A Phase 2 interventional study of AMO-01 in Phelan-McDermid Syndrome and Epilepsy, sponsored by Alexander Kolevzon. Completed at 2 sites in United States. Open to participants aged 12 Years to 45 Years. Per ClinicalTrials.gov, last updated 2025-06-27.
Sponsored by Alexander Kolevzon · Phase 2, Interventional, and Treatment
The purpose of this study is to investigate the safety, tolerability and efficacy of a single 6-hour intravenous infusion of AMO-01 to treat adolescents and adults with PMS and co-morbid epilepsy. Phelan-McDermid Syndrome (PMS) is a neurodevelopmental disorder characterized by a chromosomal deletion or mutation at 22q13.3 that contains the SHANK3/ProSAP2 gene. A key co-morbidity in PMS is the presence of epilepsy. Currently there are no approved treatments for PMS. Furthermore, there has been relatively little clinical study of pharmacological interventions for PMS. AMO-01 may provide benefit to PMS patients exhibiting behavioral abnormalities and seizures.
1,848 studies on the registry are indexed under Epilepsy; 422 are open to participants now.
This study's enrollment of 6 is below the median of 50 across 1,239 interventional studies indexed under Epilepsy.
Browse Epilepsy studies →Alexander Kolevzon is the lead sponsor of 4 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Intravenous Infusion
Drug: AMO-01
Subjects will receive a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.
Number of Adverse Events
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.
Time frame: 8 weeks
Number of Weekly Seizure Counts
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.
Time frame: 4 weeks
Change in CGI - Improvement and Severity Scale
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.
Time frame: baseline, Week 1, Week 2, and Week 4
Clinician-completed PMS Domain Specific Causes for Concerns Visual Analogue Scale (VAS)
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.
Time frame: 8 weeks
Aberrant Behavior Checklist (ABC)
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.
Time frame: baseline, Week 1, Week 2, and Week 4
Repetitive Behavior Scale-Revised (RBS-R)
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.
Time frame: Baseline, Week 1, Week 2, Week 4
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.
| Milestone | AMO-01 |
|---|---|
| Started | 6 |
| Completed | 6 |
| Not completed | 0 |
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.
| events | AMO-01 |
|---|---|
| Number of Adverse Events | 19 |
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.
| seizure event | Participants With Phelan-McDermid Syndrome (PMS) at Baseline | 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 4 |
|---|---|---|---|---|
| Number of Weekly Seizure Counts | 22.6 ± 42.9 | 4.3 ± 7.8 | 15.8 ± 37.3 | 14.3 ± 33.2 |
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.
| score on a scale | Participants With Phelan-McDermid Syndrome (PMS) at Baseline | 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 4 |
|---|---|---|---|---|
| Improvement | 0 ± 0 | 0.83 ± 0.98 | 1 ± 1.1 | 1 ± 0.89 |
| Severity | 5.33 ± 0.82 | 5.33 ± 0.82 | 5.33 ± 0.82 | 5.17 ± 0.75 |
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.
| score on a scale | Participants With Phelan-McDermid Syndrome (PMS) at Baseline | 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 4 |
|---|---|---|---|---|
| Speech | 86.83 ± 8.13 | 84.33 ± 5.89 | 84.17 ± 5.85 | 81.67 ± 6.06 |
| Thinking and Learning | 86.67 ± 6.83 | 77.5 ± 9.87 | 81.67 ± 6.83 | 78.33 ± 2.58 |
| Seizures | 62 ± 28.43 | 41.67 ± 30.11 | 47.5 ± 34.31 | 44.17 ± 36.66 |
| Gross Motor | 28.33 ± 28.23 | 26.67 ± 24.01 | 24.17 ± 22.45 | 25 ± 23.45 |
| Repetitive Behavior | 70.33 ± 21.97 | 65 ± 24.08 | 60.83 ± 24.17 | 54 ± 25.77 |
| Social Communication | 80.83 ± 8.61 | 77.5 ± 5.24 | 77.5 ± 6.89 | 74.17 ± 4.92 |
| Sensory | 50.83 ± 21.78 | 48.33 ± 20.17 | 52.5 ± 22.97 | 50.33 ± 22.82 |
| Activities of Daily Living | 78.33 ± 10.33 | 75 ± 10.95 | 76.5 ± 10.75 | 78.33 ± 8.76 |
| Sleep | 43.33 ± 19.66 | 29.17 ± 17.15 | 43.33 ± 24.43 | 45 ± 30.66 |
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.
| score on a scale | Participants With Phelan-McDermid Syndrome (PMS) at Baseline | 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 4 |
|---|---|---|---|---|
| Irritability | 8.67 ± 6.02 | 9.33 ± 7.89 | 6.67 ± 3.72 | 4.83 ± 3.19 |
| Lethargy | 15 ± 7.13 | 12 ± 7.9 | 9.83 ± 6.01 | 8.83 ± 4.96 |
| Stereotypy | 6.67 ± 6.09 | 4.83 ± 5.08 | 4.17 ± 3.76 | 3.17 ± 2.93 |
| Hyperactivity | 20.33 ± 11.34 | 14.33 ± 7.79 | 15.67 ± 9.09 | 13.83 ± 6.27 |
| Inappropriate Speech | 1.17 ± 2.4 | 1.17 ± 2.4 | 0.17 ± 0.41 | 0 ± 0 |
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.
| score on a scale | Participants With Phelan-McDermid Syndrome (PMS) at Baseline | 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 4 |
|---|---|---|---|---|
| Stereotyped Behavior | 3.67 ± 3.5 | 3 ± 2.68 | 3.5 ± 2.07 | 2.67 ± 2.42 |
| Self-Injury | 1.83 ± 1.94 | 1.33 ± 1.51 | 2 ± 2.45 | 1.5 ± 2.35 |
| Compulsive Behavior | 2.5 ± 3.27 | 1.17 ± 1.47 | 2 ± 2.76 | 2.5 ± 2.81 |
| Ritualistic Behavior | 0 ± 0 | 0.17 ± 0.41 | 0.17 ± 0.41 | 0 ± 0 |
| Sameness Behavior | 2 ± 2.68 | 1.33 ± 1.75 | 2 ± 2.76 | 2.33 ± 2.66 |
| Restricted Behavior | 1.83 ± 2.4 | 1.83 ± 2.04 | 1.67 ± 1.97 | 1.83 ± 2.56 |
| Total | 11.83 ± 9.26 | 8.83 ± 5.56 | 11.33 ± 10.48 | 10.83 ± 10.87 |
Collected over 8 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| AMO-01 | 0/6 (0%) | 0/6 (0%) | 4/6 (66.7%) |
| Event | AMO-01 |
|---|---|
| Increased appetiteMetabolism and nutrition disorders | 2/6 |
| SeizureNervous system disorders | 2/6 |
| ConstipationGastrointestinal disorders | 1/6 |
| Sleep IssuesGeneral disorders | 1/6 |
| Congestion/CoughRespiratory, thoracic and mediastinal disorders | 1/6 |
| HeadacheGeneral disorders | 1/6 |
| CellulitisSkin and subcutaneous tissue disorders | 1/6 |
| Redness on handsSkin and subcutaneous tissue disorders | 1/6 |
| Decreased appetite/weight lossMetabolism and nutrition disorders | 1/6 |
| Viral InfectionInfections and infestations | 1/6 |
| Age, Continuous(years) | AMO-01 |
|---|---|
| Mean | 20.6 ± 2.07 |
| Sex: Female, Male(Participants) | AMO-01 |
|---|---|
| Female | 3 |
| Male | 3 |
| Ethnicity (NIH/OMB)(Participants) | AMO-01 |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 6 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | AMO-01 |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 6 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Undecided
Ran from May 2018 to primary completion in Mar 2020, 22 months.
Results were first posted in Jun 2021, 1 year 2 months after primary completion.
Of 66 phase 2 interventional studies under Epilepsy, half reached primary completion within 23 months of starting (the median), counting those still running; a quarter within 14 and three quarters within 40. This study's 22 months is close to that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Diazepinomicin
Alexander Kolevzon