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

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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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Attention Deficit Disorder With Hyperactivity
Interventions Drug: NFC-1
Drug: Placebo
Enrollment 101
Recruitment Details  
Pre-assignment Details  
Arm/Group Title NFC-1 Placebo
Hide Arm/Group Description Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules). Matching placebo capsules
Period Title: Overall Study
Started [1] 49 52
Safety Population [2] 47 50
Modified Intent to Treat Population [3] 46 50
Completed 37 39
Not Completed 12 13
[1]
All randomized subjects (Intent to Treat Population)
[2]
All randomized subjects who took at least 1 dose of study drug
[3]
Subjects who took at least 1 dose of study drug and have valid baseline and post baseline assessment
Arm/Group Title NFC-1 Placebo Total
Hide Arm/Group Description Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules). Matching placebo capsules Total of all reporting groups
Overall Number of Baseline Participants 47 50 97
Hide Baseline Analysis Population Description
Safety Population
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 47 participants 50 participants 97 participants
13.8  (1.40) 14.4  (1.68) 14.1  (1.58)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 47 participants 50 participants 97 participants
Female
21
  44.7%
15
  30.0%
36
  37.1%
Male
26
  55.3%
35
  70.0%
61
  62.9%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 47 participants 50 participants 97 participants
American Indian or Alaska Native
1
   2.1%
1
   2.0%
2
   2.1%
Asian
0
   0.0%
1
   2.0%
1
   1.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
11
  23.4%
18
  36.0%
29
  29.9%
White
29
  61.7%
26
  52.0%
55
  56.7%
More than one race
6
  12.8%
3
   6.0%
9
   9.3%
Unknown or Not Reported
0
   0.0%
1
   2.0%
1
   1.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 47 participants 50 participants 97 participants
47
 100.0%
50
 100.0%
97
 100.0%
1.Primary Outcome
Title Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score
Hide Description

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.

Time Frame Baseline to Visit 8 (Week 6)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Modified Intent to Treat Population = all randomized subjects who took at least one dose of randomized study drug and have a valid baseline assessment and at least 1 valid post baseline assessment.
Arm/Group Title NFC-1 Placebo
Hide Arm/Group Description:
Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules).
Matching placebo capsules
Overall Number of Participants Analyzed 46 50
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
-14.2  (1.82) -12.1  (1.75)
2.Primary Outcome
Title Clinical Global Impression – Global Improvement (CGI –I) Response
Hide Description

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

Time Frame Visit 3 to Visit 8 (Week 6)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Modified Intent to Treat Population = all randomized subjects who took at least one dose of randomized study drug and have a valid baseline assessment and at least 1 valid post baseline assessment.
Arm/Group Title NFC-1 Placebo
Hide Arm/Group Description:
Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules).
Matching placebo capsules
Overall Number of Participants Analyzed 46 50
Measure Type: Count of Participants
Unit of Measure: Participants
26
  56.5%
16
  32.0%
Time Frame 8 months, 5 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title NFC-1 Placebo
Hide Arm/Group Description Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules). Matching placebo capsules
All-Cause Mortality
NFC-1 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/47 (0.00%)      0/50 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
NFC-1 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/47 (0.00%)      0/50 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
NFC-1 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   33/47 (70.21%)      28/50 (56.00%)    
Gastrointestinal disorders     
Nausea * 1  3/47 (6.38%)  3 4/50 (8.00%)  4
General disorders     
Fatigue * 1  7/47 (14.89%)  7 3/50 (6.00%)  3
Infections and infestations     
Nasopharyngitis * 1  1/47 (2.13%)  1 4/50 (8.00%)  4
Upper respiratory tract infection * 1  2/47 (4.26%)  2 5/50 (10.00%)  5
Injury, poisoning and procedural complications     
Accidental overdose * 1  5/47 (10.64%)  7 3/50 (6.00%)  3
Investigations     
Weight increase * 1  7/47 (14.89%)  7 2/50 (4.00%)  2
Metabolism and nutrition disorders     
Increased appetite * 1  3/47 (6.38%)  3 2/50 (4.00%)  2
Nervous system disorders     
Headache * 1  4/47 (8.51%)  5 5/50 (10.00%)  10
Psychiatric disorders     
Irritability * 1  1/47 (2.13%)  1 3/50 (6.00%)  3
1
Term from vocabulary, MedDRA (19.0)
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The information generated by this study is the property of Medgenics. Publication or other public presentation of NFC-1 data resulting from this study requires prior review and written approval of Medgenics. Abstracts, manuscripts, and presentation materials should be provided to Medgenics for review at least 30 days prior to the relevant submission deadline.
Results Point of Contact
Name/Title: Garry A Neil, MD
Organization: Aevi Genomic Medicine
Phone: 610-254-4208
Responsible Party: Aevi Genomic Medicine
ClinicalTrials.gov Identifier: NCT02777931     History of Changes
Other Study ID Numbers: MDGN-NFC1-ADHD-201
First Submitted: May 13, 2016
First Posted: May 19, 2016
Results First Submitted: January 25, 2018
Results First Posted: March 29, 2018
Last Update Posted: April 25, 2018