PETITE-T1D: Teplizumab in Pediatric Stage 2 Type 1 Diabetes

• Sponsor: Provention Bio, Inc. (Industry)
• Overall Status: Not yet recruiting
• CT.gov ID: NCT05757713
• Collaborator: (none)
• Enrollment: 20
• Arm: 1
• Anticipated Duration (Months): 41

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Type 1 Diabetes	Biological: teplizumab	Phase 4

Detailed 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 and serologic response to vaccines and exploratory T1D clinical features will be explored.

Approximately 20 participants will be enrolled The regimen consists 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.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 20 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention Model Description: Single arm, non-randomized, open-label, multi-center studySingle arm, non-randomized, open-label, multi-center study
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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)
• Anticipated Study Start Date: May 1, 2023
• Anticipated Primary Completion Date: Oct 1, 2026
• Anticipated Study Completion Date: Oct 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: teplizumab injection teplizumab injection, sterile solution for intravenous use	Biological: teplizumab; CD3-directed humanized monoclonal antibody

Outcome Measures

Primary Outcome Measures

1. Frequency of TEAEs, AESIs, TEAEs leading to withdrawal, SAEs [104 Weeks]

   Safety Endpoint

2. Serum concentrations of teplizumab [104 weeks]

   Pharmacokinetics endpoint

Secondary Outcome Measures

1. Serum concentration of ADA anti-drug antibodies (anti-teplizumab antibodies in ng/mL) [104 weeks]

   Immunogenicity endpoint

2. Serum concentration of NAb anti-drug antibodies (anti-teplizumab neutralizing antibodies in ng/mL) [104 weeks]

   Immunogenticity endpoint

3. Serum concentration of CD3 receptor occupancy on T cells [104 weeks]

   Pharmacodynamic endpoint

Eligibility Criteria

Criteria

• Ages Eligible for Study: 0 Years to 7 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• Provention Bio, Inc.

Investigators

• Study Director: Provention Bio, Inc., MD, Provention Bio, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Provention Bio, Inc.
• ClinicalTrials.gov Identifier: NCT05757713
• Other Study ID Numbers: PRV-031-005
• First Posted: Mar 7, 2023
• Last Update Posted: Mar 8, 2023
• Last Verified: Mar 1, 2023

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Provention Bio, Inc.: Type 1 Diabetes; Stage 2 T1D; Pediatric T1D
• Additional relevant MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 1

Study Results

No Results Posted as of Mar 8, 2023