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Efficacy and Safety of NFC-1 in Adolescents With Genetic Disorders Impacting mGluR and ADHD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02777931
Recruitment Status : Completed
First Posted : May 19, 2016
Results First Posted : March 29, 2018
Last Update Posted : April 25, 2018
Sponsor:
Information provided by (Responsible Party):
Aevi Genomic Medicine

Tracking Information
First Submitted Date  ICMJE May 13, 2016
First Posted Date  ICMJE May 19, 2016
Results First Submitted Date  ICMJE January 25, 2018
Results First Posted Date  ICMJE March 29, 2018
Last Update Posted Date April 25, 2018
Study Start Date  ICMJE June 2016
Actual Primary Completion Date February 10, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 28, 2018)
  • Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score [ Time Frame: Baseline to Visit 8 (Week 6) ]
    The ADHD-RS-5 is comprised of 18 frequency items and 12 impairment items. Each frequency item was scored on a scale from 0 = "Never or rarely" to 3 = "Very often". The ADHD-RS-5 total score was calculated as the sum of the 18 frequency item scores. The total score ranges from 0 to 54. Higher scores indicate greater symptom severity. Change from baseline value were calculated as the assessment value minus the baseline value.
  • Clinical Global Impression - Global Improvement (CGI -I) Response [ Time Frame: Visit 3 to Visit 8 (Week 6) ]
    The CGI-I item is rated on a 7-point scale from 1 = "Very much improved", 2 = "Much improved", 3 = "Minimally improved", 4 = "No change", 5 = "Minimally worse", 6 = "Much worse", 7 = "Very much worse". Response is defined as achieving a CGI-I score of 1 or 2, scores of 3 to 7 or missing are defined as Non Response
Original Primary Outcome Measures  ICMJE
 (submitted: May 18, 2016)
Change from baseline in ADHD-rating scale (ADHD-RS-5) Total Score [ Time Frame: Baseline to Visit 8 (Week 6) ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: May 18, 2016)
Change from baseline in Clinical Global Impression - Global Improvement (CGI -I) Scale. [ Time Frame: Baseline to Visit 8 (Week 6) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of NFC-1 in Adolescents With Genetic Disorders Impacting mGluR and ADHD
Official Title  ICMJE A Multicenter, 6-week, Double-blind, Randomized, Placebo-controlled, Parallel-design Study to Assess the Efficacy and Safety of NFC-1 in Adolescents (Ages 12-17) With Genetic Disorders Impacting Metabotropic Glutamate Receptors and ADHD
Brief Summary This is a randomized, double-blind, placebo-controlled, parallel-group study of NFC-1 versus placebo in adolescents with ADHD who have genetic disorders impacting mGluRs.
Detailed Description This is a randomized, double-blind, placebo-controlled, parallel-group study of adolescents with ADHD who have genetic disorders impacting mGluRs. Approximately 90 subjects will receive randomized treatment with NFC-1 or placebo. Dosing will be optimized during the first 4 weeks of treatment, based on clinical response and tolerability, and maintained for an additional 2 weeks.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Attention Deficit Disorder With Hyperactivity
Intervention  ICMJE
  • Drug: NFC-1
    NFC-1 is supplied as size 2 hard gelatin capsules.
    Other Name: NFC1
  • Drug: Placebo
    Matching placebo capsules
    Other Name: Control
Study Arms  ICMJE
  • Experimental: NFC-1
    Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules for oral administration.
    Intervention: Drug: NFC-1
  • Placebo Comparator: Placebo
    Matching placebo capsules.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 28, 2018)
101
Original Estimated Enrollment  ICMJE
 (submitted: May 18, 2016)
90
Actual Study Completion Date  ICMJE February 17, 2017
Actual Primary Completion Date February 10, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Subject has ADHD as defined by the Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5) and Version 5 of the Attention Deficit Hyperactivity Disorder Rating Scale (ADHD-RS-5) ≥ 28 at Baseline with or without conventional ADHD therapy.
  • Subject has an intelligence quotient (IQ) > 79, based on the Wechsler Abbreviated Scale of Intelligence, second edition (WASI-II).
  • Subject has been genotyped previously and determined to have disruptive mutations in genes within the glutamate receptor metabotropic (GRM)-network as determined by the presence of copy number variations (CNVs) (GRM biomarker-positive subjects). The confirmation of a subject's positive status will be provided by the sponsor.
  • Subject is judged to be in general good health, other than having ADHD, based on medical history, physical examination, vital signs measurements, laboratory safety tests, and the Columbia Suicide Severity Rating Scale (C-SSRS) performed at the Screening Visit and/or prior to administration of investigational product (IP).
  • Subject has no clinically significant abnormality on electrocardiogram (ECG) performed at the Screening Visit and/or prior to administration of IP such as serious arrhythmia, bradycardia, tachycardia, cardiac conduction problems, or other abnormalities deemed to be a potential safety issue.
  • Parent/legal guardian and subject 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 signature on the assent form.

Exclusion Criteria:

  • Subjects with prior diagnosis of comorbid major psychiatric disorders (ie, aside from ADHD), including major depression, bipolar disease, schizophrenia, pervasive development disorder, and intellectual disability.
  • Subject is currently taking a prohibited medication and/or is unwilling to wean off current ADHD medication to participate in the study
  • Subject has a history of any illness that in the opinion of the study investigator might confound the results of the study or poses an additional risk to the subject by his or her participation in the study.
  • Subject has a known history or presence of syncope, cardiac conduction problems (eg, clinically significant heart block), exercise-related cardiac events including syncope and pre-syncope, or clinically significant bradycardia.
  • Subject has a history of stroke, chronic seizures, or 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 study.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02777931
Other Study ID Numbers  ICMJE MDGN-NFC1-ADHD-201
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Aevi Genomic Medicine
Study Sponsor  ICMJE Aevi Genomic Medicine
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Aevi Genomic Medicine
Verification Date March 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP