List of Batten Disease Clinical Trial Postings



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Clinical Trial for Children with Juvenile Neuronal Ceroid Lipofuscinosis

Last Updated 2011-07-26 at 11:06:19

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 12 study visits. Four visits require travel to University of Rochester Medical Center in Rochester, New York. Four visits will be with your child’s local physician. Four visits will take place by telephone. Travel costs will be 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.

For further information, please contact Amy Vierhile at (585) 275‐4762.

Below is a pdf brochure with more details, for you to download



Infantile and Late Infantile Batten disease - CLN1 & CLN2 - stem cells

Last Updated 2010-05-23 at 15:46:06
StemCells, Inc. (NASDAQ: STEM) announced today that it has submitted a protocol to the FDA for initiation of a second clinical trial of its proprietary HuCNS-SC human neural stem cells in neuronal ceroid lipofuscinosis (NCL), which is also often referred to as Batten disease.

Late Infantile/LINCL/CLN2 - Gene therapy

Last Updated 2010-05-23 at 15:45:13
The aim of this study is to treat the signs and symptoms of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal inherited disease in the brain. This will be accomplished by using delivery of a gene (method called gene transfer) to administer to the brain an experimental drug called AAV2CUhCLN2, a gene transfer vector.

Late Infantile Batten disease/LINCL/CLN2

Last Updated 2010-05-23 at 15:43:22
In a separate protocol the Department of Genetic Medicine is proposing to carry out a study using gene transfer to treat the central nervous system (CNS) manifestations of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal, rare, recessive disorder of the (CNS) in children. In the context that there is little known about the genotype - phenotype correlations of LINCL, and that our referral far exceed the number (n=11) of children that will be entered into the gene transfer protocol, we are proposing to capitalize on this unique opportunity to evaluate this disorder in this separate study. In this context, the aim of this protocol is to study the genotype - phenotype correlations of the CNS manifestations of late infantile neuronal ceroid lipofuscinosis. This will be accomplished by comparing the genotype to a neurologic assessment, and LINCL clinical rating scale; magnetic resonance imaging (MRI) and magnetic resonance spectroscopic (MRS) assessments of the CNS; and routine clinical evaluations.

Infantile Batten disease/INCL/CLN1 - Drug trial

Last Updated 2010-05-23 at 15:42:16
The primary aim of the study is to assess the genotype - phenotype correlations of the CNS manifestations of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal, rare, recessive disorder of the CNS in children. This study will be accomplished by comparing the genotype to a neurologic assessment and Weill Cornell LINCL scale, the UBDRS scale, the standardized CHQ quality of life scale, and the Mullen scale; magnetic resonance imaging (MRI); and routine clinical evaluations. This study is designed to run parallel to a separate study which is being done by the Department of Genetic Medicine, which will use gene transfer to treat the central nervous system (CNS) manifestations of late infantile neuronal ceroid lipofuscinosis.