Trial Outcomes & Findings for PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations (NCT NCT03265119)

Last Updated: 2021-07-07

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

69 participants

Primary outcome timeframe

Baseline to Visit 8 (Week 6)

Results posted on

2021-07-07

Participant Flow

Participant milestones

Participant milestones
Measure	AEVI-001 100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.	Placebo Placebo b.i.d.
Overall Study STARTED	34	35
Overall Study COMPLETED	24	28
Overall Study NOT COMPLETED	10	7

Reasons for withdrawal

Reasons for withdrawal
Measure	AEVI-001 100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.	Placebo Placebo b.i.d.
Overall Study Adverse Event	2	1
Overall Study Lack of Efficacy	3	0
Overall Study Protocol Violation	1	2
Overall Study Withdrawal by Subject	2	4
Overall Study Lost to Follow-up	1	0
Overall Study Lack of Compliance	1	0

Baseline Characteristics

PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	AEVI-001 n=34 Participants 100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.	Placebo n=35 Participants Placebo b.i.d.	Total n=69 Participants Total of all reporting groups
Age, Continuous	9.9 years STANDARD_DEVIATION 2.95 • n=99 Participants	10.8 years STANDARD_DEVIATION 2.86 • n=107 Participants	10.4 years STANDARD_DEVIATION 2.92 • n=206 Participants
Age, Customized 6 to 12 years	26 Participants n=99 Participants	27 Participants n=107 Participants	53 Participants n=206 Participants
Age, Customized 13 to 17 years	8 Participants n=99 Participants	8 Participants n=107 Participants	16 Participants n=206 Participants
Sex: Female, Male Female	7 Participants n=99 Participants	14 Participants n=107 Participants	21 Participants n=206 Participants
Sex: Female, Male Male	27 Participants n=99 Participants	21 Participants n=107 Participants	48 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=99 Participants	4 Participants n=107 Participants	10 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	28 Participants n=99 Participants	31 Participants n=107 Participants	59 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	1 Participants n=99 Participants	0 Participants n=107 Participants	1 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	24 Participants n=99 Participants	20 Participants n=107 Participants	44 Participants n=206 Participants
Race (NIH/OMB) White	7 Participants n=99 Participants	12 Participants n=107 Participants	19 Participants n=206 Participants
Race (NIH/OMB) More than one race	2 Participants n=99 Participants	3 Participants n=107 Participants	5 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Region of Enrollment United States	34 participants n=99 Participants	35 participants n=107 Participants	69 participants n=206 Participants
Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score	39.0 units on a scale STANDARD_DEVIATION 6.76 • n=99 Participants	38.1 units on a scale STANDARD_DEVIATION 8.29 • n=107 Participants	38.6 units on a scale STANDARD_DEVIATION 7.53 • n=206 Participants

PRIMARY outcome

Timeframe: Baseline to Visit 8 (Week 6)

Population: Full Analysis Set

Outcome measures

Outcome measures
Measure	AEVI-001 n=33 Participants 100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.	Placebo n=34 Participants Placebo b.i.d.
Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score	-9.17 units on a scale Standard Error 2.235	-11.34 units on a scale Standard Error 2.122

SECONDARY outcome

Timeframe: Visit 8 (Week 6)

Population: Full Analysis Set

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

Outcome measures
Measure	AEVI-001 n=33 Participants 100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.	Placebo n=34 Participants Placebo b.i.d.
Clinical Global Impression - Global Improvement (CGI -I) Response	9 Participants	11 Participants

Adverse Events

AEVI-001

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	AEVI-001 n=34 participants at risk 100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.	Placebo n=35 participants at risk Placebo b.i.d.
Psychiatric disorders Irritability	2.9% 1/34 • Number of events 1 • 1 year, 1 month, 4 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.	0.00% 0/35 • 1 year, 1 month, 4 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.

Other adverse events

Other adverse events
Measure	AEVI-001 n=34 participants at risk 100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.	Placebo n=35 participants at risk Placebo b.i.d.
Gastrointestinal disorders Abdominal pain upper	5.9% 2/34 • Number of events 2 • 1 year, 1 month, 4 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.	2.9% 1/35 • Number of events 1 • 1 year, 1 month, 4 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.
General disorders Pyrexia	5.9% 2/34 • Number of events 2 • 1 year, 1 month, 4 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.	0.00% 0/35 • 1 year, 1 month, 4 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.
General disorders Fatigue	2.9% 1/34 • Number of events 1 • 1 year, 1 month, 4 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.	8.6% 3/35 • Number of events 3 • 1 year, 1 month, 4 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.
Infections and infestations Upper respiratory tract infection	5.9% 2/34 • Number of events 2 • 1 year, 1 month, 4 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.	2.9% 1/35 • Number of events 1 • 1 year, 1 month, 4 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.
Investigations Weight increased	11.8% 4/34 • Number of events 4 • 1 year, 1 month, 4 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.	11.4% 4/35 • Number of events 4 • 1 year, 1 month, 4 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	8.8% 3/34 • Number of events 4 • 1 year, 1 month, 4 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.	11.4% 4/35 • Number of events 4 • 1 year, 1 month, 4 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 Sedation	5.9% 2/34 • Number of events 2 • 1 year, 1 month, 4 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.	0.00% 0/35 • 1 year, 1 month, 4 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

Garry A. Neil, MD

Aevi Genomic Medicine

Phone: 610-254-4208

Email: [email protected]

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