Rapalogues for Autism Phenotype in TSC: A Feasibility Study
Status:
Completed
Trial end date:
2016-08-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to assess the feasibility and safety of administering
rapalogues, sirolimus or everolimus, in participants with Tuberous Sclerosis Complex (TSC)
and self-injury and to measure cognitive and behavioral changes, including reduction in
autistic symptoms, self-injurious and aggressive behaviors, as well as improvements in
cognition across multiple domains of cognitive function.
Phase:
Phase 2
Details
Lead Sponsor:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.