An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
NCT05011851 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 17
Last updated 2025-01-31
Summary
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Angelman syndrome
Conditions
Interventions
- DRUG
-
NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Sponsors & Collaborators
-
Neuren Pharmaceuticals Limited
lead INDUSTRY
Principal Investigators
-
James Shaw · Neuren Pharmaceuticals
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 3 Years
- Max Age
- 17 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2022-07-12
- Primary Completion
- 2024-05-13
- Completion
- 2024-07-16
- FDA Drug
- Yes
Countries
- Australia
Study Locations
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