Protege Extension Trial - Long Term Follow Up Trial for Subjects Who Completed the Protege Study (CP-MGA031-01)

Not Applicable

Terminated

• Conditions: Type 1 Diabetes Mellitus
• Interventions: Diagnostic Test: Blood samples for safety; Behavioral: Patient reported outcome questionnaires; Diagnostic Test: Analysis of T-cell subsets

• Registration Number: NCT00870818
• Lead Sponsor: MacroGenics

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.

Study Timeline

• Study Start: 2009-02
• Study First Submitted: 2009-03-26
• Study First Submitted QC: 2009-03-26
• Study First Posted: 2009-03-27
• Primary Completion: 2011-02
• Study Completion: 2011-05
• Results First Submitted: 2023-03-08
• Status Verified: 2023-07
• Results First Submitted QC: 2023-07-19
• Last Update Submitted: 2023-07-19
• Results First Posted: 2023-08-09
• Last Update Posted: 2023-08-09

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 219

Inclusion Criteria

1. Complete Protocol CP-MGA031-01 (i.e., all subjects who complete Study Day 728, regardless of how many doses of study drug are received).
2. Provide written informed consent.

Exclusion Criteria

None

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Open-label Herold Regimen	Analysis of T-cell subsets	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.
Double-blind Curtailed Herold Regimen	Analysis of T-cell subsets	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.
Double-blind Herold Regimen	Patient reported outcome questionnaires	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.
Double-blind Curtailed Herold Regimen	Blood samples for safety	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.
Double-blind 33.3% Herold Regimen	Blood samples for safety	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.
Double-blind 33.3% Herold Regimen	Analysis of T-cell subsets	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.
Open-label Herold Regimen	Blood samples for safety	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.
Double-blind Herold Regimen	Analysis of T-cell subsets	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.
Double-blind 33.3% Herold Regimen	Patient reported outcome questionnaires	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.
Double-blind Placebo	Blood samples for safety	Patients who had been assigned to Placebo in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Double-blind Placebo	Analysis of T-cell subsets	Patients who had been assigned to Placebo in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.
Open-label Herold Regimen	Patient reported outcome questionnaires	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.
Double-blind Herold Regimen	Blood samples for safety	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.
Double-blind Curtailed Herold Regimen	Patient reported outcome questionnaires	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.
Double-blind Placebo	Patient reported outcome questionnaires	Patients who had been assigned to Placebo in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.

Primary Outcome Measures

Name	Time	Method
The Number of Participants Who Experience an Adverse Event, Serious Adverse Event or Adverse Event of Special Interest.	Duration of study participation up to 15 months

Secondary Outcome Measures

Name	Time	Method
Total Daily Insulin Usage at 6 Months	Month 6
Mean HbA1c at 12 Months	Month 12
C-peptide Area Under the Curve (AUC) at 6 Months	Month 6	This outcome measure summarizes the mean and standard deviation of the observed value.
Mean Values for Participant-reported Outcomes on the 5-dimension European Quality of Life Questionnaire. (EQ-5D)	Month 6	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	Month 12	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.
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%.	Month 12
Proportion of Subjects With HbA1c <6.5%	Month 6
Mean HbA1c at 6 Months	6 months
Mean Values for Participant-reported Outcomes on the EQ-5D	Month 12	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.
Human Anti-human Antibody (HAHA) Levels	Month 6 and 12	Patients with HAHA levels \> 0.
C-peptide AUC at 12 Months	Month 12	This outcome measure summarizes the mean and standard deviation of the observed value.
Total Daily Insulin Usage at 12 Months	Month 12
Percentage of Cells by Immunophenotype	Month 6

Trial Locations

Locations (1)

Atlanta Diabetes Associates; 🇺🇸 Atlanta, Georgia, United States