NPTUNE Trial for SMA Type I
Last year, Children's Hospital Boston was selected to be a site for a collaborative NINDS (National Institute of Neurological Disorders and Stroke) trial for SMA. Over the last 6 months we have worked hard to get the necessary approvals in order to begin conducting this study. We are happy to report that we now have approval for this study, which is a study aimed at finding the safest dose of sodium phenylbutyrate. This study is not designed to determine if this medication is effective at treating symptoms of SMA or not. Here in Boston we will be enrolling patients with SMA type I who are: 1) between 2-24 months of age, 2) weigh over 7kg, 3) are on respiratory support less than 16 hours a day. Currently there is an administrative hold on enrollment nationally but we expect this to change soon. If you are interested in more information about this study please contact the study coordinator, Hailly Butler at 857-218-4677 or Hailly.Butler@childrens.harvard.edu
PNCR Clinical Trials
The PNCR Network hopes to offer clinical trials in the near future. We are sad to report that our application for funding for these clinical trials last year was denied. However, we are making the changes to address the concerns of the NIH, and plan to resubmit our request next month.
Project Cure Clinical Trials
Please visit Project Cure directly for up to date information about their research.
Stanford University Clinical Trials
AmSMART Clinical Trials
Please visit AmSMART directly for up to date information about their research.
|
