BACKGROUND: Type 1 diabetes is an autoimmune disease characterized by 
progressive loss of pancreatic beta cells. Golimumab is a human monoclonal 
antibody specific for tumor necrosis factor α that has already been approved for 
the treatment of several autoimmune conditions in adults and children. Whether 
golimumab could preserve beta-cell function in youth with newly diagnosed overt 
(stage 3) type 1 diabetes is unknown.
METHODS: In this phase 2, multicenter, placebo-controlled, double-blind, 
parallel-group trial, we randomly assigned, in a 2:1 ratio, children and young 
adults (age range, 6 to 21 years) with newly diagnosed overt type 1 diabetes to 
receive subcutaneous golimumab or placebo for 52 weeks. The primary end point 
was endogenous insulin production, as assessed according to the area under the 
concentration-time curve for C-peptide level in response to a 4-hour mixed-meal 
tolerance test (4-hour C-peptide AUC) at week 52. Secondary and additional end 
points included insulin use, the glycated hemoglobin level, the number of 
hypoglycemic events, the ratio of fasting proinsulin to C-peptide over time, and 
response profile.
RESULTS: A total of 84 participants underwent randomization - 56 were assigned 
to the golimumab group and 28 to the placebo group. The mean (±SD) 4-hour 
C-peptide AUC at week 52 differed significantly between the golimumab group and 
the placebo group (0.64±0.42 pmol per milliliter vs. 0.43±0.39 pmol per 
milliliter, P<0.001). A treat-to-target approach led to good glycemic control in 
both groups, and there was no significant difference between the groups in 
glycated hemoglobin level. Insulin use was lower with golimumab than with 
placebo. A partial-remission response (defined as an insulin dose-adjusted 
glycated hemoglobin level score [calculated as the glycated hemoglobin level 
plus 4 times the insulin dose] of ≤9) was observed in 43% of participants in the 
golimumab group and in 7% of those in the placebo group (difference, 36 
percentage points; 95% CI, 22 to 55). The mean number of hypoglycemic events did 
not differ between the trial groups. Hypoglycemic events that were recorded as 
adverse events at the discretion of investigators were reported in 13 
participants (23%) in the golimumab group and in 2 (7%) of those in the placebo 
group. Antibodies to golimumab were detected in 30 participants who received the 
drug; 29 had antibody titers lower than 1:1000, of whom 12 had positive results 
for neutralizing antibodies.
CONCLUSIONS: Among children and young adults with newly diagnosed overt type 1 
diabetes, golimumab resulted in better endogenous insulin production and less 
exogenous insulin use than placebo. (Funded by Janssen Research and Development; 
T1GER ClinicalTrials.gov number, NCT02846545.).