Desipramine in Infantile Neuroaxonal Dystrophy (INAD).
Stopped early · Phase 4
Conditions studied: Infantile Neuroaxonal Dystrophy
In brief
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.
Key facts
- Study ID
- NCT03726996
- Run by
- Duke University
- People needed
- 4
- Starts
- 2019-01-14
- Expected to finish
- 2019-08-30
- Last updated by the study team
- 2020-10-14
Who can join
Age: 3 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 03-17years.
- Any gender
- Confirmed homozygotes or compound heterozygotes of pathogenic mutation variant(s) in PLA2G6
- Confirmed homozygotes of pathogenic mutation in PLA2G6
- Documentation of clinical presentation (signs and symptoms of neurodegenerative process) of INAD
You may not qualify if…
- Patient has sign and symptom suggesting an ongoing acute or chronic illness such as fever of unknown origin or infection.
- Patient has a second genetic condition
- Parents are unable or unwilling to return for continued care for up to 12 months
Where it is running
- Duke University Health Center — Durham, North Carolina, United States
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.