Health Research

Cellcept in the Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (Batten Disease) (JUMP)

Basic Study Information

Is Cellcept (mycophenolate) safe and well tolerated in children with JNCL? Does Cellcept affect the symptoms of JNCL?
CellCept is a medication that suppresses the immune system and is approved by the US Food and Drug Administration (FDA) to prevent rejection of transplanted organs in both children and adults, but is not approved for use in treating Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) (Batten Disease) and therefore is considered an investigational drug forBatten Disease. If you decide to allow your child to participate in this study, your child will be asked to take study drug. The drug will be in the form of a syrup. The study syrup will be given for two, eight-week periods. In between these two periods, there will be a single four-week period when he/she will not take study syrup. For one of the two eight week periods, the study syrup will be CellCept. For the other eight weeks, the syrup will be a placebo (matching syrup without any medication in it). Both syrups will look and smell identical. The study personnel will not know which eight weeks your child is receiving CellCept and which eight weeks your child is receiving placebo. You will not know this either. The study syrup is a liquid and your child will need to be able to swallow it. The study syrup will be taken twice a day. The study has ten study visits. Four of these visits will take place at the University of Rochester Medical Center. Four will take place at your child's local doctor's office, that is, in/near your home town. Two visits will take place via telephone. The study team will work with you to identify a local doctor (either a neurologist or primary care doctor) who will be responsible for conducting the local visits.The subjects have to be able to walk 10 feet unsupported (walker is ok.)
University of Rochester

Lead Researcher (Principal Investigator)

Frederick Marshall

Study Contact Information

Amy Vierhile
(585) 275-4762

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Additional Study Information

For the visits at the University of Rochester Medical Center, your airfare or mileage will be paid by the research group, as well as that of your child. You will also be provided with hotel accommodations for up to 2 nights at each visit at no cost to you.
Six to Ten
We are reimbursing for meals related to the visits (i.e. meals during travel to and from our site and meals while you are here).