Evidence map›Paper›PMID 41196293›Full record

ArticleDiabetologia2026

Safety and pharmacokinetics of teplizumab in children less than 8 years of age with stage 2 type 1 diabetes.

Stephen E Gitelman, Kimber Simmons, Jennifer L Sherr, Steven B Leichter, Teresa Quattrin, William E Russell, Bhuvana Sunil, Steven M Willi, Laura A Knecht, Elisabeth Niemoeller and 4 more

Registry-linked trialAbstract readMulticenter Study
In one paragraph

Article in Diabetologia, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05757713 (Single Arm, Open-label Study to Assess the Safety and Pharmacokinetics of a 14-day Regimen of Teplizumab in Pediatric Stage 2 Type 1 Diabetes), which is not on this map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT05757713 phase4active not recruitingnot on this map

Single Arm, Open-label Study to Assess the Safety and Pharmacokinetics of a 14-day Regimen of Teplizumab in Pediatric Stage 2 Type 1 Diabetes (Participants <8 Years of Age With at Least Two Autoantibodies and Dysglycemia)

TypeinterventionalSponsorSanofiRan2023 to 2026Enrolled20ConditionsDiabetes Mellitus, Type 1Armsteplizumab
3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Article
  2. Article
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  5. Article
  6. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

14 authors.

Stephen E GitelmanDepartment of Pediatrics, University of California at San Francisco, San Francisco, CA, USA. Stephen.Gitelman@ucsf.edu.ORCID http://orcid.org/0000-0003-4186-4107
Kimber SimmonsBarbara Davis Center for Diabetes/University of Colorado School of Medicine, Aurora, CO, USA.ORCID http://orcid.org/0000-0003-0560-5773
Jennifer L SherrDepartment of Pediatrics, Pediatric Endocrinology, Yale University School of Medicine, New Haven, CT, USA.ORCID http://orcid.org/0000-0001-9301-3043
Steven B LeichterPiedmont Endocrinology Columbus, Columbus, GA, USA.ORCID http://orcid.org/0000-0002-0784-7605
Teresa QuattrinDepartment of Pediatrics, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, Buffalo, NY, USA.ORCID http://orcid.org/0000-0002-2385-9278
William E RussellDepartment of Pediatrics, Vanderbilt University Medical Center, Nashville, TN, USA.ORCID http://orcid.org/0000-0001-7609-0434
Bhuvana SunilMary Bridge Children's Hospital, Tacoma, WA, USA.
Steven M WilliDivision of Endocrinology and Diabetes, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0002-4910-2425
Laura A KnechtSanofi, Morristown, NJ, USA.
Elisabeth NiemoellerSanofi, Frankfurt, Germany.
Idlir LicajSanofi, Paris, France.ORCID http://orcid.org/0000-0003-3389-0735
Wolfgang SchmiderSanofi, Frankfurt, Germany.
Diana MillerSanofi, Morristown, NJ, USA.
Linda A DiMeglioDivision of Pediatric Endocrinology and Diabetology, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.ORCID http://orcid.org/0000-0002-8033-6078

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

aims/hypothesisTeplizumab is approved in the USA and seven other countries to delay stage 3 type 1 diabetes onset in individuals ≥8 years of age with stage 2 type 1 diabetes. As part of a US Food and Drug Administration post-marketing requirement, this study evaluated the safety, tolerability and pharmacokinetics of teplizumab in children aged <8 years with stage 2 type 1 diabetes.

methodsThe PETITE-T1D trial is a 2 year single-arm, open-label, multicentre study of 23 children <8 years of age with stage 2 type 1 diabetes. Participants received a 14 day teplizumab course. A prespecified interim analysis was performed after 15 participants completed 1 year of follow-up and included all 23 participants. Primary endpoints included treatment-emergent adverse events (TEAEs), TEAEs causing treatment discontinuation, and serious adverse events (SAEs). Other endpoints assessed immunogenicity, pharmacokinetics, pharmacodynamics and time from study treatment to stage 3 type 1 diabetes.

resultsMean participant age was 4.8 years (range 1.7-6.8). Median follow-up duration was 51.9 weeks (range 3.9-77.1). All participants experienced one or more TEAE, with most being mild to moderate. No grade 4 or 5 TEAEs were reported. Three participants (13%) had TEAEs leading to teplizumab discontinuation: anaemia, elevated liver enzymes and maculo-papular rash. Two participants (9%) each had two SAEs. Serum teplizumab concentrations peaked at day 14. Two participants progressed to stage 3 type 1 diabetes. The estimated probability of lack of progression to stage 3 was 89.6% (95% CI 64.3%, 97.3%) at the time of interim analysis. CONCLUSIONS/

interpretationTeplizumab was safe and well tolerated in children <8 years of age with stage 2 type 1 diabetes. Adverse events were consistent with those seen in previous studies, with no new safety risks identified. Two participants progressed to stage 3 type 1 diabetes during the observation period; surveillance is ongoing.

trial registrationClinicalTrials.gov NCT05757713.

Indexed as

Antibodies, Monoclonal, HumanizedDiabetes Mellitus, Type 1ChildChild, PreschoolFemaleHumansMaleAntibodies, Monoclonal, HumanizedteplizumabChildDisease progressionImmunogenicityPaediatricsPharmacodynamicsPharmacokineticsPharmacologySafetyTeplizumabType 1 diabetes

Identifiers

PMID41196293
PMCPMC12779737

What Socratic holds

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LicenceCC BY
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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.