NCT03882918

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 141 (actual)
• Sponsor: Healx AI · Industry
• Sex: All
• Age: 13 Years – 49 Years
• Healthy volunteers: Not accepted

Summary

This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001 (NCT02996305). Subjects with AS who completed the pharmacokinetic Study OV101-16-001 (NCT03109756) will also be permitted to participate, provided they meet all entry criteria.

Detailed description

This will be an open-label, long-term safety study for evaluation of further treatment with OV101 in subjects with AS who have completed previous Ovid studies (OV101-15-001 or OV101-16-001). There will be no placebo treatment. As this study will enroll subjects who have completed previous AS studies for different periods of time before entering this study, subjects will be required to complete screening and baseline visits before receiving OV101 under this protocol.The secondary objective of this study is to evaluate the long-term efficacy of OV101 treatment assessed by changes in behavior, sleep, and functioning in individuals with AS.

Conditions

• Angelman Syndrome

Interventions

Type	Name	Description
DRUG	OV101	Each subject will be titrated to his or her maximal tolerated daily dose, up to a maximum daily dose of 15 mg at bedtime.

Timeline

• Start date: 2019-01-31
• Primary completion: 2021-06-04
• Completion: 2021-06-30
• First posted: 2019-03-20
• Last updated: 2025-07-18
• Results posted: 2025-07-18

Locations

11 sites across 2 countries: United States, Israel

Regulatory

• FDA-regulated drug study