Desipramine in Infantile Neuroaxonal Dystrophy (INAD).
Status:
Terminated
Trial end date:
2019-08-30
Target enrollment:
Participant gender:
Summary
This is a research study to find out if clinically prescribed desipramine is effective at
improving the symptoms and slowing the progression of Infantile Neuroaxonal Dystrophy (INAD)
in affected children.
Participants will receive an initial oral dose of study drug once a day. This dose may be
changed depending on response to study drug Clinically collected data will be recorded for up
to 5 years. Investigators will also ask for participant permission to obtain a sample of
child's skin biopsy from unused clinical sample previously collected for standard of care.