Phase II study of CFZ533 in paeds and young adults with new onset T1DM
Research type
Research Study
Full title
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)
IRAS ID
271290
Contact name
Tabitha Randel
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2018-004553-25
Clinicaltrials.gov Identifier
Duration of Study in the UK
6 years, 3 months, 23 days
Research summary
The purpose of the is study is to determine if CFZ533 is safe, tolerable and can preserve pancreatic beta-cell function in children and young adults newly diagnosed with type 1 diabetes mellitus (T1DM). The study will also look to see how long the effects of the study drug will last and disease remission.
Insulin is produced by the body to control blood sugar levels. T1DM occurs when the body cannot produce enough insulin because the body’s own immune system has attacked the insulin-producing beta cells in the pancreas (it is an auto-immune disease). T1DM has serious short-and long-term health complications, can result in reduced life-expectancy and there is no cure. Currently, the only therapy is control of blood sugar levels with life-long insulin injections. Most cases of T1DM start between the age of 5 and 15 years and disease progression is often fast. As such, there is high unmet clinical need.
This study is investigating CFZ533 which acts to block the immune cells attacking the pancreatic beta-cells. By administering the CFZ533 soon after T1DM onset, it may be possible to slow or stop the autoimmune attack on the beta cells and so preserve insulin production. Many lines of laboratory and clinical evidence support the study of CFZ533 in T1DM. This would be the first study to give CFZ533 to T1DM patient and the first to give it to children. To date, CFZ533 has been administered to several hundred patients with other autoimmune diseases and transplant patients. The safety data from these studies supports the use of CFZ533 in children and young adults with T1DM.
The study includes a screening/baseline period (8 weeks), treatment period (52 weeks) and a follow-up period (4 months - 2 years). Eligible patients will be randomly assigned to receive CFZ533 or placebo (2:1 ratio); this will be double blind so no one knows which treatment has been allocated. Assessments will include things such as: physical, examinations, blood and urine samples, study diaries, continuous glucose monitoring, tests to see how well the body deals with a meal.REC name
Wales REC 3
REC reference
21/WA/0003
Date of REC Opinion
15 Feb 2021
REC opinion
Further Information Favourable Opinion