Clinical Trial: Safety and Efficacy Study of NFC-1 in Subjects Aged 12-17 Years With 22q11.2DS & Associated Neuropsychiatric Conditions
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A 5-Week, Multi-center, Open-label Study to Assess the Safety and Efficacy of NFC-1 in Subjects Aged 12-17 Years With 22q11.2 Deletion Syndrome and Commonly Associated Neu
Brief Summary: This is a 5-week, multi-center, open-label, dose optimization trial in subjects aged 12-17 years with 22q11DS who have a diagnosis of anxiety disorder, and/or ADHD, and/or ASD. Approximately 12 subjects will be initiated, dose optimized, and maintained on NFC-1 over a period of 5 weeks.
Detailed Summary:
Sponsor: Aevi Genomic Medicine
Current Primary Outcome: Time to symptom relapse [ Time Frame: Through study completion, up to 5 weeks ]
Original Primary Outcome: Time to symptom relapse [ Time Frame: Randomized withdrawal period (up to 7 weeks) ]
Current Secondary Outcome:
- Response to treatment based on Clinical Global Impression - Improvement scale [ Time Frame: Through study completion, up to 5 weeks ]
- Safety and tolerability as determined by AEs, laboratory results, C-SSRS, and K-SADS [ Time Frame: Through study completion, up to 5 weeks ]
Original Secondary Outcome:
- Response to treatment based on Clinical Global Impression - Improvement scale [ Time Frame: Open label treatment period (up to 5 weeks) ]
- Safety and tolerability as determined by AEs, laboratory results, C-SSRS, and K-SADS [ Time Frame: Through study completion, up to 12 weeks ]
Information By: Aevi Genomic Medicine
Dates:
Date Received: August 29, 2016
Date Started: November 28, 2016
Date Completion:
Last Updated: April 26, 2017
Last Verified: April 2017
