Stopped: Funding exhausted
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.
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Change in Gross Motor Function as Measured by Gross Motor Function Measure (GMFM-66)
Timeframe: Baseline, 3, 6, 9, and 12 months
Change in Motor Function as Measured by Quick Motor Function Test (QMFT)
Timeframe: Baseline, 3, 6, 9, and 12 months
Change in Cognitive Function as Measured by the Vineland Adaptive Behavioral Scale
Timeframe: Baseline, 3, 6, 9, and 12 months
Number of Participants With Change in Q-T Interval on ECG
Timeframe: Baseline, 3, 6, 9, and 12 months
Number of Participants With Abnormal Transaminase Values
Timeframe: Baseline, 3, 6, 9, and 12 months