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).
- Alder Hey, Liverpool
- Nottingham Children’s Hospital
- Birmingham Children’s Hospital
- St James University Hospital, Leeds
Clinical study of a new medication called CFZ533 (or iscalimab) in children and young adults aged 12-21 years with newly diagnosed Type 1 diabetes.
By administering CFZ533 soon after T1DM starts, it is thought that it may be possible to slow or stop destruction of the remaining insulin-producing beta cells in the pancreas, and so preserve remaining insulin production.
To investigate CFZ533 in terms of safety and effectiveness in preserving residual pancreatic beta cell function in children and young adults who have recently been diagnosed with T1DM.
Eligibility to take part
The research team will check eligibility carefully before enroling a participant in to the study. Some of the main criteria are listed below:
- Age 12-21 years
- Body weight range 20-125 Kg
- Able to have first dose of study medication within 56 days of inital T1DM diagnosis (may be extended to 100 days in specific circumstances)
If you are interested in taking part, please click the “Get Involved” button below:
What will I be asked to do?
Adult participants will be asked to provide their informed consent before starting the study. Where a participant is under 16 years, they will be asked to provide assent, and their parent/guardian will be asked to provide informed consent on the participant’s behalf.
The study has an initial screening and baseline period where the doctors check that a participant is eligible, and collect some initial data. The treatment period (when study drug is taken) comes after this and will last for around a year, with hospital visits every 4 weeks. After the treatment period is over, no more study drug is taken but some further follow-up visits at the hospital will happen (follow-up will last for up to 2 years after finishing treatment) – these visits will be monthly for the first 4 months and then every 6 months.
Participants in the study will get either CFZ533 or a dummy drug (also known as ‘placebo’) and this will be ‘blinded’ which means that no one knows if its CFZ533 or placebo (though this can be found out in an emergency). It will not be possible to choose between CFZ533 and placebo as this will be decided at random (by chance) by a special computer program. The first dose will be given by intravenous injection, then all other doses will be done by subcutaneous injection. Participants can continue to take their regular insulin therapy throughout the study.
Assessments during the study 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.
Who is running this study?
This study is being organised and funded by Novartis Pharma AG.