SLOW Start to Teplizumab Trial

I keep watching the Teplizumab Trial through Trialnet.  I'm watching because eventually we'll have to decide whether or not to enroll Kaitlyn.  That is, rather, if we have the opportunity to enroll before she is diagnosed.  The process is going so slowly, thanks to the FDA. 

First, let me explain the trial.  Teplizumab is an anti-CD3 drug that may stop the autoimmune attack and halt the disease progression.  For Kaitlyn, that would be fantastic.  For Andrew, it wouldn't do much.  It has been tested in various trials at varying strengths and has passed safety tests.  However, it has only been tested in newly diagnosed patients.  Most remained on insulin, but c-peptide tests show they do make more insulin than the control group.  The closer to diagnosis and younger the patient, the more dramatic the results.  This is the first trial to target the at-risk group like Kaitlyn is in.  If it was effective in halting her disease, she would never face type 1 diabetes!

Here is where the FDA comes in.  There is a normal protocal to test adults first, then teens, and then children in a clinical trial.  There is good logic in that idea, but it seems a ridiculous standard here.  This study is for people at risk for a disease that normally presents in childhood.  They are not allowed to give kids this drug, which has already been FDA approved to give to kids in other trials, until they find 10 adults who have antibodies, impaired glucose, but no diabetes.  Then they have to agree to the trial.  That is statistically very difficult!  Trialnet applied to the FDA to lower the enrollment age based on the fact that this drug has safely been administered to many children.  The answer:  No.  It is going to be a very long wait searching for ten adults still in process of getting a childhood disease who are willing to enroll in a study requiring daily infusions for two weeks.