Interim Report on the world’s first Double Blind Clinical Trial using the drug Sirolimus (Rapamycin) presented to LAM patients at the April 2010 LAM Conference held in Cincinnati USA

19 April 2010

MlLES Trial Interim Results The Data Safety Monitoring Board (DSMB) recently completed their review of an early interim analysis of study data for the MlLES Trial. The review included study data from the first forty MlLES Trial participants who have completed their first year in the study.

The DSMB recommends that the MlLES Trial continue through August, 2010 until all actively-enrolled study participants have completed their 12-month study visit. During this time, the MlLES study staff and participants will remain “blinded” to their treatment group (that is, they will not know who is on placebo and who is on sirolimus).

After August, 2010, the final analysis will be conducted. At that time more information will be available regarding the effectiveness and safety of sirolimus in treating LAM. All study staff and participants will then be “unblinded” to individual participant treatment assignments.


What is an interim analysis and why was it conducted at this point? 
An interim analysis is an early look at the data collected from study participants and is done before the study is completed. The study statistician and the DSMB look at the data together for safety and treatment results. For MILES, the DSMB recommended an interim analysis so that LAM patients might have a shorter time to wait for an answer about the study results.

What does the DSMB review mean to me as a study participant? 
It means that all aspects of the study have been examined by a group of scientists and physicians who are experts in clinical trials and or LAM. Unlike the study investigators, they know who is on the study drug and who is not. They look at the data and determine if it is already clear that sirolimus is effective or ineffective, and if it is productive and safe for you to continue participating in the study. The DSMB concluded that not enough information is available at this halfway point to make a decision about whether sirolimus is an effective treatment for LAM and that the trial should continue as originally planned. This is a very common DSMB recommendation at the interim point.

Does my participation make a difference in the MILES Trial at this point? 
The data that will be collected from now through August will be very important in deciding if sirolimus is an effective and safe treatment for LAM. Not enough is known at this point to decide this key study question.

When will I find out what I have been taking on the study? 
You will find out what your treatment has been after the final analysis has been completed. We anticipate that this will be in late 2010 or early 2011 when all of the data has been analyzed.