Safety and Efficacy Study of NFC-1 in Subjects Aged 12-17 Years With 22q11.2DS & Associated Neuropsychiatric Conditions

Descriptive Information
Brief Title † Safety and Efficacy Study of NFC-1 in Subjects Aged 12-17 Years With 22q11.2DS & Associated Neuropsychiatric Conditions
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 Neuropsychiatric Conditions (Anxiety, ADHD, ASD)
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 Description
Study Phase Phase 1
Study Type † Interventional
Study Design †
Primary Outcome Measure † Time to symptom relapse
Secondary Outcome Measure † Response to treatment based on Clinical Global Impression - Improvement scale
Condition † 22q11.2 Deletion Syndrome
Intervention † DrugNFC-1
Study Arms / Comparison Groups NFC-1 Doses of NFC-1 will be administered as 50, 100, 200, or 400 mg twice daily as capsules for oral administration.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 2
Start Date † November 28, 2016
Completion Date April 20, 2017
Primary Completion Date April 13, 2017
Eligibility Criteria † Inclusion Criteria: - Subject has a diagnosis of a 22q11.2 deletion prior to screening. - Subject has a diagnosis of anxiety disorder and/or ADHD and/or ASD based upon diagnostic evaluations performed at screening, history, and clinical judgement. - Subject is judged to be in general good health, other than having anxiety disorder and/or ADHD and/or ASD and 22q11DS. Medical conditions related to 22q11DS must be clinically stable post surgical correction and/or medical management. - Subject has no clinically significant abnormality on 12-lead electrocardiogram (ECG) performed at screening or baseline such as serious arrhythmia, bradycardia, tachycardia, cardiac conduction problems, or other abnormalities deemed to be a potential safety issue. - Subject and parent/legal guardian understand the study procedures and agree to the subject's participation in the study as indicated by parental/legal guardian signature on the subject informed consent form and subject's signature on the assent form. Exclusion Criteria: - Subject has a diagnosis of co-morbid major psychiatric disorders (ie, aside from anxiety disorder, ADHD, and/or ASD), including major depression, bipolar disease, schizophrenia (or any psychotic disorder), and moderate or severe intellectual disability, which in the opinion of the investigator may interfere with the conduct of study evaluations. - Subject has an IQ
Gender All
Ages 12 Years - 17 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT02895906
Organization ID MDGN-NFC1-22Q-101
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Aevi Genomic Medicine
Collaborators ††
Investigators † : ,
Information Provided By
Verification Date April 2017
First Received Date † August 29, 2016
Last Updated Date April 26, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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