Less Than 6.5%.
Time frame: Month 6
Proportion of Subjects in Segment 2 With Both a Total Daily Insulin Dose of Less Than 0.5 U/kg/Day and Hemoglobin A1c (HbA1c) Level of Less Than 6.5%.
Time frame: Month 12
Proportion of Subjects With HbA1c <6.5%
Time frame: Month 6
Mean HbA1c at 6 Months
Time frame: 6 months
Mean HbA1c at 12 Months
Time frame: Month 12
C-peptide Area Under the Curve (AUC) at 6 Months
This outcome measure summarizes the mean and standard deviation of the observed value.
Time frame: Month 6
C-peptide AUC at 12 Months
This outcome measure summarizes the mean and standard deviation of the observed value.
Time frame: Month 12
Total Daily Insulin Usage at 6 Months
Time frame: Month 6
Total Daily Insulin Usage at 12 Months
Time frame: Month 12
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Mean Values for Participant-reported Outcomes on the 5-dimension European Quality of Life Questionnaire. (EQ-5D)
The EQ-5D is a self-reported survey that measures quality of life across 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension is scored on a 3-level severity ranking that ranges from "no problems", "some problems", and "extreme problems". The lowest possible score is 5 and the highest possible score is 15. Lower scores indicate better quality of life.
Time frame: Month 6
Mean Values for Participant-reported Outcomes on the EQ-5D
The EQ-5D is a self-reported survey that measures quality of life across 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension is scored on a 3-level severity ranking that ranges from "no problems", "some problems", and "extreme problems". The lowest possible score is 5 and the highest possible score is 15. Lower scores indicate better quality of life.
Time frame: Month 12
Pediatric Quality of Life Questionnaire Total Score
The 23-item PEDs QL generic core scales were designed to measure the core dimensions of health (physical, emotional, social and school functioning). Answers are scored from 0 meaning never to 4 meaning almost always. Lower scores indicated higher quality of life. Items on the questionnaire were reversely scored and linearly transformed to a 0-100 scale, so that higher scores indicated better patient reported outcome. The mean was computed as the sum of the items over the number of items answered (to account for missing data). If more than 50% of the items in the scale were missing, the score was not computed.
Time frame: Month 6
Pediatric Quality of Life Questionnaire Total Score
The 23-item PEDs QL generic core scales were designed to measure the core dimensions of health (physical, emotional, social and school functioning). Answers are scored from 0 meaning never to 4 meaning almost always. Lower scores indicated higher quality of life. Items on the questionnaire were reversely scored and linearly transformed to a 0-100 scale, so that higher scores indicated better patient reported outcome. The mean was computed as the sum of the items over the number of items answered (to account for missing data). If more than 50% of the items in the scale were missing, the score was not computed.
Time frame: Month 12
Percentage of Cells by Immunophenotype
Time frame: Month 6
Human Anti-human Antibody (HAHA) Levels
Patients with HAHA levels \> 0.
Time frame: Month 6 and 12