NCT00129259

Brief Summary

Anti-CD3 monoclonal antibody (a.k.a. hOKT3gamma1 \[Ala-Ala\],teplizumab, MGA031) is a humanized antibody that is commonly used to prevent organ rejection. The purpose of this study is determine whether anti-CD3 mAb treatment can halt the progression of newly diagnosed type 1 diabetes.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
83

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Sep 2005

Longer than P75 for phase_2

Geographic Reach
1 country

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 9, 2005

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 11, 2005

Completed
21 days until next milestone

Study Start

First participant enrolled

September 1, 2005

Completed
5.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2011

Completed
2.5 years until next milestone

Results Posted

Study results publicly available

September 16, 2013

Completed
Last Updated

May 22, 2017

Status Verified

April 1, 2017

Enrollment Period

5.5 years

First QC Date

August 9, 2005

Results QC Date

June 21, 2013

Last Update Submit

April 11, 2017

Conditions

Keywords

T1Dtype 1 diabetestype 1 diabetes mellitusjuvenile diabetesautoimmune diabetesautoimmuneanti-CD3 mAbmAb hOKT3g1(Ala-Ala)teplizumab

Outcome Measures

Primary Outcomes (1)

  • Change in Mean C-peptide Area Under the Curve (AUC) Response to a Mixed Meal Tolerance Test (MMTT)

    C-peptide AUC is computed using the trapezoidal rule and dividing by the interval of time from the 4 hour Mixed Meal Tolerance Test (MMTT) where assessments are taken every 30 minutes after initial assessments 15 minutes apart. A higher C-peptide AUC is desirable as detectable C-peptide is a marker for the ability of the pancreas to produce insulin in response to a MMTT. The baseline data was used to adjust for the C-peptide AUC primary endpoint at 24 months. Missing month 24 C-peptide results are imputed using a conservative scenario.

    Baseline (Pre-treatment), Month 24

Secondary Outcomes (2)

  • Change in HbA1c

    Baseline (Pre-treatment), Month 24

  • Change in Average Total Insulin Dose Per Body Weight

    Baseline (Pre-treatment), Month 24

Study Arms (2)

Anti-CD3 mAb Plus Diabetes Standard of Care Treatment

EXPERIMENTAL

Subjects receive 1.) a 14-day course of anti-CD3 monoclonal antibody (mAb) intravenously (IV) comprised of daily doses of 51 µg/m2, 103 µg/m2, 207 µg/m2, 413 µg/m2, and 10 days of 826 µg/m2 \[Cycle 1\] and, when eligible per protocol, receipt of a second 14-day course after a 12-month interval (at month 13)\[Cycle 2\]. Note: Prior to May 2007, the course of IV daily doses of anti-CD3 mAb were: 57 µg/m2, 115 µg/m2, 230 µg/m2, 460 µg/m2, and 10 days of 919 µg/m2 and, when eligible per protocol, a second course after a 12-month interval (at month 13). 2.) and intensive diabetes standard of care treatment/management under the care of a physician: dietary counseling, insulin dosing and multiple consultations during the course of the trial with the clinical diabetes management team. Iron supplementation initiated status post treatment randomization.

Biological: Anti-CD3 mAbOther: Diabetes Standard of Care TreatmentDietary Supplement: Iron supplementation

Diabetes Standard of Care Treatment

ACTIVE COMPARATOR

Subjects receive intensive diabetes standard of care treatment/management under the care of a physician: dietary counseling, insulin dosing and multiple consultations during the course of the trial with the clinical diabetes management team. Iron supplementation initiated status post treatment randomization.

Other: Diabetes Standard of Care TreatmentDietary Supplement: Iron supplementation

Interventions

Anti-CD3 mAbBIOLOGICAL

Daily 14-day dose escalation course at study entry, with possible second course after 12-month interval

Also known as: mAb hOKT3gamma1(Ala-Ala), teplizumab, MGA031
Anti-CD3 mAb Plus Diabetes Standard of Care Treatment

Receipt of intensive diabetes standard of care treatment/management under the care of a physician: dietary counseling, insulin dosing and multiple consultations during the course of the trial with the clinical diabetes management team.

Anti-CD3 mAb Plus Diabetes Standard of Care TreatmentDiabetes Standard of Care Treatment
Iron supplementationDIETARY_SUPPLEMENT

Immediately following randomization, all participants regardless of arm allocation begin iron supplementation with either ferrous sulfate or multivitamin with iron.

Also known as: MVI, Fer-In-Sol, Feosol, Fer-Iron, Ferolix, FeroSul, Irospan
Anti-CD3 mAb Plus Diabetes Standard of Care TreatmentDiabetes Standard of Care Treatment

Eligibility Criteria

Age8 Years - 30 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of type 1 diabetes (according to American Diabetes Association \[ADA\] criteria) within the 8 weeks prior to study entry
  • Weigh at least 25 kg (55 lbs)
  • Insulin autoantibodies assessed within 10 days of any insulin use OR anti-glutamic acid decarboxylase (GAD) autoantibodies OR anti-ICA512/IA-2 autoantibodies
  • Subjects or guardian(s) willing to provide informed consent

You may not qualify if:

  • Prior participation in a clinical trial that could potentially affect diabetes condition or immunologic status
  • Participation in another investigational clinical trial within the 6 weeks prior to study entry
  • Pregnancy or breastfeeding

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

The Diabetes Center at UCSF

San Francisco, California, 94143, United States

Location

Barbara Davis Center for Childhood Diabetes

Denver, Colorado, 80262, United States

Location

Yale University

New Haven, Connecticut, 06520, United States

Location

Medical College of Georgia

Augusta, Georgia, 30912, United States

Location

Dept. of Medicine, Division of Endocrinology and the Naomi Berrie Diabetes Center/Columbia University

New York, New York, 10032, United States

Location

Benaroya Research Institute

Seattle, Washington, 98108, United States

Location

Pacific Northwest Research Institute/University of Washington

Seattle, Washington, 98122, United States

Location

Related Publications (7)

  • Herold KC, Gitelman SE, Masharani U, Hagopian W, Bisikirska B, Donaldson D, Rother K, Diamond B, Harlan DM, Bluestone JA. A single course of anti-CD3 monoclonal antibody hOKT3gamma1(Ala-Ala) results in improvement in C-peptide responses and clinical parameters for at least 2 years after onset of type 1 diabetes. Diabetes. 2005 Jun;54(6):1763-9. doi: 10.2337/diabetes.54.6.1763.

    PMID: 15919798BACKGROUND
  • Herold KC, Hagopian W, Auger JA, Poumian-Ruiz E, Taylor L, Donaldson D, Gitelman SE, Harlan DM, Xu D, Zivin RA, Bluestone JA. Anti-CD3 monoclonal antibody in new-onset type 1 diabetes mellitus. N Engl J Med. 2002 May 30;346(22):1692-8. doi: 10.1056/NEJMoa012864.

    PMID: 12037148BACKGROUND
  • Herold KC, Gitelman SE, Ehlers MR, Gottlieb PA, Greenbaum CJ, Hagopian W, Boyle KD, Keyes-Elstein L, Aggarwal S, Phippard D, Sayre PH, McNamara J, Bluestone JA; AbATE Study Team. Teplizumab (anti-CD3 mAb) treatment preserves C-peptide responses in patients with new-onset type 1 diabetes in a randomized controlled trial: metabolic and immunologic features at baseline identify a subgroup of responders. Diabetes. 2013 Nov;62(11):3766-74. doi: 10.2337/db13-0345. Epub 2013 Jul 8.

  • Flammer ER, Higdon LE, Sanda S, Garrett TJ, Ismail HM. Baseline Serum Metabolites as Predictors of Teplizumab Response in Individuals with Type 1 Diabetes. medRxiv [Preprint]. 2025 Dec 23:2025.12.22.25342822. doi: 10.64898/2025.12.22.25342822.

  • Ajmal N, Bogart MC, Khan P, Max-Harry IM, Healy AM, Nunemaker CS. Identifying Promising Immunomodulators for Type 1 Diabetes (T1D) and Islet Transplantation. J Diabetes Res. 2024 Dec 20;2024:5151171. doi: 10.1155/jdr/5151171. eCollection 2024.

  • Boyle KD, Keyes-Elstein L, Ehlers MR, McNamara J, Rigby MR, Gitelman SE, Weiner LJ, Much KL, Herold KC. Two- and Four-Hour Tests Differ in Capture of C-Peptide Responses to a Mixed Meal in Type 1 Diabetes. Diabetes Care. 2016 Jun;39(6):e76-8. doi: 10.2337/dc15-2077. Epub 2016 Apr 13. No abstract available.

  • Sherr JL, Ghazi T, Wurtz A, Rink L, Herold KC. Characterization of residual beta cell function in long-standing type 1 diabetes. Diabetes Metab Res Rev. 2014 Feb;30(2):154-62. doi: 10.1002/dmrr.2478.

Related Links

MeSH Terms

Conditions

Diabetes Mellitus, Type 1

Interventions

teplizumabferrous sulfateIron-Dextran ComplexFIT1 protein, Arabidopsis

Condition Hierarchy (Ancestors)

Diabetes MellitusGlucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

Coordination ComplexesOrganic ChemicalsDextransGlucansPolysaccharidesCarbohydrates

Results Point of Contact

Title
Director, Clinical Research Operations Program
Organization
DAIT/NIAID

Study Officials

  • Kevan Herold, MD

    Yale University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Masking Details
The staff at Northwest Lipid Research Laboratory were masked to the treatment assignment since they performed the mixed-meal tolerance test (MMTT) and the HgA1C assays. All study staff including the PI were masked to the results of the MMTT. They were notified of a detectable or undetectable fasting C-peptide result to assess for continuation to the next treatment cycle.
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 9, 2005

First Posted

August 11, 2005

Study Start

September 1, 2005

Primary Completion

March 1, 2011

Study Completion

March 1, 2011

Last Updated

May 22, 2017

Results First Posted

September 16, 2013

Record last verified: 2017-04

Data Sharing

IPD Sharing
Will share

Participant level data and additional relevant materials are available to the public in: 1.) the Immunology Database and Analysis Portal (ImmPort), a long-term archive of clinical and mechanistic data from Division of Allergy, Immunology, and Transplantation (DAIT)-funded grants and contracts that also provides data analysis tools available to researchers; and 2.) TrialShare, the Immune Tolerance Network (ITN) Clinical Trials Research Portal that makes data from the consortium's clinical trials publicly available.

Available IPD Datasets

Individual Participant Data Set (SDY524)Access
Study summary, -design (and protocol synopsis), adverse events, medications, demographics, lab tests, study files. (SDY524)Access
Individual Participant Data Set (AbaTE - ITN027AI)Access
Study overview and synopsis, -data and reports, -schedule of assessments, -participant flow diagram, -abstracts and manuscripts (AbaTE-ITN027AI)Access

Locations