NCT03726996

Desipramine in Infantile Neuroaxonal Dystrophy (INAD).

Novel Off-label Use of Desipramine in Infantile Neuroaxonal Dystrophy: Targeting the Sphingolipid Metabolism Pathway to Reduce Accumulation of Ceramide.

• Status: Terminated
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Duke University · Academic / Other
• Sex: All
• Age: 3 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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. .

Conditions

• Infantile Neuroaxonal Dystrophy

Interventions

Type	Name	Description
DRUG	Desipramine	Study drug (desipramine) provided in tablet form to be taken daily.

Timeline

• Start date: 2019-01-14
• Primary completion: 2019-08-30
• Completion: 2019-08-30
• First posted: 2018-11-01
• Last updated: 2020-10-14
• Results posted: 2020-10-14

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study