Infantile Neuroaxonal Dystrophy Clinical Trial
Official title:
Novel Off-label Use of Desipramine in Infantile Neuroaxonal Dystrophy: Targeting the Sphingolipid Metabolism Pathway to Reduce Accumulation of Ceramide.
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.
To be eligible participants must be able to swallow tablets The study drug is to be taken
once daily Schedule of events. Day 0 - ECG and blood tests (4 ml or ¾ teaspoon) Day 3 - ECG
and blood tests (4 ml or ¾ teaspoon) Day 7 - ECG and blood tests (4 ml or ¾ teaspoon) Weeks
2, 3, 4, 8 & 12. ECG and blood tests (4 ml or ¾ teaspoon) Every 3 months for up to 5 years.
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| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Active, not recruiting |
NCT03570931 -
A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
|
Phase 2/Phase 3 | |
| Completed |
NCT03999814 -
Natural History of Infantile Neuroaxonal Dystrophy
|