Teplizumab in Pediatric Stage 2 Type 1 Diabetes

Diabetes Mellitus, Type 1 Clinical Trial

— PETITE-T1D  

Official 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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05757713
• Other study ID #: PRV-031-005
• Status: Recruiting
• Phase: Phase 4
• Start date: July 25, 2023
• Est. completion date: October 2026

Study information

• Verified date: April 2024
• Source: Sanofi
• Contact: Trial Transparency email recommended (Toll free for US & Canada)
• Phone: 800-633-1610
• Email: Contact-us[@]sanofi.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: October 2026
• Est. primary completion date: October 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 7 Years

Eligibility

Inclusion Criteria:

1. Participant is a male or female 0 to < 8 years of age, inclusive, at Day 1

2. Participant has Stage 2 T1D (two T1D-related autoantibodies and dysglycemia)

Exclusion Criteria:

1. Participant has any autoimmune disease other than T1D with the exception of stable thyroid or celiac disease

2. Has an active infection and/or fever

3. 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).

4. Has any condition that, in the opinion of the Investigator, would interfere with the study conduct or the safety of the participant.

Related Conditions & MeSH terms

• Diabetes Mellitus
• Diabetes Mellitus, Type 1

Intervention

Biological:
• teplizumab
CD3-directed humanized monoclonal antibody

Locations

Country	Name	City	State
United States	Clinical Site 102	Aurora	Colorado
United States	Clinical Site 105	Buffalo	New York
United States	Clinical Site 104	Columbus	Georgia
United States	Clinical Site 110	Indianapolis	Indiana
United States	Clinical Site 109	Nashville	Tennessee
United States	Clinical Site 101	New Haven	Connecticut
United States	Clinical Site 108	Philadelphia	Pennsylvania
United States	Clinical Site 107	San Francisco	California
United States	Clinical Site 106	Sioux Falls	South Dakota
United States	Clinical Site 103	Tacoma	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Provention Bio, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs)	Safety and tolerability endpoint	Through 104 Weeks
Secondary	Serum concentrations of teplizumab	Pharmacokinetic (PK) endpoint	Sparse PK samples between Days 1 and 28
Secondary	Anti-drug antibody (ADA) titers and presence of neutralizing antibodies (Nab)	Immunogenicity endpoint	Through 104 weeks
Secondary	CD3 receptor occupancy	To assess the effects of teplizumab on cluster of differentiation 3 (CD3) receptor occupancy	Days 1 and 9