Back to resultsProtege Extension Trial - Long Term Follow Up Trial for Subjects Who Completed the Protege Study (CP-MGA031-01)
Primary Purpose
Type 1 Diabetes Mellitus
Status
Terminated
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Blood samples for safety
Patient reported outcome questionnaires
Analysis of T-cell subsets
Sponsored by
About this trial
This is an interventional other trial for Type 1 Diabetes Mellitus focused on measuring Teplizumab, Protege, MGA031, Monoclonal antibody, Type 1 Diabetes Mellitus, T1DM, MacroGenics
Eligibility Criteria
Inclusion Criteria:
- Complete Protocol CP-MGA031-01 (i.e., all subjects who complete Study Day 728, regardless of how many doses of study drug are received).
- Provide written informed consent.
Exclusion Criteria:
None
Sites / Locations
- Atlanta Diabetes Associates
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm Type
Experimental
Experimental
Experimental
Placebo Comparator
Experimental
Arm Label
Double-blind Herold Regimen
Double-blind 33.3% Herold Regimen
Double-blind Curtailed Herold Regimen
Double-blind Placebo
Open-label Herold Regimen
Arm Description
Patients who had been assigned to Herold Regimen in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Patients who had been assigned to 33.3% Herold Regimen in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Patients who had been assigned to Curtailed Herold Regimen in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Patients who had been assigned to Placebo in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Patients who had been assigned to Herold Regimen in Segment 1 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Outcomes
Primary Outcome Measures
The Number of Participants Who Experience an Adverse Event, Serious Adverse Event or Adverse Event of Special Interest.
Secondary Outcome Measures
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%.
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%.
Proportion of Subjects With HbA1c <6.5%
Mean HbA1c at 6 Months
Mean HbA1c at 12 Months
C-peptide Area Under the Curve (AUC) at 6 Months
This outcome measure summarizes the mean and standard deviation of the observed value.
C-peptide AUC at 12 Months
This outcome measure summarizes the mean and standard deviation of the observed value.
Total Daily Insulin Usage at 6 Months
Total Daily Insulin Usage at 12 Months
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.
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.
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.
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.
Percentage of Cells by Immunophenotype
Human Anti-human Antibody (HAHA) Levels
Patients with HAHA levels > 0.
Full Information
NCT ID
NCT00870818
First Posted
March 26, 2009
Last Updated
July 19, 2023
Sponsor
MacroGenics
Collaborators
Eli Lilly and Company
1. Study Identification
Unique Protocol Identification Number
NCT00870818
Brief Title
Protege Extension Trial - Long Term Follow Up Trial for Subjects Who Completed the Protege Study (CP-MGA031-01)
Official Title
An Extension of Study CP-MGA031-01 to Evaluate the Long-Term Efficacy and Safety of Teplizumab (MGA031), a Humanized, FcR Non-Binding, Anti-CD3 Monoclonal Antibody, in Patients With Recent-Onset Type 1 Diabetes Mellitus
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Terminated
Why Stopped
Business decision
Study Start Date
February 2009 (undefined)
Primary Completion Date
February 2011 (Actual)
Study Completion Date
May 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MacroGenics
Collaborators
Eli Lilly and Company
4. Oversight
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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).
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type 1 Diabetes Mellitus
Keywords
Teplizumab, Protege, MGA031, Monoclonal antibody, Type 1 Diabetes Mellitus, T1DM, MacroGenics
7. Study Design
Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Non-Randomized
Enrollment
219 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Double-blind Herold Regimen
Arm Type
Experimental
Arm Description
Patients who had been assigned to Herold Regimen in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Arm Title
Double-blind 33.3% Herold Regimen
Arm Type
Experimental
Arm Description
Patients who had been assigned to 33.3% Herold Regimen in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Arm Title
Double-blind Curtailed Herold Regimen
Arm Type
Experimental
Arm Description
Patients who had been assigned to Curtailed Herold Regimen in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Arm Title
Double-blind Placebo
Arm Type
Placebo Comparator
Arm Description
Patients who had been assigned to Placebo in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Arm Title
Open-label Herold Regimen
Arm Type
Experimental
Arm Description
Patients who had been assigned to Herold Regimen in Segment 1 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Intervention Type
Diagnostic Test
Intervention Name(s)
Blood samples for safety
Intervention Description
serum chemistry, hematology, infection screen, thyroid function, Insulin, hemoglobin A1c,and autoantibodies
Intervention Type
Behavioral
Intervention Name(s)
Patient reported outcome questionnaires
Intervention Description
EQ-5D, Peds QL, Low blood sugar survey, and hospitalization information.
Intervention Type
Diagnostic Test
Intervention Name(s)
Analysis of T-cell subsets
Intervention Description
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
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
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
Title
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
Title
Proportion of Subjects With HbA1c <6.5%
Time Frame
Month 6
Title
Mean HbA1c at 6 Months
Time Frame
6 months
Title
Mean HbA1c at 12 Months
Time Frame
Month 12
Title
C-peptide Area Under the Curve (AUC) at 6 Months
Description
This outcome measure summarizes the mean and standard deviation of the observed value.
Time Frame
Month 6
Title
C-peptide AUC at 12 Months
Description
This outcome measure summarizes the mean and standard deviation of the observed value.
Time Frame
Month 12
Title
Total Daily Insulin Usage at 6 Months
Time Frame
Month 6
Title
Total Daily Insulin Usage at 12 Months
Time Frame
Month 12
Title
Mean Values for Participant-reported Outcomes on the 5-dimension European Quality of Life Questionnaire. (EQ-5D)
Description
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
Title
Mean Values for Participant-reported Outcomes on the EQ-5D
Description
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
Title
Pediatric Quality of Life Questionnaire Total Score
Description
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
Title
Pediatric Quality of Life Questionnaire Total Score
Description
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
Title
Percentage of Cells by Immunophenotype
Time Frame
Month 6
Title
Human Anti-human Antibody (HAHA) Levels
Description
Patients with HAHA levels > 0.
Time Frame
Month 6 and 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
37 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Complete Protocol CP-MGA031-01 (i.e., all subjects who complete Study Day 728, regardless of how many doses of study drug are received).
Provide written informed consent.
Exclusion Criteria:
None
Facility Information:
Facility Name
Atlanta Diabetes Associates
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30309
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Protege Extension Trial - Long Term Follow Up Trial for Subjects Who Completed the Protege Study (CP-MGA031-01)
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