December 18, 2013
Workshop on JNCL Clinical Trials Outcomes
There are a number of recent and ongoing clinical trials in Batten Disease. Most of these are focused on asking if these potential new treatments are safe. After safety studies are completed, the next stage of clinical trials research is to learn if these or other interventions are effective. In order to answer this question, we will need a way to measure whether or not an intervention actually makes a meaningful difference in disease course.
Therefore, the URBC hosted a workshop titled, Outcome Measures and Infrastructure for Phase III Studies in JNCL (December 6-7, 2013). The workshop brought together experts in the clinical features of JNCL, experts from other fields (clinical trials in rare diseases, statistics, etc.), and Batten family representatives. Each expert was invited to bring trainee to accompany him/her, so that we can continue cultivating the next generation of researchers who are focused on Batten Disease. Our goals were to:
- Identify possible outcome measures for future clinical trials
- Develop a roadmap for further research to test and refine outcome measures
- Strengthen research collaborations to support outcome measure development
November 1, 2013
The University of Rochester Medical Center is currently recruiting subjects with JNCL for a clinical trial. This research study will focus on evaluating whether an investigational drug is safe and well tolerated in children with JNCL. Mycophenolate mofetil (also known as Cellcept) is a medication that suppresses the immune system. The study is 22 weeks long with a total of 8 in-person visits and 4 telephone contacts. Four visits require travel to University of Rochester Medical Center in Rochester, New York, and four visits are with your child’s local physician. Four contacts take place by telephone. Travel costs are covered by the study. Children enrolled in the study will take mycophenolate syrup twice a day, and will have blood drawn at each study visit to monitor safety.
For further information, please contact Amy Vierhile at (585) 275-4762.
Support for this conference was provided by:
The National Institutes of Health – National Institute of Neurological Diseases and Stroke (NIH/NINDS): 1R13 NS078981,
Outcomes and Infrastructure for Phase III Clinical Trials in JNCL - Batten Disease
The University of Rochester Child Neurology Research Fund