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Active, Not Recruiting

NCT Number: NCT05757713

Teplizumab in Pediatric Stage 2 Type 1 Diabetes

The purpose of this study is to assess the safety and pharmacokinetics (PK) of teplizumab in participants with Stage 2 type 1 diabetes who are <8 years of age.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Diabetes Mellitus, Type 1 Autoimmune Diseases Diabetes Mellitus Endocrine System Diseases Glucose Metabolism Disorders Immune System Diseases Metabolic Diseases Nutritional and Metabolic Diseases

Age range

0 year–7 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

UCSF Medical Center Site Number : 107, San Francisco, California, United States

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About this study

This is a single-arm, non-randomized, open-label, multicenter study in the U.S. to assess the safety and pharmacokinetics of teplizumab in pediatric participants with Stage 2 type 1 diabetes (T1D) (two T1D-related autoantibodies and dysglycemia) who are 0 to <8 years of age. Anti-drug antibodies/neutralizing antibodies (ADA/NAb) will also be assessed.

Approximately 20 participants will be enrolled. The regimen consists of an intravenous infusion of teplizumab once daily for 14 consecutive days. The study duration for each individual may last up to approximately 26 months.

The safety, tolerability, PK, ADA, and T1D clinical feature data will be summarized using descriptive statistics.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participant is a male or female 0 to < 8 years of age, inclusive, at Day 1
  • Participant has Stage 2 T1D (two T1D-related autoantibodies and dysglycemia)

Exclusion criteria

  • Participant has any autoimmune disease other than T1D with the exception of stable thyroid or celiac disease
  • Has an active infection and/or fever
  • Has a history of or serologic evidence at screening of current or past infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV).
  • Has any condition that, in the opinion of the Investigator, would interfere with the study conduct or the safety of the participant.

Treatment and study plan

Teplizumab

Biological

CD3-directed humanized monoclonal antibody

Primary outcomes

  1. Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs)

    Time frame: Through 104 Weeks

    Safety and tolerability endpoint

Secondary outcomes

  1. Serum concentrations of teplizumab

    Time frame: Sparse PK samples between Days 1 and 28

    Pharmacokinetic (PK) endpoint

  2. Anti-drug antibody (ADA) titers and presence of neutralizing antibodies (Nab)

    Time frame: Through 104 weeks

    Immunogenicity endpoint

  3. CD3 receptor occupancy

    Time frame: Days 1 and 9

    To assess the effects of teplizumab on cluster of differentiation 3 (CD3) receptor occupancy

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

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)

Acronym: PETITE-T1D

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Mar 7, 2023
Registry last updated
Jan 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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