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CellCept for the Treatment of Juvenile Neuronal Ceroid Lipofuscinoses (JUMP)

The primary objective of this trial was to establish the safely and tolerability of short-term administration of mycophenolate (Cellcept). Dr. Erika Augustine (University of Rochester Medical Centre) presented results of the study: Developing therapies for individuals with CLN3 disease – results from a phase 2, double-blind, crossover study of mycophenolate at NCL2016 held in October in Boston, USA.

Nineteen people were enrolled into a double-blind crossover study. This meant that they were given 8 weeks of the drug, mycophenolate, followed by 8 weeks of placebo, or vice versa. The most common adverse events, more commonly referred to as side effects, in participants on mycophenolate were vomiting, diarrhea, and cough. These symptoms were mild in severity.

This study has now been completed. Overall, mycophenolate was well tolerated in the short-term by those taking part in the study. Evaluation of long-term mycophenolate exposure is needed to evaluate whether mycophenolate is beneficial in people with CLN3 disease.

A useful source of information about clinical trials is the National Institutes of Health (NIH) website and then search for Batten disease or Neuronal Ceroid Lipofuscinoses (NCL).

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