NCT03493607

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Alexander Kolevzon · Academic / Other
• Sex: All
• Age: 12 Years – 45 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Phelan-McDermid Syndrome
• Epilepsy

Interventions

Type	Name	Description
DRUG	AMO-01	Subjects will receive a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.

Timeline

• Start date: 2018-05-30
• Primary completion: 2020-03-31
• Completion: 2020-03-31
• First posted: 2018-04-10
• Last updated: 2025-06-27
• Results posted: 2021-06-22

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study