Investigator- and subject-blinded, randomized, placebo-controlled study to evaluate safety, tolerability, pharmacokinetics and efficacy of CFZ533 in pediatric and young adults with new onset type 1 diabetes mellitus (T1DM)

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Description

1st treatment of study drug or placebo given by IV infusion Weekly subcutaneous injections of study drug or placebo for 1 year (52 weeks) Completion of a study diary to record insulin dose, and hypoglycemic events CGM measurements 8 mixed-meal tolerance tests (MMTTs): a test where the participant drinks high protein drink without giving insulin coverage. Blood samples are then collected from an IV over time to monitor glucose levels and the participant’s natural ability to still produce insulin.

Details
Age

Child to Adult

Eligibility

Ages 6-21 years old. Body weight 20 to 125 Able to receive first dose of study drug within 56 days of type 1 diabetes diagnosis Meet ADA criteria for Type 1 Diabetes; have 1 genetic marker for diabetes (islet autoantibody) Have a certain amount of C-peptide protein in blood, which indicates some natural insulin production

Written informed consent, and if needed assent from the child on the trial, must be obtained before any assessment is performed. 2. Males and females aged between 6 and 21 years (inclusive, and enrolled in stages) at screening. 3. Body weight range from 20 to 125 kg (inclusive). 4. 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. 5. 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). 6. 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). 7. 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 (see Sections on Risks Section 4.5 and Screening procedures Section 8.1). 8. Must be willing to comply with the standard of care for diabetes management. 9. A negative pregnancy test at screening is required for all sexually mature female subjects prior to participation in the study. 10. Subject and/or guardian must be able to communicate well with the investigator, to understand and comply with the requirements of the study.

Type of Study

Treatment

Locations

Barbara Davis Center
Childrens Hospital Colorado
University of Colorado Hospital

Principal Investigator
Photograph of Kimberly Simmons,  MD, MPH/MSPH

Kimberly Simmons, MD, MPH/MSPH

Study ID

Protocol Number: 22-0191

More information available at ClinicalTrials.gov: NCT04129528

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