Clinical trial NCT05757713: Teplizumab in Pediatric Stage 2 Type 1 Diabetes
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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE4
Conditions: Diabetes Mellitus, Type 1
Keywords: Type 1 diabetes
Stage 2
Pediatric type 1 diabetes
T1D
At-risk
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2023-03-07
Enrollment: 20
Sex: ALL
Minimum age (years): 0
Maximum age (years): 7
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single arm, non-randomized, open-label, multi-center study
Primary purpose: TREATMENT

Brief 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs)
timeFrame: Through 104 Weeks
Secondary outcomes:
measure: Serum concentrations of teplizumab
timeFrame: Sparse PK samples between Days 1 and 28
measure: Anti-drug antibody (ADA) titers and presence of neutralizing antibodies (Nab)
timeFrame: Through 104 weeks
measure: CD3 receptor occupancy
timeFrame: Days 1 and 9
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: UCSF Medical Center Site Number : 107
state: California
city: Aurora
country: United States
facility: Barbara Davis Center for Diabetes Site Number : 102
state: Colorado
city: New Haven
country: United States
facility: Yale University School of Medicine Site Number : 101
state: Connecticut
city: Columbus
country: United States
facility: Centricity Research Site Number : 104
state: Georgia
city: Indianapolis
country: United States
facility: Indianapolis University Health Riley Hospital for Children Site Number : 110
state: Indiana
city: Buffalo
country: United States
facility: UBMD Pediactrics Site Number : 105
state: New York
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia Site Number : 108
state: Pennsylvania
city: Sioux Falls
country: United States
facility: Sanford Diabetes and Thyroid Clinic Site Number : 106
state: South Dakota
city: Nashville
country: United States
facility: Vanderbilt Univerity Medical Center Site Number : 109
state: Tennessee
city: Tacoma
country: United States
facility: MulitCare Institute for Research & Innovation Site Number : 103
state: Washington
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: