Trial · NCT03493607

AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy

NCT03493607, Phase 2, Completed, Alexander Kolevzon; results first posted 2021-06-22

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy. Trials, record NCT03493607. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03493607

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
NCT number: NCT03493607
Status: Completed
Phases: Phase 2
Enrollment: 6 participants, actual
Lead sponsor: Alexander Kolevzon
Conditions: Phelan-McDermid Syndrome, Epilepsy
Interventions: AMO-01
First posted: 2018-04-10
Last updated: 2025-06-27
Results first posted: 2021-06-22

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Adverse Events	8 weeks	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.
Secondary	Number of Weekly Seizure Counts	4 weeks	Seizure 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.
Secondary	Change in CGI - Improvement and Severity Scale	baseline, Week 1, Week 2, and Week 4	Clinical 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.
Secondary	Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)	8 weeks	9 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.
Secondary	Aberrant Behavior Checklist (ABC)	baseline, Week 1, Week 2, and Week 4	rating 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.
Secondary	Repetitive Behavior Scale-Revised (RBS-R)	Baseline, Week 1, Week 2, Week 4	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.

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Adverse Events	8 weeks		events	NUMBER
SECONDARY	Number of Weekly Seizure Counts	4 weeks		seizure event	MEAN
SECONDARY	Change in CGI - Improvement and Severity Scale	baseline, Week 1, Week 2, and Week 4		score on a scale	MEAN
SECONDARY	Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)	8 weeks		score on a scale	MEAN
SECONDARY	Aberrant Behavior Checklist (ABC)	baseline, Week 1, Week 2, and Week 4		score on a scale	MEAN
SECONDARY	Repetitive Behavior Scale-Revised (RBS-R)	Baseline, Week 1, Week 2, Week 4	Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.	score on a scale	MEAN

Source ClinicalTrials.gov

Results by group97

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Number of Adverse Events	AMO-01		19		events
Number of Weekly Seizure Counts	Participants With Phelan-McDermid Syndrome (PMS) at Baseline		22.6	Standard Deviation 42.9	seizure event
Number of Weekly Seizure Counts	Participants With Phelan-McDermid Syndrome (PMS) at Week 1		4.3	Standard Deviation 7.8	seizure event
Number of Weekly Seizure Counts	Participants With Phelan-McDermid Syndrome (PMS) at Week 2		15.8	Standard Deviation 37.3	seizure event
Number of Weekly Seizure Counts	Participants With Phelan-McDermid Syndrome (PMS) at Week 4		14.3	Standard Deviation 33.2	seizure event
Change in CGI - Improvement and Severity Scale	Participants With Phelan-McDermid Syndrome (PMS) at Baseline	Improvement	0	Standard Deviation 0	score on a scale
Change in CGI - Improvement and Severity Scale	Participants With Phelan-McDermid Syndrome (PMS) at Week 1	Improvement	0.83	Standard Deviation 0.98	score on a scale
Change in CGI - Improvement and Severity Scale	Participants With Phelan-McDermid Syndrome (PMS) at Week 2	Improvement	1	Standard Deviation 1.1	score on a scale
Change in CGI - Improvement and Severity Scale	Participants With Phelan-McDermid Syndrome (PMS) at Week 4	Improvement	1	Standard Deviation 0.89	score on a scale
Change in CGI - Improvement and Severity Scale	Participants With Phelan-McDermid Syndrome (PMS) at Baseline	Severity	5.33	Standard Deviation 0.82	score on a scale

1 to 10 of 97Source ClinicalTrials.gov

See all 97 results by group

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	AMO-01	6
Overall Study	COMPLETED	AMO-01	6
Overall Study	NOT COMPLETED	AMO-01	0

Source ClinicalTrials.gov

Adverse events17

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Constipation	Gastrointestinal disorders	AMO-01	1	6
other	Sleep Issues	General disorders	AMO-01	1	6
other	Congestion/Cough	Respiratory, thoracic and mediastinal disorders	AMO-01	1	6
other	Headache	General disorders	AMO-01	1	6
other	Cellulitis	Skin and subcutaneous tissue disorders	AMO-01	1	6
other	Redness on hands	Skin and subcutaneous tissue disorders	AMO-01	1	6
other	Decreased appetite/weight loss	Metabolism and nutrition disorders	AMO-01	1	6
other	Viral Infection	Infections and infestations	AMO-01	1	6
other	Rash on nect	Skin and subcutaneous tissue disorders	AMO-01	1	6
other	Ear Infection	Ear and labyrinth disorders	AMO-01	1	6

1 to 10 of 17Source ClinicalTrials.gov

See all 17 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	AMO-01	0	6
Total, other adverse events	AMO-01	4	6
Total, serious adverse events	AMO-01	0	6

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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