Teplizumab for Type 1 Diabetes

(βETA PRESERVE Trial)

No longer recruiting at 5 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Must be taking: Insulin

Must not be taking: Steroids, Antipsychotics

Disqualifiers: Type 2 diabetes, Active infection, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called teplizumab to determine if it can help people with Stage 3 type 1 diabetes manage blood sugar levels better and reduce insulin dependence. Participants will receive either teplizumab or a placebo through an IV to compare effects. The study seeks individuals diagnosed with Stage 3 type 1 diabetes within the last 8 weeks who are already using insulin.

As a Phase 3 trial, this study represents the final step before FDA approval, offering participants an opportunity to contribute to a potentially groundbreaking treatment for diabetes.

Do I have to stop taking my current medications for the trial?

The trial requires that you stop using any anti-hyperglycemic agents other than insulin and any medications that significantly influence glucose tolerance or the course of type 1 diabetes. If you're on such medications, you may need to stop them before joining the trial.

Is there any evidence suggesting that teplizumab is likely to be safe for humans?

Research has shown that teplizumab is generally well-tolerated. In previous studies, most side effects were mild to moderate and resolved on their own. About 5.8% of participants experienced cytokine release syndrome (CRS), which can cause fever and tiredness. Other common side effects included headaches and stomach issues. Importantly, these effects were similar in those who received a placebo. Long-term data indicate that teplizumab does not increase infection rates over time. This suggests that teplizumab is safe for treating type 1 diabetes, especially since it has been studied for similar conditions. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Teplizumab is unique because it targets the autoimmune process that causes Type 1 Diabetes, potentially preserving insulin-producing beta cells. Most treatments for Type 1 Diabetes focus on managing blood sugar levels with insulin injections. However, Teplizumab works by modulating the immune system to delay the onset of full-blown diabetes in individuals at high risk. Researchers are excited about this approach because it addresses the root cause of the disease rather than just its symptoms, offering hope for a longer-term solution.

What evidence suggests that teplizumab might be an effective treatment for type 1 diabetes?

Research has shown that teplizumab can help individuals with new-onset type 1 diabetes by preserving their insulin production. This preservation may reduce the need for insulin injections to manage blood sugar. In this trial, participants will receive either teplizumab or a placebo. Studies have found that teplizumab significantly improved levels of C-peptide, a marker of insulin production. Additionally, teplizumab reduced insulin requirements and improved blood sugar control, as evidenced by better HbA1c levels, which measure long-term blood sugar management. Another benefit is that it caused minimal side effects in patients. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for young people aged 1-25 with newly diagnosed Stage 3 Type 1 Diabetes. They must have certain levels of C-peptide, be positive for at least one diabetes-related autoantibody, and meet contraceptive requirements if applicable. Exclusions include serious infections, other types of diabetes, abnormal lab results, recent participation in another study or use of certain medications.

Inclusion Criteria

Were you diagnosed with type 1 diabetes in the last month?

Are you currently taking insulin?

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive teplizumab or placebo by intravenous infusion to measure change in glycemic control and prandial insulin independency

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

32 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Teplizumab

Trial Overview The trial tests Teplizumab's effectiveness against a placebo in improving blood sugar control and reducing the need for mealtime insulin over a year. Participants will receive either Teplizumab or placebo through IV infusion alongside standard insulin therapy in this double-blind study where neither they nor the researchers know who gets which treatment.

How Is the Trial Designed?

Group I: Teplizumab Experimental Treatment 1 Intervention

Participants will receive teplizumab in increasing doses by intravenous administration

Group II: Placebo Placebo Group 1 Intervention

Participants will receive volume matching placebo doses to the Teplizumab arm by intravenous administration

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

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

Teplizumab in Type 1 Diabetes Mellitus: An Updated Review The findings indicate that the drug might preserve insulin synthesis and decrease the need for exogenous insulin in new-onset T1DM. Phase III ...

2. 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 ...

3. 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 ... In November 2022, teplizumab-mzwv became the first drug approved to delay the onset of stage 3 type 1 diabetes in adults and children age ≥8 ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S1043661824002871

From pre-clinical efficacy to promising clinical trials that ... The teplizumab infusion resulted in a delay of progression to Stage 3 T1D onset by a median of 24 months and improved β-cell function compared to placebo [29].

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11292331/

Evaluation of teplizumab's efficacy and safety in treatment ... In type 1 diabetics, teplizumab decreased insulin consumption, improved C-peptide response, and significantly changed HbA1c levels with negligible side effects.

6. trialnet.org trialnet.org/our-research/completed-studies/teplizumab

Teplizumab Prevention Study | Type 1 Diabetes ... 72% of people in the control group developed clinical diabetes, compared to only 43% of the teplizumab group. The median time for people in the control group to ...

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

A Real-world Pharmacovigilance Study Based on the FDA ... Data support the safety of teplizumab in stage 2 type 1 diabetes mellitus when used as indicated. ... Although previous clinical trials have ...