Teplizumab for Pediatric Type 1 Diabetes

(PETITE-T1D Trial)

Not currently recruiting at 20 trial locations

Overseen By Trial Transparency email recommended (Toll free for US & Canada)

Age: < 18

Sex: Any

Trial Phase: Phase 4

Sponsor: Provention Bio, a Sanofi Company

Disqualifiers: Autoimmune diseases, Active infection, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 14

Duration: 2 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and metabolism of teplizumab, a medication for children with early-stage type 1 diabetes, a condition where the body attacks its own insulin-producing cells. It focuses on children under 8 years old who have specific diabetes-related markers and irregular blood sugar levels. Children with stable thyroid or celiac disease can participate, but those with other autoimmune diseases, active infections, or past infections like HIV or hepatitis cannot. As a Phase 4 trial, this research seeks to understand how this FDA-approved and effective treatment benefits more patients.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the study team for guidance.

What is the safety track record for teplizumab?

Research has shown that teplizumab has undergone thorough safety testing. In one study, about 873 side effects were linked to teplizumab, with 9.31% being serious, primarily affecting the immune system and causing stomach problems. However, most side effects are mild to moderate and typically resolve on their own.

Another study found that common side effects included headaches and stomach issues, mostly occurring around the time of medication administration. A specific reaction called cytokine release syndrome (CRS) occurred in about 5.8% of patients. This involves an immune system overreaction but is usually manageable.

The FDA reviewed safety data from several studies involving 773 patients, showing that teplizumab is generally well-tolerated. While some side effects can occur, they are usually not severe and can be managed. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Researchers are excited about teplizumab for pediatric type 1 diabetes because it offers a novel approach to managing the condition. Unlike standard treatments that primarily focus on managing blood sugar levels with insulin, teplizumab targets the immune system directly. It works by modulating T-cells, which are responsible for attacking insulin-producing cells in the pancreas. This unique mechanism may help preserve the body’s ability to produce insulin, potentially slowing disease progression and reducing reliance on insulin injections.

What is the effectiveness track record for teplizumab in treating type 1 diabetes?

Research has shown that teplizumab can help treat type 1 diabetes. One study found that two 12-day courses of teplizumab significantly improved the function of beta cells, which produce insulin, in people recently diagnosed with type 1 diabetes. Another study showed that children who received teplizumab retained more insulin-producing cells and often required lower insulin doses. Teplizumab is already approved to delay the onset of advanced type 1 diabetes in individuals aged 8 and older who are in the early stages of the disease. These findings suggest that teplizumab could be beneficial in managing type 1 diabetes, particularly in children. ^{2 4 6 7 8}

Who Is on the Research Team?

Clinical Sciences & Operations

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

This trial is for children under 8 years old with Stage 2 Type 1 Diabetes, which means they have certain diabetes-related antibodies and blood sugar issues. Kids can't join if they have other autoimmune diseases (except stable thyroid or celiac), infections like HIV/HBV/HCV, or any condition that might affect the study or their safety.

Inclusion Criteria

I am under 8 years old.

I have Stage 2 Type 1 Diabetes with certain autoantibodies and blood sugar issues.

Exclusion Criteria

You have any autoimmune disease other than type 1 diabetes, except for stable thyroid or celiac disease.

You have had HIV, hepatitis B, or hepatitis C in the past or currently have evidence of these infections.

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive an intravenous infusion of teplizumab once daily for 14 consecutive days

2 weeks

14 visits (in-person)

Follow-up

Participants are monitored for safety, tolerability, and pharmacokinetics, including ADA and NAb assessments

104 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Teplizumab

Trial Overview The trial is testing teplizumab's safety and how it's processed in the body (pharmacokinetics) in young kids with early-stage Type 1 Diabetes to see if it could help manage their condition.

How Is the Trial Designed?

Group I: teplizumab injection Experimental Treatment 1 Intervention

teplizumab injection, sterile solution for intravenous use

Who Is Running the Clinical Trial?

Provention Bio, a Sanofi Company

Lead Sponsor

Trials

5

Recruited

540+

Provention Bio, Inc.

Lead Sponsor

Trials

11

Recruited

1,300+

Published Research Related to This Trial

In the Phase III DEFEND-2 trial involving 179 patients (including 54 adolescents), otelixizumab did not significantly preserve C-peptide secretion in patients with new-onset Type 1 diabetes, showing a change from baseline that was not statistically significant (P=0.051).

The study found that the efficacy and tolerability of otelixizumab at a 3.1 mg dose were similar to previous results from DEFEND-1, indicating that this dose was ineffective for both adults and adolescents, and further research is needed to explore higher doses and their mechanisms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of low-dose otelixizumab anti-CD3 monoclonal antibody in preserving C-peptide secretion in adolescent type 1 diabetes: DEFEND-2, a randomized, placebo-controlled, double-blind, multi-centre study. Ambery, P., Donner, TW., Biswas, N., et al.[2018]

Citations

1. nejm.org nejm.org/doi/full/10.1056/NEJMoa2308743

Teplizumab and β-Cell Function in Newly Diagnosed Type ... The present trial showed that two 12-day courses of intravenous teplizumab significantly improved stimulated C-peptide levels, as compared with ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10769466/

Teplizumab in Type 1 Diabetes Mellitus: An Updated Review This article reviews the mechanism of action and the clinical trials of teplizumab in individuals with T1DM or at risk of developing the disease.

3. ucsf.edu ucsf.edu/news/2023/10/426396/breakthrough-drug-helps-children-new-onset-type-1-diabetes

Breakthrough Drug Helps Children with New-Onset Type 1 ... The teplizumab group of 217 patients preserved more of their remaining insulin-producing beta cells and trended toward needing lower doses of ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT03875729

NCT03875729 | Recent-Onset Type 1 Diabetes Trial ... The purpose of this study is to determine whether teplizumab slows the loss of β cells and preserves β cell function in children and adolescent 8-17 years old

5. diabetesjournals.org diabetesjournals.org/care/article/46/10/1848/153536/Teplizumab-A-Disease-Modifying-Therapy-for-Type-1

Teplizumab: A Disease-Modifying Therapy for Type 1 ... Teplizumab-mzwv became the first drug approved to delay the onset of stage 3 type 1 diabetes in adults and children age ≥8 years with stage 2 type 1 diabetes.

6. sciencedirect.com sciencedirect.com/science/article/pii/S0149291825003194

Assessment of Adverse Events of Teplizumab: A Real ... There are 873 adverse events (AEs) associated with teplizumab, with 9.31% serious AEs, primarily involving immune-related and gastrointestinal ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12416869/

Pediatric Endocrine Society Statement on Considerations for ... In addition to 44 persons randomized to teplizumab in TN-10, the FDA new drug application cited safety data from four stage 3 T1D clinical trials including 773 ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT05757713

Teplizumab in Pediatric Stage 2 Type 1 Diabetes (PETITE- ... 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.