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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AEVI-001 | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Global Impression - Global Improvement (CGI -I) Response | 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 | Visit 3 to Visit 8 (Week 6) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Aevi Genomic Medicine | Wayne | Pennsylvania | 19087 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | AEVI-001 | Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d. |
| FG001 | Placebo | Oral doses of Placebo administered b.i.d. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
ITT Analysis Set
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| ID | Title | Description |
|---|---|---|
| BG000 | AEVI-001 | Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d. |
| BG001 | Placebo | Oral doses of Placebo administered b.i.d. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline to Visit 8 (Week 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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | AEVI-001 | Oral doses of 100 mg, 200 mg or 400 mg administered b.i.d. | 0 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Weight increased | Investigations | MedDRA version 19.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Garry A. Neil, MD | Aevi Genomic Medicine | 610-254-4208 | garry.neil@aevigenomics.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 25, 2018 | Jun 25, 2019 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 5, 2018 | Jun 25, 2019 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D001289 | Attention Deficit Disorder with Hyperactivity |
| ID | Term |
|---|---|
| D019958 | Attention Deficit and Disruptive Behavior Disorders |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| Placebo | Drug | Oral doses of Placebo will be administered twice daily, during the treatment period. |
|
| Protocol Violation |
|
| Withdrawal by Subject |
|
| Lost to Follow-up |
|
| BG002 | Total | Total of all reporting groups |
| years |
|
| Age, Customized | Count of Participants | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| 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. | Mean | Standard Deviation | units on a scale |
|
Oral doses of Placebo administered b.i.d.
|
|
| Secondary | Clinical Global Impression - Global Improvement (CGI -I) Response | 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 | Posted | Count of Participants | Participants | Visit 3 to Visit 8 (Week 6) |
|
|
|
| 53 |
| 0 |
| 53 |
| 3 |
| 53 |
| EG001 | Placebo | Oral doses of Placebo administered b.i.d. | 0 | 55 | 0 | 55 | 9 | 53 |
| Headache | Nervous system disorders | MedDRA version 19.0 | Non-systematic Assessment |
|
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.