Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
Status:
Completed
Trial end date:
2015-11-01
Target enrollment:
Participant gender:
Summary
The primary objective of this trial is to establish the safety and tolerability of short-term
(8 weeks) administration of mycophenolate mofetil in ambulatory children with JNCL. The
secondary objective is to gather preliminary evidence of the short-term (8 week) impact of
mycophenolate mofetil on clinically relevant features of JNCL as measured by the Unified
Batten Disease Rating Scale (UBDRS), including motor features, seizures, behavior, cognitive
and functional measures.
Funding source-FDA Office of Orphan Product Development (OOPD).
Phase:
Phase 2
Details
Lead Sponsor:
University of Rochester
Collaborator:
Batten Disease Support and Research Assocation (BDSRA)