Trial Outcomes & Findings for Efficacy and Safety of NFC-1 in Adolescents With Genetic Disorders Impacting mGluR and ADHD (NCT NCT02777931)
NCT ID: NCT02777931
Last Updated: 2021-08-11
Results Overview
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.
COMPLETED
PHASE2/PHASE3
101 participants
Baseline to Visit 8 (Week 6)
2021-08-11
Participant Flow
Participant milestones
| Measure |
NFC-1
Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules).
|
Placebo
Matching placebo capsules
|
|---|---|---|
|
Overall Study
STARTED
|
49
|
52
|
|
Overall Study
Safety Population
|
47
|
50
|
|
Overall Study
Modified Intent to Treat Population
|
46
|
50
|
|
Overall Study
COMPLETED
|
37
|
39
|
|
Overall Study
NOT COMPLETED
|
12
|
13
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy and Safety of NFC-1 in Adolescents With Genetic Disorders Impacting mGluR and ADHD
Baseline characteristics by cohort
| Measure |
NFC-1
n=47 Participants
Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules).
|
Placebo
n=50 Participants
Matching placebo capsules
|
Total
n=97 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
13.8 years
STANDARD_DEVIATION 1.40 • n=39 Participants
|
14.4 years
STANDARD_DEVIATION 1.68 • n=41 Participants
|
14.1 years
STANDARD_DEVIATION 1.58 • n=35 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=39 Participants
|
15 Participants
n=41 Participants
|
36 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=39 Participants
|
35 Participants
n=41 Participants
|
61 Participants
n=35 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Black or African American
|
11 Participants
n=39 Participants
|
18 Participants
n=41 Participants
|
29 Participants
n=35 Participants
|
|
Race (NIH/OMB)
White
|
29 Participants
n=39 Participants
|
26 Participants
n=41 Participants
|
55 Participants
n=35 Participants
|
|
Race (NIH/OMB)
More than one race
|
6 Participants
n=39 Participants
|
3 Participants
n=41 Participants
|
9 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
|
Region of Enrollment
United States
|
47 Participants
n=39 Participants
|
50 Participants
n=41 Participants
|
97 Participants
n=35 Participants
|
PRIMARY outcome
Timeframe: Baseline to Visit 8 (Week 6)Population: 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.
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.
Outcome measures
| Measure |
NFC-1
n=46 Participants
Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules).
|
Placebo
n=50 Participants
Matching placebo capsules
|
|---|---|---|
|
Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score
|
-14.2 units on a scale
Standard Error 1.82
|
-12.1 units on a scale
Standard Error 1.75
|
PRIMARY outcome
Timeframe: Visit 3 to Visit 8 (Week 6)Population: 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.
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
Outcome measures
| Measure |
NFC-1
n=46 Participants
Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules).
|
Placebo
n=50 Participants
Matching placebo capsules
|
|---|---|---|
|
Clinical Global Impression - Global Improvement (CGI -I) Response
|
26 Participants
|
16 Participants
|
Adverse Events
NFC-1
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
NFC-1
n=47 participants at risk
Doses of NFC-1 will be administered as 100, 200, or 400 mg twice daily as capsules (size 2 hard gelatin capsules).
|
Placebo
n=50 participants at risk
Matching placebo capsules
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
6.4%
3/47 • Number of events 3 • 8 months, 5 days
|
8.0%
4/50 • Number of events 4 • 8 months, 5 days
|
|
General disorders
Fatigue
|
14.9%
7/47 • Number of events 7 • 8 months, 5 days
|
6.0%
3/50 • Number of events 3 • 8 months, 5 days
|
|
Infections and infestations
Nasopharyngitis
|
2.1%
1/47 • Number of events 1 • 8 months, 5 days
|
8.0%
4/50 • Number of events 4 • 8 months, 5 days
|
|
Infections and infestations
Upper respiratory tract infection
|
4.3%
2/47 • Number of events 2 • 8 months, 5 days
|
10.0%
5/50 • Number of events 5 • 8 months, 5 days
|
|
Injury, poisoning and procedural complications
Accidental overdose
|
10.6%
5/47 • Number of events 7 • 8 months, 5 days
|
6.0%
3/50 • Number of events 3 • 8 months, 5 days
|
|
Investigations
Weight increase
|
14.9%
7/47 • Number of events 7 • 8 months, 5 days
|
4.0%
2/50 • Number of events 2 • 8 months, 5 days
|
|
Metabolism and nutrition disorders
Increased appetite
|
6.4%
3/47 • Number of events 3 • 8 months, 5 days
|
4.0%
2/50 • Number of events 2 • 8 months, 5 days
|
|
Nervous system disorders
Headache
|
8.5%
4/47 • Number of events 5 • 8 months, 5 days
|
10.0%
5/50 • Number of events 10 • 8 months, 5 days
|
|
Psychiatric disorders
Irritability
|
2.1%
1/47 • Number of events 1 • 8 months, 5 days
|
6.0%
3/50 • Number of events 3 • 8 months, 5 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER