A Study to Investigate Efficacy and Safety of Teplizumab Compared With Placebo in Participants 1 to 25 Years of Age With Stage 3 Type 1 Diabetes

Purpose

This is a multicenter, randomized, double-blind, parallel, placebo-controlled Phase 3, 2-arm study for treatment. The purpose of this study is to measure change in glycemic control and prandial insulin independency over 52 weeks with teplizumab compared with placebo, both administered by intravenous (IV) infusion, in participants with recently diagnosed Stage 3 type 1 diabetes (T1D) aged 1 to 25 years, on standard insulin therapy.

Condition

  • Type 1 Diabetes Mellitus

Eligibility

Eligible Ages
Between 1 Year and 25 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Participants are eligible to be included in the study only if all of the following criteria apply: - Participant must be 1 to 25 years of age inclusive, at the time of signing the informed consent. - Participants diagnosed with T1D Stage 3 according to American Diabetes Association 2025 criteria - Participants able to be randomized and initiate study drug within 8 weeks (56 days) of the Stage 3 T1D diagnosis - Participants must be positive for at least one T1D autoantibody at screening: - Glutamic acid decarboxylase (GAD-65), - Insulinoma Antigen-2 (IA-2), - Zinc-transporter 8 (ZnT8), or - Insulin (if obtained not later than 14 days after exogenous insulin therapy initiation). - Islet cell cytoplasmic autoantibodies (ICAs) - Have random C-peptide level ≥0.2 nmol/L obtained at screening - Enter Inclusion Criteria Sex - Both male and female participants are eligible. - Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. - A female participant is eligible to participate if she is not pregnant, and one of the following conditions applies: - Is a woman of nonchildbearing potential (WONCBP) OR - Is a woman of childbearing potential (WOCBP) and agrees to use a contraceptive method that is highly effective, with a failure rate of <1% during the study intervention period (to be effective before starting the intervention) and for at least 30 days after the last administration of study intervention. - A WOCBP must have a negative highly sensitive pregnancy test at screening (serum) and within 24 hours (urine or serum as required by local regulations) before the first administration of study intervention. - Lactating woman must interrupt breastfeeding and pump and discard breast milk during and for 20 days after last administration of study intervention. - Capable of giving signed informed consent as described in Appendix 1 of the protocol which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. Note: For minor participants, a specific ICF must also be signed by the participant's legally authorized representative (LAR).

Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply: - Participant has diabetes other than autoimmune T1D that includes but is not limited to genetic forms of diabetes, maturity-onset diabetes of the young (MODY), diabetes secondary to medications or surgery and type 2 diabetes by judgement of the Investigator. - Participant has an active serious infection and/or fever ≥38.5°C (101.3°F) within the 48 hours prior to the first dose (except if localized skin infection), or has chronic, recurrent or opportunistic infectious disease. - At screening, participant has laboratory or clinical evidence of acute or clinically active infection with Epstein-Barr virus (EBV), cytomegalovirus (CMV). - At screening, participant has positive serology for human immunodeficiency virus (HIV), hepatitis B (HBV), or hepatitis C (HCV). - Participant has evidence of active or latent tuberculosis (TB) as documented by medical history and examination, chest X-rays (posterior anterior and lateral), and/or TB testing. Blood testing (eg, QuantiFERON® TB Gold test) is strongly preferred; if not available, any local approved TB test is allowed. - Has other autoimmune diseases, (eg, rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, multiple sclerosis, systemic lupus erythematosus etc), except clinically stable autoimmune thyroid disease, or controlled celiac disease (at discretion of Investigator). - Any clinically significant abnormality identified either in medical/surgical history or during screening evaluation (eg, physical examination, laboratory tests, vital signs), or any adverse event (AE) during screening period which, in the judgment of the investigator, would preclude safe completion of the study or constrains efficacy assessment. - Participant has recent or planned vaccinations as follows: - Live-attenuated (live) vaccines (eg, varicella, measles, mumps, rubella, cold-attenuated intranasal influenza vaccine, and smallpox) within the 8 weeks before first dose of the investigational medicinal product (IMP) or planned/required administration during treatment or up to 26 weeks after last IMP administration in any treatment course - Inactivated or mRNA vaccines within 2 weeks before the first dose of IMP or planned required administration during treatment or up to 6 weeks after last IMP administration in any treatment course. - Current or prior use (within 30 days before screening) of any anti-hyperglycemic agents other than insulin - Past (within 30 days prior to screening) or current administration of any treatment that is known to cause a significant, ongoing change in the course of T1D or immunologic status (including but not limited to oral, inhaled or systemically injected steroids with duration >14 days, adrenocorticotropic hormone, verapamil). - Past systemic immunosuppression medicine or immune modulatory biologic therapy (such as monoclonal antibodies), within 3 months or 5 half-lifes (whichever is longer) prior to dosing. - Current or prior (within 30 days before screening) use of any medication known to significantly influence glucose tolerance (eg, atypical antipsychotics, diphenylhydantoin, niacin). - Participant has previously received teplizumab or other anti-CD3 treatment. - Other medications not compatible or interfering with IMP at discretion of Investigator. - Current enrollment OR past participation in another investigational study in which an investigational intervention (eg, drug, vaccine, invasive device) was administered within the last 8 weeks or 5 half-lifes, whichever is longer, prior to screening. - Participant has any of the following laboratory parameters, at screening prior to first dose: - Lymphocyte count: <1000/µL, - Neutrophil count: <1500/µL, - Platelet count: <150,000 platelets/µL, - Hemoglobin: <10 g/dL, - Aspartate aminotransferase (AST) >2.0 × upper limit of normal (ULN), - Alanine aminotransferase (ALT) >2.0 × ULN, - Total bilirubin >1.5 × ULN with the exception of participants with the diagnosis of Gilbert's syndrome who may be eligible provided they have no other causes leading to hyperbilirubinemia The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Teplizumab
Participants will receive teplizumab in increasing doses by intravenous administration
  • Drug: Teplizumab
    Pharmaceutical form: Solution for injection Route of administration: Intravenous infusion
    Other names:
    • SAR446681
    • TZIELD
Placebo Comparator
Placebo
Participants will receive volume matching placebo doses to the Teplizumab arm by intravenous administration
  • Other: Placebo
    Pharmaceutical form: Solution for injection Route of administration: Intravenous infusion

Recruiting Locations

Scottsdale Clinical Trials- Site Number : 8400044
Scottsdale, Arizona 85260
Contact:
Lisette Partipilo
480-382-7909
lpartipilo@scottsdaletrials.com

Marvel Clinical Research- Site Number : 8400042
Huntington Beach, California 92647

University of California San Francisco - Mission Bay- Site Number : 8400011
San Francisco, California 94158
Contact:
Rebecca Wesch
844-813-8273
clinicalresearch@ucsf.edu

Barbara Davis Center for Childhood Diabetes- Site Number : 8400031
Aurora, Colorado 80045

Yale University School of Medicine- Site Number : 8400005
New Haven, Connecticut 06510

University of Florida College of Medicine- Site Number : 8400054
Gainesville, Florida 32610
Contact:
Michael Haller
352-294-5760
hallemj@peds.ufl.edu

Encore Medical Research - Hollywood- Site Number : 8400061
Hollywood, Florida 33024

University of South Florida- Site Number : 8400013
Tampa, Florida 33612
Contact:
Bethany Dunn
813-974-4886
eadunn3@usf.edu

AdventHealth Winter Park- Site Number : 8400033
Winter Park, Florida 32792

Atlanta Diabetes Associates- Site Number : 8400036
Atlanta, Georgia 30318
Contact:
Heidi Capria
404-357-0880
hcapria@atlantadiabetes.com

IACT Health - Columbus - Talbotton Road- Site Number : 8400003
Columbus, Georgia 31904
Contact:
Dr. Steven Leichter
706-996-2921
Sleichter@centricityresearch.com

Institute of Endocrinology Diabetes, Health & Hormones- Site Number : 8400050
Stockbridge, Georgia 30281
Contact:
Luis Molina
919-250-3320
luis.molina@sitebridgeresearch.com

St. Luke's Children's Hospital- Site Number : 8400014
Boise, Idaho 83712
Contact:
Xanti Gerrard
208-381-9433
gerrardx@slhs.org

Rocky Mountain Clinical Research - Idaho Falls- Site Number : 8400001
Idaho Falls, Idaho 83404
Contact:
Joshua Smith
208-525-3739
jms-research@idahomed.com

Norton Children's Research Institute - Wendy Novak Diabetes Institute- Site Number : 8400016
Louisville, Kentucky 40202

C.S. Mott Children's Hospital- Site Number : 8400015
Ann Arbor, Michigan 48109
Contact:
Aimee Katona
734-615-4079
PEDS-ENDO-DM-Research@med.umich.edu

Mayo Clinic in Rochester - Minnesota- Site Number : 8400019
Rochester, Minnesota 55905
Contact:
Yogish Kudva
507-284-3964
kudva.yogish@mayo.edu

Park Nicollet Institute - Oncology Research- Site Number : 8400059
Saint Louis Park, Minnesota 55426
Contact:
Landon Haber
952-993-3042
Landon.H.Haber@HealthPartners.com

Children's Mercy Adelle Hall Campus- Site Number : 8400010
Kansas City, Missouri 64108
Contact:
Angela Wiedner
816-460-1097
endoclinicaltrials@cmh.edu

University At Buffalo - Jacobs School Of Medicine- Site Number : 8400006
Buffalo, New York 14203

Endocrine Associates of West Village- Site Number : 8400064
New York, New York 10001
Contact:
Dr. Anastasios Manessis
516-574-9232
ymesen@endocrinenyc.com

Columbia University Irving Medical Center- Site Number : 8400009
New York, New York 10032

Asheville Clinical Research- Site Number : 8400045
Asheville, North Carolina 28803
Contact:
Lauren Elizabeth Bernstein
18285792273
bbernstein@ashevilleclinicaltrials.com

Cincinnati Children's Hospital Medical Center- Site Number : 8400018
Cincinnati, Ohio 45229
Contact:
Cierra Farrell
cierra.farrell@cchmc.org

The Ohio State University- Site Number : 8400051
Columbus, Ohio 43210
Contact:
Lindsey Aldrich
614-814-0772
lindsey.aldrich@osumc.edu

The Children's Hospital of Philadelphia- Site Number : 8400039
Philadelphia, Pennsylvania 19104

AM Diabetes & Endocrinology Center- Site Number : 8400004
Bartlett, Tennessee 38133

Vanderbilt University Medical Center- Site Number : 8400020
Nashville, Tennessee 37232

Baylor College of Medicine- Site Number : 8400057
Houston, Texas 77030

UVA Children's Battle Building- Site Number : 8400060
Charlottesville, Virginia 22903
Contact:
Christine Burt Solorzano
434-243-8236
car2r@uvahealth.org

Benaroya Research Institute at Virginia Mason- Site Number : 8400038
Seattle, Washington 98101
Contact:
Sandra Lord
206-341-8956
slord@benaroyaresearch.org

MultiCare Mary Bridge Children's Health Center- Site Number : 8400037
Tacoma, Washington 98405
Contact:
Rebekah Schaefer
253-403-0776
becky.schaefer@multicare.org

More Details

Status
Recruiting
Sponsor
Sanofi

Study Contact

Trial Transparency email recommended (Toll free for US & Canada)
800-633-1610
contact-us@sanofi.com

Detailed Description

The study duration for one participant will be approximately 84 weeks (18 months).