Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects

Inclusion Criteria

• Written informed consent, and if needed assent from the child on the trial, must be obtained before any assessment is performed.
• Males and females aged between 12 and 21 years (inclusive, and enrolled in stages) at screening.
• Body weight range from 30 to 125 kg (inclusive).
• Evidence of one or more type 1 diabetes autoantibody(ies) against: glutamic acid decarboxylase (anti-GAD), protein tyrosine, phosphatase-like protein (anti-IA-2); zinc transporter 8 (anti-ZnT8); islet cell (cytoplasmic) (anti-ICA) at screening or baseline in the central laboratory OR historical clinical record of one or more of the T1DM diabetes autoantibodies. As part of the historical record insulin autoantibodies (IAA) may have been used as part of the autoantibody panel but the blood sample must have been obtained prior to or within one week of starting exogenous insulin treatment.
• Able to receive first dose of study drug within 56 days of diagnosis of T1DM (which may be extended to within 100 days of diagnosis in the event a screening assessment needs to be confirmed or vaccine administered).
• Peak stimulated C-peptide levels ≥0.2 nmol/L (0.6 ng/mL) following standard liquid mixed meal tolerance test (MMTT), to be conducted when the subject is metabolically stable, at least 2 weeks from diagnosis and within 56 days prior to randomization (or within 100 days of diagnosis in the event a screening assessment needs to be confirmed or vaccine is required).
• Study participants are to complete all recommended immunizations with live, attenuated vaccine at least eight weeks prior and killed, inactivated vaccine at least two weeks prior to first dose with study drug and in accordance with local immunization guidelines. In the event a subject has not had all vaccinations recommended according to local guidance, the screening period may be extended beyond 56 days to allow these vaccinations to be administered, but first dose of study drug must be administered within 100 days of diagnosis of T1DM.
• Must be willing to comply with the standard of care for diabetes management.
• A negative pregnancy test at screening is required for all sexually mature female subjects prior to participation in the study.
• Subject and/or guardian must be able to communicate well with the investigator, to understand and comply with the requirements of the study.

Exclusion Criteria

• Diabetes forms other than auto immune type 1 such as maturity-onset diabetes of the young (MODY), latent autoimmune diabetes of the adult (LADA), acquired diabetes (secondary to medications or surgery), type 2 diabetes by judgement of the investigator.
• Diabetic ketoacidosis within 2 weeks of the baseline MMTT test.
• Polyglandular auto immune disease, Addison's disease, pernicious anemia, celiac sprue. Note: Investigators are not mandated to test for Celiac disease (also known as Sprue). Subjects suspected of having Celiac disease should be tested for the presence of disease, as part of good medical care, as treatment would differ. Treated, stable Hashimoto's thyroiditis is not exclusionary.
• Any of the following abnormal laboratory values at screening: total white blood cell count (WBC) outside the range of 1,500-15,000/mm3 (1.5-15.0 x 109/L), neutrophil count (<1500/mm3) (<1.5 X 109 / L), lymphocyte count <500/mm3 (<0.5 X 109 / L), hemoglobin (Hgb) <8.0 g/dL, platelets <100,000/mm3 (<100 x 109/L) 5. History of immunodeficiency disorders, such as HyperIgM syndrome; history of recurrent infections suggestive of immunodeficiency disorders.
• History of or active coagulation disorder with increased thromboembolic risk; a PTT and PT/ INR below lower limit of normal prior to inclusion.
• Tuberculosis infection assessed by positive QuantiFERON TB-Gold test (QFT) at screening. Subjects with a positive QFT test may participate in the study if further work up (according to local practice/guidelines) establishes conclusively that the subject has no evidence