Diabetes Mellitus, Type 1Phase 4PETITE-T1DNCT05757713
Teplizumab in Pediatric Stage 2 Type 1 Diabetes
A Phase 4 study testing teplizumab in people with Diabetes Mellitus, Type 1, run by Sanofi at 10 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 0 to 7 years
- Conditions the study is for
- Diabetes Mellitus, Type 1
- What is being tested?
- teplizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 10 sitesUnited States
- Study dates
- 25 Jul 2023to 28 Aug 2026
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participant is a male or female 0 to < 8 years of age, inclusive, at Day 1
- Participant has Stage 2 T1D (two T1D-related autoantibodies and dysglycemia)
Who cannot join
- Participant has any autoimmune disease other than T1D with the exception of stable thyroid or celiac disease
- Has an active infection and/or fever
- 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).
- Has any condition that, in the opinion of the Investigator, would interfere with the study conduct or the safety of the participant.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Barbara Davis Center for Diabetes Site Number : 102
Aurora, Colorado, 80045
UBMD Pediactrics Site Number : 105
Buffalo, New York, 14203
Centricity Research Site Number : 104
Columbus, Georgia, 31904
Indianapolis University Health Riley Hospital for Children Site Number : 110
Indianapolis, Indiana, 46202
Vanderbilt Univerity Medical Center Site Number : 109
Nashville, Tennessee, 37232
Yale University School of Medicine Site Number : 101
New Haven, Connecticut, 06511
Children's Hospital of Philadelphia Site Number : 108
Philadelphia, Pennsylvania, 19104
UCSF Medical Center Site Number : 107
San Francisco, California, 94143
Sanford Diabetes and Thyroid Clinic Site Number : 106
Sioux Falls, South Dakota, 57105
MulitCare Institute for Research & Innovation Site Number : 103
Tacoma, Washington, 98405
About this study
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)
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.
Read the 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.
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.
- Conditions
- Diabetes Mellitus, Type 1
- Being tested
- teplizumab
- Keywords
- Type 1 diabetes, Stage 2, Pediatric type 1 diabetes, T1D, At-risk
- Medical subject headings
- Diabetes Mellitus, Type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
teplizumabBiological
CD3-directed humanized monoclonal antibody
| Study group | Role | What they receive |
|---|---|---|
| teplizumab injection | Experimental | teplizumab injection, sterile solution for intravenous use Receives: Biological: teplizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs)
Safety and tolerability endpoint
Measured: Through 104 Weeks
Secondary outcomes (3)
Serum concentrations of teplizumab
Pharmacokinetic (PK) endpoint
Measured: Sparse PK samples between Days 1 and 28
Anti-drug antibody (ADA) titers and presence of neutralizing antibodies (Nab)
Immunogenicity endpoint
Measured: Through 104 weeks
CD3 receptor occupancy
To assess the effects of teplizumab on cluster of differentiation 3 (CD3) receptor occupancy
Measured: Days 1 and 9
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2023
- Study start25 Jul 2023
- Primary completion28 Aug 2026
- Study completion28 Aug 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Diabetes Mellitus, Type 1Studies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single arm, non-randomized, open-label, multi-center study
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05757713
- Sponsor's protocol ID
- PRV-031-005
- Other IDs
- SFY18116, 2024-000581-19
- Acronym
- PETITE-T1D
- Status last verified
- Sep 2026
- First submitted
- 13 Feb 2023
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
Gitelman SE, Simmons K, Sherr JL, Leichter SB, Quattrin T, Russell WE, Sunil B, Willi SM, Knecht LA, Niemoeller E, Licaj I, Schmider W, Miller D, DiMeglio LA. Safety and pharmacokinetics of teplizumab in children less than 8 years of age with stage 2 type 1 diabetes. Diabetologia. 2026 Feb;69(2):330-342. doi: 10.1007/s00125-025-06586-1. Epub 2025 Nov 6.
PubMed 41196293Novograd J, Frishman WH. Teplizumab Therapy to Delay the Onset of Type 1 Diabetes. Cardiol Rev. 2024 Nov-Dec 01;32(6):572-576. doi: 10.1097/CRD.0000000000000563. Epub 2023 May 9.
PubMed 37158990
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05757713
- Acronym / sponsor ID
- PETITE-T1D / PRV-031-005
- Conditions
- Diabetes Mellitus, Type 1
- Keywords
- Type 1 diabetes, Stage 2, Pediatric type 1 diabetes, T1D, At-risk
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 25 Jul 2023 (actual)
- Primary completion
- 28 Aug 2026 (actual)
- Study completion
- 28 Aug 2026 (actual)
- First posted
- 7 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single arm, non-randomized, open-label, multi-center study
| Arm | Type | Receives |
|---|---|---|
| teplizumab injection | Experimental | Biological: teplizumabteplizumab injection, sterile solution for intravenous use |
Who may qualify
- Ages
- 0 to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Participant is a male or female 0 to < 8 years of age, inclusive, at Day 1
- Participant has Stage 2 T1D (two T1D-related autoantibodies and dysglycemia)
Key exclusion criteria (4 listed)
- Participant has any autoimmune disease other than T1D with the exception of stable thyroid or celiac disease
- Has an active infection and/or fever
- 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).
- Has any condition that, in the opinion of the Investigator, would interfere with the study conduct or the safety of the participant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| teplizumabCD3-directed humanized monoclonal antibody | Biological | teplizumab injection |
Primary outcome
Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs)
Time frame: Through 104 Weeks
Safety and tolerability endpoint
3 secondary outcomes
Serum concentrations of teplizumab
Time frame: Sparse PK samples between Days 1 and 28
Anti-drug antibody (ADA) titers and presence of neutralizing antibodies (Nab)
Time frame: Through 104 weeks
CD3 receptor occupancy
Time frame: Days 1 and 9
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2026.
- Derived from this study: PubMed 41196293Gitelman SE, Simmons K, Sherr JL, Leichter SB, Quattrin T, Russell WE, Sunil B, Willi SM, Knecht LA, Niemoeller E, Licaj I, Schmider W, Miller D, DiMeglio LA. Safety and pharmacokinetics of teplizumab in children less than 8 years of age with stage 2 type 1 diabetes. Diabetologia. 2026 Feb;69(2):330-342. doi: 10.1007/s00125-025-06586-1. Epub 2025 Nov 6.
- Derived from this study: PubMed 37158990Novograd J, Frishman WH. Teplizumab Therapy to Delay the Onset of Type 1 Diabetes. Cardiol Rev. 2024 Nov-Dec 01;32(6):572-576. doi: 10.1097/CRD.0000000000000563. Epub 2023 May 9.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing teplizumab in people with Diabetes Mellitus, Type 1, run by Sanofi at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs) (time frame: Through 104 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05757713, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.