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

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ClinicalTrials.gov Identifier: NCT02895906
Recruitment Status : Completed
First Posted : September 12, 2016
Last Update Posted : April 27, 2017
Sponsor:
Information provided by (Responsible Party):
Aevi Genomic Medicine

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
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 : November 28, 2016
Actual Primary Completion Date : April 13, 2017
Actual Study Completion Date : April 20, 2017


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 Name: NFC1




Primary Outcome Measures :
  1. Time to symptom relapse [ Time Frame: Through study completion, up to 5 weeks ]

Secondary Outcome Measures :
  1. Response to treatment based on Clinical Global Impression - Improvement scale [ Time Frame: Through study completion, up to 5 weeks ]
  2. Safety and tolerability as determined by AEs, laboratory results, C-SSRS, and K-SADS [ Time Frame: Through study completion, up to 5 weeks ]


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Ages Eligible for Study:   12 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 < 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02895906


Locations
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Aevi Genomic Medicine

Responsible Party: Aevi Genomic Medicine
ClinicalTrials.gov Identifier: NCT02895906     History of Changes
Other Study ID Numbers: MDGN-NFC1-22Q-101
First Posted: September 12, 2016    Key Record Dates
Last Update Posted: April 27, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Aevi Genomic Medicine:
22q11.2 DS

Additional relevant MeSH terms:
Syndrome
DiGeorge Syndrome
Disease
Pathologic Processes
22q11 Deletion Syndrome
Craniofacial Abnormalities
Musculoskeletal Abnormalities
Musculoskeletal Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Lymphatic Abnormalities
Lymphatic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Hypoparathyroidism
Parathyroid Diseases
Endocrine System Diseases