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

Sponsor

Aevi Genomic Medicine, LLC, a Cerecor company (Industry)

Overall Status

Completed

CT.gov ID

NCT02895906

Collaborator

(none)

Enrollment

Location

Arm

4.7

Actual Duration (Months)

0.4

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
22q11.2 Deletion Syndrome	Drug: NFC-1	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

2 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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)

Actual Study Start Date :

Nov 28, 2016

Actual Primary Completion Date :

Apr 13, 2017

Actual Study Completion Date :

Apr 20, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: NFC-1 Doses of NFC-1 will be administered as 50, 100, 200, or 400 mg twice daily as capsules for oral administration.	Drug: NFC-1 Doses of NFC-1 will be administered as 50, 100, 200, or 400 mg twice daily as size 2, hard gelatin capsules for oral administration. Other Names: NFC1

Arm

Intervention/Treatment

Experimental: NFC-1

Doses of NFC-1 will be administered as 50, 100, 200, or 400 mg twice daily as capsules for oral administration.

Drug: NFC-1

Doses of NFC-1 will be administered as 50, 100, 200, or 400 mg twice daily as size 2, hard gelatin capsules for oral administration.

Other Names:

NFC1

Outcome Measures

Primary Outcome Measures

Time to symptom relapse [Through study completion, up to 5 weeks]

Secondary Outcome Measures

Response to treatment based on Clinical Global Impression - Improvement scale [Through study completion, up to 5 weeks]
Safety and tolerability as determined by AEs, laboratory results, C-SSRS, and K-SADS [Through study completion, up to 5 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 17 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 < 65 as determined by the Wechsler Abbreviated Scale of Intelligence.
Subject has a history of any illness that, in the opinion of the study investigator, might confound the results of the study or pose an additional risk to the subject by his/her participation in the study.
Subject has a history of clinically significant endocrine, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or disease which is not currently stable clinically. Subjects with a history of uncomplicated kidney stones may be enrolled in the study at the discretion of the investigator.
Subject has a history of stroke, chronic seizures, or other major neurological disorder which, in the opinion of the investigator, would interfere with the subject's ability to participate and/or be evaluated in the trial.
Subject is currently considered at risk for suicide (in the opinion of the investigator), has previously made a suicide attempt, or is currently demonstrating active suicidal ideation.
Subject has taken any antidepressants, antipsychotics, anxiolytics, or non-stimulant ADHD medication within 30 days of the Screening Visit.
Subject is taking a prohibited medication

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104

Sponsors and Collaborators

Aevi Genomic Medicine, LLC, a Cerecor company

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Aevi Genomic Medicine, LLC, a Cerecor company

ClinicalTrials.gov Identifier:

NCT02895906

Other Study ID Numbers:

MDGN-NFC1-22Q-101

First Posted:

Sep 12, 2016

Last Update Posted:

Mar 25, 2022

Last Verified:

Mar 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Undecided

Plan to Share IPD:

Undecided

Keywords provided by Aevi Genomic Medicine, LLC, a Cerecor company

22q11.2 DS

Additional relevant MeSH terms:

DiGeorge Syndrome

Syndrome

Study Results

No Results Posted as of Mar 25, 2022