AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
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|ClinicalTrials.gov Identifier: NCT03493607|
Recruitment Status : Recruiting
First Posted : April 10, 2018
Last Update Posted : April 24, 2019
|Condition or disease||Intervention/treatment||Phase|
|Phelan-McDermid Syndrome Epilepsy||Drug: AMO-01||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Single Group, open label|
|Masking:||None (Open Label)|
|Official Title:||An Open-label Study to Investigate the Safety, Tolerability and Efficacy of a Single 6-hour Intravenous Infusion of AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy|
|Actual Study Start Date :||May 31, 2018|
|Estimated Primary Completion Date :||July 2019|
|Estimated Study Completion Date :||July 2019|
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 [ Time Frame: 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.
- Change in CGI - Improvement Scale [ Time Frame: baseline and 8 weeks ]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 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.
- Clinician-completed PMS domain specific causes for concerns Visual Analogue Scale (VAS) [ Time Frame: 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.
- Top 3 caregiver Concerns VAS [ Time Frame: 4 weeks ]The Top 3 concerns visual analogue scale allows caregivers to identify their main three causes of concern, related to the subject's PMS. Caregivers will be asked to rate each of the three causes for concern by drawing a vertical mark on a 10 cm long visual analogue scale, with anchors of 0 "not at all severe" at the left end and 100 "very severe" at the right end.
- Aberrant Behavior Checklist (ABC) [ Time Frame: 4 weeks ]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.
- Repetitive Behavior Scale-Revised (RBS-R) [ Time Frame: 4 weeks ]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).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03493607
|Contact: Jordana Weissman, BAfirstname.lastname@example.org|
|United States, New York|
|Seaver Autism Center for Research and Treatment at Mount Sinai||Recruiting|
|New York, New York, United States, 10029|
|Contact: Jordana Weissman, BA 212-241-3072 email@example.com|
|Principal Investigator: Alexander Kolevzon, MD|
|Principal Investigator:||Alexander Kolevzon, MD||Icahn School of Medicine at Mount Sinai|