AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
Status:
Completed
Trial end date:
2020-03-31
Target enrollment:
Participant gender:
Summary
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.