Trial Outcomes & Findings for PART B: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and Without mGluR Mutations (NCT NCT03609619)
NCT ID: NCT03609619
Last Updated: 2021-07-30
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
109 participants
Primary outcome timeframe
Baseline to Visit 8 (Week 6)
Results posted on
2021-07-30
Participant Flow
Participant milestones
| Measure |
AEVI-001
Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d.
|
Placebo
Oral doses of Placebo administered b.i.d.
|
|---|---|---|
|
Overall Study
STARTED
|
54
|
55
|
|
Overall Study
COMPLETED
|
44
|
49
|
|
Overall Study
NOT COMPLETED
|
10
|
6
|
Reasons for withdrawal
| Measure |
AEVI-001
Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d.
|
Placebo
Oral doses of Placebo administered b.i.d.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Lack of Efficacy
|
1
|
2
|
|
Overall Study
Protocol Violation
|
5
|
0
|
|
Overall Study
Withdrawal by Subject
|
2
|
3
|
|
Overall Study
Lost to Follow-up
|
1
|
1
|
Baseline Characteristics
PART B: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and Without mGluR Mutations
Baseline characteristics by cohort
| Measure |
AEVI-001
n=53 Participants
Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d.
|
Placebo
n=55 Participants
Oral doses of Placebo administered b.i.d.
|
Total
n=108 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
10.4 years
STANDARD_DEVIATION 3.04 • n=99 Participants
|
10.3 years
STANDARD_DEVIATION 2.70 • n=107 Participants
|
10.4 years
STANDARD_DEVIATION 2.86 • n=206 Participants
|
|
Age, Customized
6 to 12 years
|
41 Participants
n=99 Participants
|
42 Participants
n=107 Participants
|
83 Participants
n=206 Participants
|
|
Age, Customized
13 to 17 years
|
12 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=99 Participants
|
19 Participants
n=107 Participants
|
38 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
34 Participants
n=99 Participants
|
36 Participants
n=107 Participants
|
70 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
12 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
41 Participants
n=99 Participants
|
46 Participants
n=107 Participants
|
87 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
6 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
41 Participants
n=99 Participants
|
41 Participants
n=107 Participants
|
82 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
4 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
53 participants
n=99 Participants
|
55 participants
n=107 Participants
|
108 participants
n=206 Participants
|
|
Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score
|
40.5 units on a scale
STANDARD_DEVIATION 8.78 • n=99 Participants
|
40.6 units on a scale
STANDARD_DEVIATION 8.71 • n=107 Participants
|
40.6 units on a scale
STANDARD_DEVIATION 8.70 • n=206 Participants
|
PRIMARY outcome
Timeframe: 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.
Outcome measures
| Measure |
AEVI-001
n=52 Participants
Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d.
|
Placebo
n=54 Participants
Oral doses of Placebo administered b.i.d.
|
|---|---|---|
|
Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score
|
-11.04 units on a scale
Standard Error 1.730
|
-10.38 units on a scale
Standard Error 1.651
|
SECONDARY outcome
Timeframe: 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
Outcome measures
| Measure |
AEVI-001
n=52 Participants
Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d.
|
Placebo
n=54 Participants
Oral doses of Placebo administered b.i.d.
|
|---|---|---|
|
Clinical Global Impression - Global Improvement (CGI -I) Response
|
15 Participants
|
16 Participants
|
Adverse Events
AEVI-001
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
AEVI-001
n=53 participants at risk
Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d.
|
Placebo
n=53 participants at risk；n=55 participants at risk
Oral doses of Placebo administered b.i.d.
|
|---|---|---|
|
Investigations
Weight increased
|
1.9%
1/53 • Number of events 1 • 3 months, 13 days
All AEs are collected from the time of the informed consent is signed until the follow-up call is completed. This includes events occurring during the screening phase of the study, regardless of whether investigational product (IP) is administered. An AE will be considered treatment emergent if it occurs after the first dose of IP and within 3 days of a subjects last dose of IP.
|
9.4%
5/53 • Number of events 5 • 3 months, 13 days
All AEs are collected from the time of the informed consent is signed until the follow-up call is completed. This includes events occurring during the screening phase of the study, regardless of whether investigational product (IP) is administered. An AE will be considered treatment emergent if it occurs after the first dose of IP and within 3 days of a subjects last dose of IP.
|
|
Nervous system disorders
Headache
|
5.7%
3/53 • Number of events 6 • 3 months, 13 days
All AEs are collected from the time of the informed consent is signed until the follow-up call is completed. This includes events occurring during the screening phase of the study, regardless of whether investigational product (IP) is administered. An AE will be considered treatment emergent if it occurs after the first dose of IP and within 3 days of a subjects last dose of IP.
|
7.5%
4/53 • Number of events 8 • 3 months, 13 days
All AEs are collected from the time of the informed consent is signed until the follow-up call is completed. This includes events occurring during the screening phase of the study, regardless of whether investigational product (IP) is administered. An AE will be considered treatment emergent if it occurs after the first dose of IP and within 3 days of a subjects last dose of IP.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The information generated by this study is the property of Aevi Genomic Medicine, Inc. Publication or other public presentation of AEVI-001 data resulting from this study requires prior review and written approval of Aevi Genomic Medicine, Inc. Abstracts, manuscripts, and presentation materials should be provided to Aevi Genomic Medicine, Inc. for review at least 30 days prior to the relevant submission deadline.
- Publication restrictions are in place
Restriction type: OTHER