Recent-Onset Type 1 Diabetes Extension Study Evaluating the Long-Term Safety of Teplizumab (PROTECT Extension)

A Multicenter, Multinational Extension of Study PRV-031-001 to Evaluate the Long-Term Safety of Teplizumab (PRV-031), a Humanized, FcR Non-Binding, Anti-CD3 Monoclonal Antibody, in Children and Adolescents With Recent-Onset Type 1 Diabetes Mellitus

The purpose of this non-interventional extension study is to continue to collect long-term safety and other clinical data for an additional 42 months in participants who completed the PROTECT study.

Study Overview

• Status: Active, not recruiting
• Conditions: Diabetes Mellitus, Type 1
• Intervention / Treatment: Biological: Teplizumab; Other: Placebo

Detailed Description

This study is the long-term extension of the PRV-031-001 (PROTECT) study. PROTECT is a Phase 3, randomized, double-blind, placebo-controlled, multinational, multi-center study to evaluate the efficacy and safety of teplizumab, a humanized, anti-CD3 monoclonal antibody, in children and adolescents ages 8 through 17 recently diagnosed with type 1 diabetes (within 6 weeks of diagnosis) in which approximately 300 participants were randomized at a ratio of 2:1 to the teplizumab or placebo and received two courses of treatment administered 6 or 12 months apart with final follow-up at approximately 18 months (78 weeks) after the first dose.

Participants who complete the PROTECT study are invited to enroll in this observational, extension study to collect long-term safety and other clinical data. The decision to participate can be made within 12 months of completing the PROTECT study.

No study drug will be administered during this extension study. Participants will continue to receive standard care for type 1 diabetes from their primary physicians.

Participants will return to the study sites for assessments once every 6 months through Month 42. Therefore, the combined duration of the PROTECT and PROTECT Extension studies will be 60 months.

• Study Type: Observational
• Enrollment (Actual): 188

Contacts and Locations

Study Locations

• Belgium
  • Brussels Capital
    • Jette, Brussels Capital, Belgium, B-1090
      • University Hospital Brussels (Site 056202)
  • Namur
    • Namur, Namur, Belgium, B-5000
      • UCL Namur University Hospital Place Louise Godin 15 (Site 056205)
• Canada
  • Alberta
    • Edmundston, Alberta, Canada, T6G 2E1
      • Alberta Diabetes Institute, 2-004 Li Ka Shing Centre for Health Research Innovation 8602 (Site 124103)
  • British Columbia
    • Vancouver, British Columbia, Canada, V5Y 3W2
      • BCDiabetes - Medical Research Center, 210 West Broadway Suite 400 (Site 124102)
• Czechia
  • Prague, Czechia, 150 06
    • University Hospital Motol V Uvalu 84, Praha 5 - Motol (Site 203301)
• France
  • Bron, France, 69677
    • Woman Mother Child Hospital HCL
  • Dijon, France, 21079
    • Dijon University Hospital Center-Francois Mitterand Hospital, Children's Hospital
  • Marseille, France, 13385
    • CHU La Timone - La Timone Children's Hospital
  • Nice, France, 06200
    • Lenval Hospital, 57 Avenue de la Californie (Site 250508)
  • Paris, France, 75015
    • Necker Children's Hospital, 149 Rue de Sevres (Site 250502)
  • Pau, France, 64046
    • Pau Hospital Center
  • France
    • Orléans, France, France, 45067
      • Hospital Center Regional D'Orleans Hospital La Source, Pediatric Department, 14 Hospital Ave Post box 86709 (Site 250513)
• Germany
  • Augsburg, Germany, 86156
    • Hospital Augsburg, Stenglinstrasse 2 (Site 276606)
  • Dresden, Germany, 01307
    • University Hospital Carl Gustav Carus Fetscherstrasse 74 (Site 276601)
  • Hanover, Germany, 30173
    • Pediatric Hospital on the Bult Janusz-Korczak-Allee 12 (Site 276604)
  • Heidelberg, Germany, 69120
    • University Hospital Heidelberg, Im Neuenheimer Feld 430 (Site 276608)
  • Bielefeld
    • Bethel, Bielefeld, Germany, 33617
      • Evangelic Clinic Bethel, Children's Clinic Grenzweg 14/Hs 2 (Site 276602)
• Poland
  • Gdansk, Poland, 80-952
    • University Teaching Centre, Department of Pediatrics, Diabetology and Endocrinology (Site 616803)
  • Warsaw, Poland, 02-117
    • Institute of Diabetology ul. Raclawicka 129/2U, 02-117 (Site 616802)
  • Warsaw, Poland, 04-730
    • Children's Memorial Health Institute Al. Dzieci Polskich 20 (Site 616801)
  • Poland
    • Warsaw, Poland, Poland, 02-091
      • University Teaching Centre of the Medical University of Warsaw, 63A, ul. Zwirki i Wigur (Site 616804)
• United Kingdom
  • Yorkshire
    • Sheffield, Yorkshire, United Kingdom
      • Sheffield Children's Hospital Western Bank, S10 2TH (Site 826903)
• United States
  • California
    • San Diego, California, United States, 92123
      • Rady Children's Hospital 3020 Children's Way (Site 840004)
    • San Francisco, California, United States, 94158
      • UCSF Medical Center Gateway Medical Building, 1825 Fourth Street (Site 840001)
    • Walnut Creek, California, United States, 945981
      • Diablo Clinical Research2255 Ygnacio Valley Road, Ste. M (Site 840002)
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Barbara Davis Center for Diabetes - Pediatrics 1775 Aurora Court (Site 840005)
  • Florida
    • Jacksonville, Florida, United States, 32207
      • Nemours Children's Health, 807 Children's Way
    • St. Petersburg, Florida, United States, 33701
      • Johns Hopkins All Children's Hospital, 501 6th Avenue South (Site 840048)
  • Georgia
    • Atlanta, Georgia, United States, 30318
      • Atlanta Diabetes Associates 1800 Howell Mill Road. Suite 450 (Site 840009)
    • Columbus, Georgia, United States, 31904
      • Centricity Research (Site 840006)
  • Idaho
    • Boise, Idaho, United States, 83712
      • St. Luke's Children's Endocrinology, 305 E Jefferson St. (Site 840052)
    • Idaho Falls, Idaho, United States, 83404
      • Rocky Mountain Clinical Research, LLC 3910 Washington Parkway, Suite E (Site 840007)
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University Hospital, Indiana Clinical Research Center, 550 North University Boulevard (Site 840014)
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics 200 Hawkins Drive (Site 840023)
  • Maryland
    • Camp Springs, Maryland, United States, 20746
      • Capital Medical Research Associates (Site 840029)
  • Massachusetts
    • Springfield, Massachusetts, United States, 01199
      • Baystate Medical Center, 759 Chestnut Street
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Hospitals and Clinics, 2401 Gillham Road (Site 840026)
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine, Pediatric Clinical Research Unit, Suite 11W19 (Site 840018)
  • New York
    • Buffalo, New York, United States, 14203
      • UBMD Pediatrics 1001 Main Street, 4th Floor (Site 840010)
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • UNC Hospitals, The University of North Carolina at Chapel Hill, Children's Specialty Clinic, 101 Manning Drive (Site 840038)
  • Oregon
    • Bend, Oregon, United States, 97702
      • Endocrinology Service Northwest, LLC 929 SW Simpson Ave. Suite 220 (Site 840034)
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia, Division of Endocrinology 3500 Civic Center Blvd., Buerger Center 12th Floor (Site 840021)
  • Tennessee
    • Bartlett, Tennessee, United States, 38133
      • AM Diabetes & Endocrinology Center, 3025 Kate Bond Rd. (Site 840008)
    • Nashville, Tennessee, United States, 37212
      • Vanderbilt University Medical Center 1500 21st Avenue, Suite 1514, Village At Vanderbilt (Site 840024)
  • Texas
    • Dallas, Texas, United States, 75235
      • UT Southwestern Children's Medical Center of Dallas 1935 Medical District Drive (Site 840033)
  • Washington
    • Seattle, Washington, United States, 98101
      • Virginia Mason Medical Center, Benaroya Research Institute 1201 9th Ave, MS: D4-CRP (Site 840016)
    • Tacoma, Washington, United States, 98405
      • MultiCare Health System, Gateway Medical Building (Site 840003)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 9 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants who completed the PROTECT (PRV-031-001) study

Description

Inclusion Criteria:

1. Completion of the PRV-031-001 (PROTECT) study by completing the Week 78 visit in that study, regardless of how many doses of study drug were administered.
2. Provide written informed consent within 12 months of the Week 78 in the PRV-031-001 study. Written assent will be obtained for participants under 18 years of age at the time of enrollment, according to applicable regulations. If possible, written informed consent and/or assent for the current PROTECT Extension study (PRV-031-003) should be obtained at the final (Week 78) visit in the PROTECT study.

Exclusion Criteria:

1. Premature discontinuation from the PRV-031-001 (PROTECT) study for any reason.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Teplizumab; Participants who received teplizumab in the PROTECT study	Biological: Teplizumab; Received teplizumab in PROTECT Study
Placebo; Participants who received placebo in the PROTECT study	Other: Placebo; Received placebo in PROTECT study

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs), including infections and malignancies	Safety outcome	During 42 months of follow-up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Area under the time-versus concentration curve (AUC) of C-peptide after a 4-hour (4h) mixed-meal tolerance test (MMTT)	Clinical parameters of diabetes management 1	During the 42 months of follow-up
Insulin use (daily average insulin dose in U/kg/day)	Clinical parameters of diabetes management 2	During the 42 months of follow-up
HbA1c	Clinical parameters of diabetes management 3	During the 42 months of follow-up

Collaborators and Investigators

• Sponsor: Provention Bio, a Sanofi Company
• Investigators: Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): October 26, 2020
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026

Study Registration Dates

• First Submitted: October 16, 2020
• First Submitted That Met QC Criteria: October 16, 2020
• First Posted (Actual): October 22, 2020

Study Record Updates

• Last Update Posted (Estimated): December 9, 2025
• Last Update Submitted That Met QC Criteria: December 2, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Type 1 diabetes; T1D; Teplizumab; New onset type 1 diabetes; Stage 3; Recent-onset type 1 diabetes
• Additional Relevant MeSH Terms: Endocrine System Diseases; Metabolic Diseases; Autoimmune Diseases; Immune System Diseases; Glucose Metabolism Disorders; Diabetes Mellitus; Nutritional and Metabolic Diseases; Diabetes Mellitus, Type 1; teplizumab
• Other Study ID Numbers: PRV-031-003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No