Type 1 Diabetes MellitusNCT00870818

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

A study testing Blood samples for safety, Patient reported outcome questionnaires and Analysis of T-cell subsets in people with Type 1 Diabetes Mellitus, run by MacroGenics at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial 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). Read more about this study

Who is it for?: Ages 10 to 37 years
Conditions the study is for: Type 1 Diabetes Mellitus
What is being tested?: Blood samples for safety, Patient reported outcome questionnaires and Analysis of T-cell subsets
Type of treatment: Lifestyle or therapy programme
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 219 participants
Where: 1 siteUnited States
Study dates: Feb 2009to May 2011
Phase: Interventional
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 10 to 37 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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.

Who cannot join

None

Trial locations and contacts

1 site in 1 country.

United States1 site

Atlanta Diabetes Associates
Atlanta, Georgia, 30309

Other trials in United States

About this study

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

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).

Read the 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.

Conditions: Type 1 Diabetes Mellitus
Being tested: Blood samples for safety, Patient reported outcome questionnaires, Analysis of T-cell subsets
Keywords: Teplizumab, Protege, MGA031, Monoclonal antibody, Type 1 Diabetes Mellitus, T1DM, MacroGenics
Medical subject headings: Diabetes Mellitus, Type 1

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Blood samples for safetyDiagnostic test
serum chemistry, hematology, infection screen, thyroid function, Insulin, hemoglobin A1c,and autoantibodies
Patient reported outcome questionnairesBehavioural
EQ-5D, Peds QL, Low blood sugar survey, and hospitalization information.
Analysis of T-cell subsetsDiagnostic test
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

Study groups in NCT00870818
Study group	Role	What they receive
Double-blind Herold Regimen	Experimental	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. Receives: Diagnostic Test: Blood samples for safety; Behavioral: Patient reported outcome questionnaires; Diagnostic Test: Analysis of T-cell subsets
Double-blind 33.3% Herold Regimen	Experimental	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. Receives: Diagnostic Test: Blood samples for safety; Behavioral: Patient reported outcome questionnaires; Diagnostic Test: Analysis of T-cell subsets
Double-blind Curtailed Herold Regimen	Experimental	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. Receives: Diagnostic Test: Blood samples for safety; Behavioral: Patient reported outcome questionnaires; Diagnostic Test: Analysis of T-cell subsets
Double-blind Placebo	Placebo comparator	Patients who had been assigned to Placebo in Segment 2 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data. Receives: Diagnostic Test: Blood samples for safety; Behavioral: Patient reported outcome questionnaires; Diagnostic Test: Analysis of T-cell subsets
Open-label Herold Regimen	Experimental	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. Receives: Diagnostic Test: Blood samples for safety; Behavioral: Patient reported outcome questionnaires; Diagnostic Test: Analysis of T-cell subsets

What is being measured

The primary outcome is the main question the study is designed to answer.

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

Secondary outcomes (15)

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%.
Measured: 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%.
Measured: Month 12
Proportion of Subjects With HbA1c <6.5%
Measured: Month 6
Mean HbA1c at 6 Months
Measured: 6 months
Mean HbA1c at 12 Months
Measured: 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.
Measured: Month 6
C-peptide AUC at 12 Months
This outcome measure summarizes the mean and standard deviation of the observed value.
Measured: Month 12
Total Daily Insulin Usage at 6 Months
Measured: Month 6
Total Daily Insulin Usage at 12 Months
Measured: Month 12
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Month 12
Percentage of Cells by Immunophenotype
Measured: Month 6
Human Anti-human Antibody (HAHA) Levels
Patients with HAHA levels > 0.
Measured: Month 6 and 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Feb 2009May 2011

First posted27 Mar 2009 (estimated)
Study startFeb 2009
Primary completionFeb 2011
Study completionMay 2011

Why it stopped: Business decision

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Type 1 Diabetes MellitusStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase N/A
Primary purpose: Other
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 219 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00870818
Sponsor's protocol ID: CP-MGA031-02
Status last verified: Jul 2023
First submitted: 26 Mar 2009
Last updated: 9 Aug 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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All Type 1 Diabetes Mellitus trials

Study at a glance

From the registry record, last updated by the sponsor on 9 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT00870818
Acronym / sponsor ID: CP-MGA031-02
Conditions: Type 1 Diabetes Mellitus
Keywords: Teplizumab, Protege, MGA031, Monoclonal antibody, Type 1 Diabetes Mellitus, T1DM, MacroGenics
Study type: Interventional
Recruitment status: TerminatedReason stopped: Business decision
Enrolment: 219 participants (actual)
Lead sponsor: MacroGenicsIndustry (pharma, biotech, devices)
Collaborators: Eli Lilly and Company
Study start: Feb 2009
Primary completion: Feb 2011 (actual)
Study completion: May 2011 (actual)
First posted: 27 Mar 2009 (estimated)
Results: Posted 9 Aug 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Study design

Primary purpose: Other
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 5

Arm	Type	Receives
Double-blind Herold Regimen	Experimental	Diagnostic Test: Blood samples for safety, Behavioral: Patient reported outcome questionnaires, Diagnostic Test: Analysis of T-cell subsetsPatients 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	Experimental	Diagnostic Test: Blood samples for safety, Behavioral: Patient reported outcome questionnaires, Diagnostic Test: Analysis of T-cell subsetsPatients 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 Curtailed Herold Regimen	Experimental	Diagnostic Test: Blood samples for safety, Behavioral: Patient reported outcome questionnaires, Diagnostic Test: Analysis of T-cell subsetsPatients 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	Placebo comparator	Diagnostic Test: Blood samples for safety, Behavioral: Patient reported outcome questionnaires, Diagnostic Test: Analysis of T-cell subsetsPatients 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	Experimental	Diagnostic Test: Blood samples for safety, Behavioral: Patient reported outcome questionnaires, Diagnostic Test: Analysis of T-cell subsetsPatients who had been assigned to Herold Regimen in Segment 1 of Study CP-MGA031-01 were enrolled to gather additional safety and efficacy data.

Who may qualify

Ages: 10 to 37 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (2 listed)

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.

Key exclusion criteria (1 listed)

None

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Blood samples for safetyserum chemistry, hematology, infection screen, thyroid function, Insulin, hemoglobin A1c,and autoantibodies	Diagnostic test	Double-blind 33.3% Herold Regimen, Double-blind Curtailed Herold Regimen, Double-blind Herold Regimen, Double-blind Placebo, Open-label Herold Regimen
Patient reported outcome questionnairesEQ-5D, Peds QL, Low blood sugar survey, and hospitalization information.	Behavioural	Double-blind 33.3% Herold Regimen, Double-blind Curtailed Herold Regimen, Double-blind Herold Regimen, Double-blind Placebo, Open-label Herold Regimen
Analysis of T-cell subsetsCD3, 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	Diagnostic test	Double-blind 33.3% Herold Regimen, Double-blind Curtailed Herold Regimen, Double-blind Herold Regimen, Double-blind Placebo, Open-label Herold Regimen

Primary outcome

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

15 secondary outcomes

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
Time frame: Month 6
C-peptide AUC at 12 Months
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
Mean Values for Participant-reported Outcomes on the 5-dimension European Quality of Life Questionnaire. (EQ-5D)
Time frame: Month 6
Mean Values for Participant-reported Outcomes on the EQ-5D
Time frame: Month 12
Pediatric Quality of Life Questionnaire Total Score
Time frame: Month 6
Pediatric Quality of Life Questionnaire Total Score
Time frame: Month 12
Percentage of Cells by Immunophenotype
Time frame: Month 6
Human Anti-human Antibody (HAHA) Levels
Time frame: Month 6 and 12

Results and safety

Results posted to the registry on 9 Aug 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Open-label Herold Regimen	32	0
Double-blind Herold Regimen	73	0
Double-blind 33.3% Herold Regimen	38	0
Double-blind Curtailed Herold Regimen	38	0
Double-blind Placebo	38	0

Primary outcome: 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. Count of participants. Unit: Participants

Measure	Open-label Herold Regimen n = 32	Double-blind Herold Regimen n = 73	Double-blind 33.3% Herold Regimen n = 38	Double-blind Curtailed Herold Regimen n = 38	Double-blind Placebo n = 38
Value	8	8	3	10	4

15 secondary outcomes are reported in the full results.

Adverse events

Time frame: Throughout the study, up to 15 months. Other adverse events reported at 2% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Open-label Herold Regimen	1 of 32 (3.1%)	8 of 32 (25%)	0 of 32
Double-blind Herold Regimen	5 of 73 (6.8%)	2 of 73 (2.7%)	1 of 73
Double-blind 33.3% Herold Regimen	3 of 38 (7.9%)	1 of 38 (2.6%)	0 of 38
Double-blind Curtailed Herold Regimen	4 of 38 (10.5%)	10 of 38 (26.3%)	0 of 38
Double-blind Placebo	0 of 38 (0%)	4 of 38 (10.5%)	0 of 38

Limitations and caveats (from the sponsor): No formal inferential analyses were conducted due to limited number of participants. Data are summarized as mean change from baseline, both from Protégé and Extension baselines, for C-peptide AUC, HbA1c, insulin, and fasting C-peptide. Only existing data are summarized, no last observation carried forward imputation was performed. Patient reported outcomes were collected and summarized as mean change from baseline. However, only change from baseline from Extension could be summarized.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 9 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Aug 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00870818.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Aug 2023.