Trial Outcomes & Findings for AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy (NCT NCT03493607)
NCT ID: NCT03493607
Last Updated: 2025-06-27
Results Overview
An adverse event is defined as any untoward medical occurrence in a study subject, temporally associated with the use of the experimental medication, whether or not considered related to the medication. An adverse event can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of the experimental medication. Adverse events will be monitored throughout all 8 weeks of study participation.
COMPLETED
PHASE2
6 participants
8 weeks
2025-06-27
Participant Flow
Recruitment began in Feb 2017, with first enrollment in May of 2018. Study was opened for enrollment through Jan 2021 when decision was made to close study due to low enrollment. Participants completed study visits. Last participant seen March 2020.
Participant milestones
| Measure |
AMO-01
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
Baseline characteristics by cohort
| Measure |
AMO-01
n=6 Participants
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
|---|---|
|
Age, Continuous
|
20.6 years
STANDARD_DEVIATION 2.07 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 8 weeksAn adverse event is defined as any untoward medical occurrence in a study subject, temporally associated with the use of the experimental medication, whether or not considered related to the medication. An adverse event can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of the experimental medication. Adverse events will be monitored throughout all 8 weeks of study participation.
Outcome measures
| Measure |
AMO-01
n=6 Participants
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 1
Participants with Phelan-McDermid syndrome (PMS) at week 1
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 2
Participants with Phelan-McDermid syndrome (PMS) at week 2
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 4
Participants with Phelan-McDermid syndrome (PMS) at week 4
|
|---|---|---|---|---|
|
Number of Adverse Events
|
19 events
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: 4 weeksSeizure frequency was measured by a caregiver-completed seizure diary throughout the duration of the trial. A seizure diary was provided to each family at the screening visit to record each seizure event, its date/time, duration, and type. The study team reviewed the diary at each visit with the caregiver and weekly seizure frequency was calculated. The week 1 seizure count was the number of seizures in the 7 days following the infusion day. Similarly, the week 2 seizure count was the number of seizures in the 7 days between weeks 1 and 2. Lastly, the week 4 seizure count was the sum of seizures in the 14 days between weeks 2 and 4, divided by 2.
Outcome measures
| Measure |
AMO-01
n=6 Participants
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 1
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 1
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 2
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 2
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 4
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 4
|
|---|---|---|---|---|
|
Number of Weekly Seizure Counts
|
22.6 seizure event
Standard Deviation 42.9
|
4.3 seizure event
Standard Deviation 7.8
|
15.8 seizure event
Standard Deviation 37.3
|
14.3 seizure event
Standard Deviation 33.2
|
SECONDARY outcome
Timeframe: baseline, Week 1, Week 2, and Week 4Clinical Global Impressions (CGI) Rating Scales are commonly used to measure symptom severity and global improvement in treatment studies of patients with developmental disorders. There Severity Scale (CGI-S) is a 7-point scale (1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.) that requires the clinician to rate the severity of illness at the time of assessment. The Improvement Scale (CGI-I) is a 7-point scale (1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse.) that requires the clinician to assess how much the illness has improved or worsened relative to baseline.
Outcome measures
| Measure |
AMO-01
n=6 Participants
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 1
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 1
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 2
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 2
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 4
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 4
|
|---|---|---|---|---|
|
Change in CGI - Improvement and Severity Scale
Improvement
|
0 score on a scale
Standard Deviation 0
|
0.83 score on a scale
Standard Deviation 0.98
|
1 score on a scale
Standard Deviation 1.1
|
1 score on a scale
Standard Deviation 0.89
|
|
Change in CGI - Improvement and Severity Scale
Severity
|
5.33 score on a scale
Standard Deviation 0.82
|
5.33 score on a scale
Standard Deviation 0.82
|
5.33 score on a scale
Standard Deviation 0.82
|
5.17 score on a scale
Standard Deviation 0.75
|
SECONDARY outcome
Timeframe: 8 weeks9 item visual analogue scale completed by the clinician that scores the severity of concerns in domains that are clinically relevant in PMS. For each subject, the clinician is instructed to identify the top 4 or 5 that are of particular concern and that the clinician would most like to see change during the course of treatment with the study medication. The severity of the clinician's concern in each domain is scored by using a 10 cm visual analogue scale, with anchors of 0 "not at all severe" at the left and 100 "very severe" at the right end.
Outcome measures
| Measure |
AMO-01
n=6 Participants
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 1
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 1
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 2
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 2
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 4
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 4
|
|---|---|---|---|---|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Speech
|
86.83 score on a scale
Standard Deviation 8.13
|
84.33 score on a scale
Standard Deviation 5.89
|
84.17 score on a scale
Standard Deviation 5.85
|
81.67 score on a scale
Standard Deviation 6.06
|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Thinking and Learning
|
86.67 score on a scale
Standard Deviation 6.83
|
77.5 score on a scale
Standard Deviation 9.87
|
81.67 score on a scale
Standard Deviation 6.83
|
78.33 score on a scale
Standard Deviation 2.58
|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Seizures
|
62 score on a scale
Standard Deviation 28.43
|
41.67 score on a scale
Standard Deviation 30.11
|
47.5 score on a scale
Standard Deviation 34.31
|
44.17 score on a scale
Standard Deviation 36.66
|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Gross Motor
|
28.33 score on a scale
Standard Deviation 28.23
|
26.67 score on a scale
Standard Deviation 24.01
|
24.17 score on a scale
Standard Deviation 22.45
|
25 score on a scale
Standard Deviation 23.45
|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Repetitive Behavior
|
70.33 score on a scale
Standard Deviation 21.97
|
65 score on a scale
Standard Deviation 24.08
|
60.83 score on a scale
Standard Deviation 24.17
|
54 score on a scale
Standard Deviation 25.77
|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Social Communication
|
80.83 score on a scale
Standard Deviation 8.61
|
77.5 score on a scale
Standard Deviation 5.24
|
77.5 score on a scale
Standard Deviation 6.89
|
74.17 score on a scale
Standard Deviation 4.92
|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Sensory
|
50.83 score on a scale
Standard Deviation 21.78
|
48.33 score on a scale
Standard Deviation 20.17
|
52.5 score on a scale
Standard Deviation 22.97
|
50.33 score on a scale
Standard Deviation 22.82
|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Activities of Daily Living
|
78.33 score on a scale
Standard Deviation 10.33
|
75 score on a scale
Standard Deviation 10.95
|
76.5 score on a scale
Standard Deviation 10.75
|
78.33 score on a scale
Standard Deviation 8.76
|
|
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
Sleep
|
43.33 score on a scale
Standard Deviation 19.66
|
29.17 score on a scale
Standard Deviation 17.15
|
43.33 score on a scale
Standard Deviation 24.43
|
45 score on a scale
Standard Deviation 30.66
|
SECONDARY outcome
Timeframe: baseline, Week 1, Week 2, and Week 4rating scaled used to monitor an array of behavioral features among patients with intellectual disabilities. It takes 15-30 minutes to complete. 16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). with total from 0 to 48.
Outcome measures
| Measure |
AMO-01
n=6 Participants
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 1
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 1
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 2
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 2
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 4
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 4
|
|---|---|---|---|---|
|
Aberrant Behavior Checklist (ABC)
Irritability
|
8.67 score on a scale
Standard Deviation 6.02
|
9.33 score on a scale
Standard Deviation 7.89
|
6.67 score on a scale
Standard Deviation 3.72
|
4.83 score on a scale
Standard Deviation 3.19
|
|
Aberrant Behavior Checklist (ABC)
Lethargy
|
15 score on a scale
Standard Deviation 7.13
|
12 score on a scale
Standard Deviation 7.9
|
9.83 score on a scale
Standard Deviation 6.01
|
8.83 score on a scale
Standard Deviation 4.96
|
|
Aberrant Behavior Checklist (ABC)
Stereotypy
|
6.67 score on a scale
Standard Deviation 6.09
|
4.83 score on a scale
Standard Deviation 5.08
|
4.17 score on a scale
Standard Deviation 3.76
|
3.17 score on a scale
Standard Deviation 2.93
|
|
Aberrant Behavior Checklist (ABC)
Hyperactivity
|
20.33 score on a scale
Standard Deviation 11.34
|
14.33 score on a scale
Standard Deviation 7.79
|
15.67 score on a scale
Standard Deviation 9.09
|
13.83 score on a scale
Standard Deviation 6.27
|
|
Aberrant Behavior Checklist (ABC)
Inappropriate Speech
|
1.17 score on a scale
Standard Deviation 2.4
|
1.17 score on a scale
Standard Deviation 2.4
|
0.17 score on a scale
Standard Deviation 0.41
|
0 score on a scale
Standard Deviation 0
|
SECONDARY outcome
Timeframe: Baseline, Week 1, Week 2, Week 4Population: Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
42-item rating scale that is completed by a parent or caregiver. It reports on the severity of repetitive behaviors. each item scored on 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. with total score from 0 (mild) to 126 (severe). Subscale ranges: stereotypic behavior (0 - 27);self-injurious behavior (0 - 24); compulsive behavior (0 - 18); ritualistic/Sameness Behavior (0 - 36); restricted Interests (0 - 9). Higher score in all subscales reflect increasing severity.
Outcome measures
| Measure |
AMO-01
n=6 Participants
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 1
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 1
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 2
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 2
|
Participants With Phelan-McDermid Syndrome (PMS) at Week 4
n=6 Participants
Participants with Phelan-McDermid syndrome (PMS) at week 4
|
|---|---|---|---|---|
|
Repetitive Behavior Scale-Revised (RBS-R)
Sameness Behavior
|
2 score on a scale
Standard Deviation 2.68
|
1.33 score on a scale
Standard Deviation 1.75
|
2 score on a scale
Standard Deviation 2.76
|
2.33 score on a scale
Standard Deviation 2.66
|
|
Repetitive Behavior Scale-Revised (RBS-R)
Restricted Behavior
|
1.83 score on a scale
Standard Deviation 2.4
|
1.83 score on a scale
Standard Deviation 2.04
|
1.67 score on a scale
Standard Deviation 1.97
|
1.83 score on a scale
Standard Deviation 2.56
|
|
Repetitive Behavior Scale-Revised (RBS-R)
Stereotyped Behavior
|
3.67 score on a scale
Standard Deviation 3.5
|
3 score on a scale
Standard Deviation 2.68
|
3.5 score on a scale
Standard Deviation 2.07
|
2.67 score on a scale
Standard Deviation 2.42
|
|
Repetitive Behavior Scale-Revised (RBS-R)
Self-Injury
|
1.83 score on a scale
Standard Deviation 1.94
|
1.33 score on a scale
Standard Deviation 1.51
|
2 score on a scale
Standard Deviation 2.45
|
1.5 score on a scale
Standard Deviation 2.35
|
|
Repetitive Behavior Scale-Revised (RBS-R)
Compulsive Behavior
|
2.5 score on a scale
Standard Deviation 3.27
|
1.17 score on a scale
Standard Deviation 1.47
|
2 score on a scale
Standard Deviation 2.76
|
2.5 score on a scale
Standard Deviation 2.81
|
|
Repetitive Behavior Scale-Revised (RBS-R)
Ritualistic Behavior
|
0 score on a scale
Standard Deviation 0
|
0.17 score on a scale
Standard Deviation 0.41
|
0.17 score on a scale
Standard Deviation 0.41
|
0 score on a scale
Standard Deviation 0
|
|
Repetitive Behavior Scale-Revised (RBS-R)
Total
|
11.83 score on a scale
Standard Deviation 9.26
|
8.83 score on a scale
Standard Deviation 5.56
|
11.33 score on a scale
Standard Deviation 10.48
|
10.83 score on a scale
Standard Deviation 10.87
|
Adverse Events
AMO-01
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
AMO-01
n=6 participants at risk
Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
|
|---|---|
|
Gastrointestinal disorders
Constipation
|
16.7%
1/6 • 8 weeks
|
|
General disorders
Sleep Issues
|
16.7%
1/6 • 8 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Congestion/Cough
|
16.7%
1/6 • 8 weeks
|
|
General disorders
Headache
|
16.7%
1/6 • 8 weeks
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
16.7%
1/6 • 8 weeks
|
|
Skin and subcutaneous tissue disorders
Redness on hands
|
16.7%
1/6 • 8 weeks
|
|
Metabolism and nutrition disorders
Decreased appetite/weight loss
|
16.7%
1/6 • 8 weeks
|
|
Infections and infestations
Viral Infection
|
16.7%
1/6 • 8 weeks
|
|
Skin and subcutaneous tissue disorders
Rash on nect
|
16.7%
1/6 • 8 weeks
|
|
Ear and labyrinth disorders
Ear Infection
|
16.7%
1/6 • 8 weeks
|
|
Nervous system disorders
Mouth movement, possible tic
|
16.7%
1/6 • 8 weeks
|
|
Metabolism and nutrition disorders
Increased appetite
|
33.3%
2/6 • 8 weeks
|
|
Psychiatric disorders
Increased rubbing/repetitive behaviors
|
16.7%
1/6 • 8 weeks
|
|
Nervous system disorders
Seizure
|
33.3%
2/6 • 8 weeks
|
|
Injury, poisoning and procedural complications
Drowsiness after infusion
|
16.7%
1/6 • 8 weeks
|
|
Injury, poisoning and procedural complications
Flushing in face and hand
|
16.7%
1/6 • 8 weeks
|
|
Psychiatric disorders
Motor Stereotypes
|
16.7%
1/6 • 8 weeks
|
Additional Information
Christina Layton
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee The Investigators agree not to publish reports, abstracts, or other data compilations concerning the Study before the first multi-site publication by Sponsor. If there is no multi-site publication within 18 months after the Study has been completed or terminated at all Study locations, Principal Investigator shall have the right to publish and or present the results of the Study generated or collected at Study Site(s) (but not the results of any other Study location).
- Publication restrictions are in place
Restriction type: OTHER