PART B: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and Without mGluR Mutations

NCT03609619 | Completed Phase 2 Results FDA Drug

• 1 other identifier: AEVI-001-ADHD-202B
• Study Type: interventional
• Target: 109
• Locations: 1 country
• Sites: 1

Brief Summary

This is PART B of a 2-part, 6-week, double-blind, dose-optimization, parallel-group study in children and adolescents (ages 6-17 years) with ADHD with and without CNVs in specific genes implicated in glutamatergic signaling and neuronal activity. PART B will assess subjects who do not have CNVs in any of the specific gene mutation(s) implicated in glutamatergic signaling and neuronal connectivity.

Conditions

Attention Deficit Hyperactivity Disorder

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score

  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.

  Baseline to Visit 8 (Week 6)

Secondary Outcomes (1)

• Clinical Global Impression - Global Improvement (CGI -I) Response

  Visit 3 to Visit 8 (Week 6)

Study Arms (2)

AEVI-001

EXPERIMENTAL

Drug: AEVI-001

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

AEVI-001 DRUG

Oral doses of 100 mg, 200 mg or 400 mg of AEVI-001 will be administered twice daily, during the treatment period.

Also known as: MDGN-001, NFC-1

AEVI-001

Placebo DRUG

Oral doses of Placebo will be administered twice daily, during the treatment period.

Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subject and parent/legally authorized representative (LAR) can speak English fluently and have provided written informed consent, and assent (as applicable) for this study.
• Subject is male or non-pregnant, non-lactating female, who if of childbearing potential agrees to comply with any applicable contraceptive requirements prior to administration of investigational product (IP).
• Subject meets Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria for a primary diagnosis of ADHD based upon DSM 5 criteria.
• Subject has a minimum score of ≥28 on the ADHD-RS-5 at the Baseline Visit (Visit 2).
• Subject has been genotyped previously and has their identity confirmed (if required).

You may not qualify if:

• Subject or parent/LAR is, in the opinion of the investigator, mentally or legally incapacitated, has significant emotional problems at the time of the Screening Visit (Visit 1) which could interfere with the conduct of study evaluations.
• Subject has autism spectrum disorder to include a DSM-IV diagnosis of autistic disorder, Asperger's disorder, or pervasive developmental disorder.
• Subject is currently taking any medication that might confound the results of safety assessments conducted in the study.
• Subject has a known history of cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope, or other serious cardiac problems.
• Subject has any clinically significant abnormality on 12-lead ECG performed at the Screening Visit (Visit 1) and/or the Baseline Visit (Visit 2) such as serious arrhythmia, cardiac conduction problems, or other abnormalities deemed to be a potential safety issue.

Sponsors & Collaborators

• Aevi Genomic Medicine, LLC, a Cerecor company: lead

Study Sites (1)

Aevi Genomic Medicine

Wayne, Pennsylvania, 19087, United States

Location

MeSH Terms

Conditions

Attention Deficit Disorder with Hyperactivity

Condition Hierarchy (Ancestors)

Attention Deficit and Disruptive Behavior Disorders; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Garry A. Neil, MD
• Organization: Aevi Genomic Medicine

garry.neil@aevigenomics.com

610-254-4208

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 31, 2018
• First Posted: August 1, 2018
• Study Start: August 17, 2018
• Primary Completion: November 23, 2018
• Study Completion: November 30, 2018
• Last Updated: July 30, 2021
• Results First Posted: July 16, 2019

Record last verified: 2019-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)