Teplizumab for Pediatric Type 1 Diabetes

(PETITE-T1D Trial)

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

20 Participants Needed

Trial Summary

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 data supports the effectiveness of the drug Teplizumab for pediatric Type 1 Diabetes?

Teplizumab has been shown to delay or prevent the onset of Type 1 Diabetes by reducing the immune system's attack on insulin-producing cells in the pancreas. It is the first drug approved by the FDA for this purpose, with research indicating it can help maintain better glucose control in high-risk individuals. ^{1 2 3 4 5}

Is Teplizumab safe for children with Type 1 Diabetes?

Teplizumab has been reported to cause mild and short-term side effects in clinical trials for Type 1 Diabetes. ^{1 3 4 6 7}

How is the drug Teplizumab unique for treating pediatric type 1 diabetes?

Teplizumab is unique because it is the first drug approved to delay the onset of stage 3 type 1 diabetes in children and adults with stage 2 of the disease. It works by preventing the immune system's T cells from attacking the insulin-producing cells in the pancreas, which is different from traditional treatments that focus on managing blood sugar levels with insulin. ^{3 4 5 7 8}

What is the purpose of this trial?

This trial tests the safety of teplizumab, an IV medication, in children under 8 with early signs of type 1 diabetes. The drug aims to protect insulin-producing cells from immune system attacks and has shown potential in delaying the onset of type 1 diabetes.

Research Team

Clinical Sciences & Operations

Principal Investigator

Sanofi

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Teplizumab: promises and challenges of a recently approved monoclonal antibody for the prevention of type 1 diabetes or preservation of residual beta cell function. [2023]

2. Italy pubmed.ncbi.nlm.nih.gov

Prevention of type 1 diabetes: where we are and where we are going. [2022]

3. Germany pubmed.ncbi.nlm.nih.gov

Teplizumab: type 1 diabetes mellitus preventable? [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Teplizumab Therapy to Delay the Onset of Type 1 Diabetes. [2023]

5. England 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. [2018]

6. New Zealand pubmed.ncbi.nlm.nih.gov

Teplizumab: First Approval. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

Teplizumab: A Disease-Modifying Therapy for Type 1 Diabetes That Preserves β-Cell Function. [2023]

8. United States pubmed.ncbi.nlm.nih.gov

Teplizumab and β-Cell Function in Newly Diagnosed Type 1 Diabetes. [2023]