What is the PROTECT Study?
The PROTECT Study will assess how well an investigational medicine, called teplizumab, works in children and adolescents recently diagnosed with Type 1 diabetes (T1D). ‘Investigational’ means that the medicine has not been approved for the treatment of T1D and is still being evaluated in clinical studies such as this one. The investigational medicine will be compared with a placebo – a substance that looks like teplizumab but contains no real treatment. The PROTECT Study will also assess whether there are any side effects from taking the investigational medicine
Who can take part?
Approximately 300 children and adolescents in study clinics located across the United States, Canada and Europe will take part in the PROTECT Study. Not everyone will be able to join the study as several inclusion criteria must be met.
Eligibility to take part includes:
- Age 8-17 years old
- Diagnosed with T1D in the previous 6 weeks
- Positive for 1 of 5 T1D autoantibodies (test to be done as part of the study)
- Ability to produce a minimum amount of your own insulin (test to be done as part of the study)
- Otherwise generally healthy, with no other significant medical conditions, recent or history of infections or taking medicines that might interfere with teplizumab
Which study medicine will I receive?
If you or your child are eligible to take part in the PROTECT Study, you or your child will be placed into one of two study treatment groups. Two thirds of participants will be assigned to the teplizumab group, and one third will be assigned to the placebo group. This is decided at random, like drawing straws. Neither you, your child, nor the study doctor will know to which study treatment group you or your child have been assigned. This ensures that the results from the different groups will be handled in the same way.
How often will I take the study medicine?
You or your child will receive two courses of study medicine over the duration of the study. Both the investigational medicine and placebo are given by intravenous (IV) infusion. This means a needle will be inserted into a vein, usually in the arm, and the liquid medication supplied continuously.
In this study, the IV infusion is given daily for a period of 12 days on two separate occasions, 6 months apart; the infusions will usually last for about 30 minutes. Further details of how and when to take the investigational medicine or placebo will be given by the study team.
How long will the study last?
Participation in the study will last up to 84 weeks (a little over a year and a half) and comprise a maximum of 36 study clinic visits. The study is divided into the following:
- Screening Visit (needed to assess ability to participate in the study) — you or your child will undergo tests and assessments to see whether you or your child are able to participate in the study.
- First treatment course (study start) — you or your child will receive the investigational medicine for 12 days in a row.
- Observation period (after the first treatment course to Week 26) — there will be about four visits to assess how you or your child are doing.
- Second treatment course (starting Week 26) — you or your child will receive the investigational medicine for 12 days in a row.
- Observation period (after the second treatment course to Week 78) — there will be about six visits to assess how you or your child are doing.
What happens during visits to the study clinic?
During visits to the study clinic, the study team will ask you or your child several questions and perform a number of study-related medical tests. The purpose of these tests is to assess you or your child’s health and determine how you or your child’s body is responding to the investigational medicine.
The following tests and assessments will occur at some point during the study:
- You or your child will be asked questions about your medical history, and current and previous medications you or your child have been or are taking.
- You or your child will complete questionnaires about how you or your child are feeling and how T1D has affected or is affecting your or your child’s life.
- You or your child will undergo a physical examination.
- You or your child will have your blood pressure, pulse, body temperature, height and weight measured.
- You or your child will be asked to give blood and urine samples.
- You or your child will be given the investigational medicine (or a placebo).