Desipramine in Infantile Neuroaxonal Dystrophy (INAD).

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.

Timeline

Start

2019-01-14

Primary completion

2019-08-30

Completion

2019-08-30

Drugs

Indications