An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome

NCT ID: NCT05011851

Last Updated: 2025-01-31

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-07-12
• Study Completion Date: 2024-07-16

Brief Summary

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Angelman syndrome

Detailed Description

The primary purpose of this study is to investigate the safety, tolerability and pharmacokinetics of treatment with NNZ-2591 oral solution, 50mg/L, in children and adolescents with Angelman syndrome. The secondary purpose is to investigate measures of efficacy of subjects will receive treatment of 50mg/mL orally administered NNZ-2591 for a total of 13 weeks

Conditions

Angelman Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

NNZ-2591

NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.

Group Type: EXPERIMENTAL

NNZ-2591

Intervention Type: DRUG

NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.

Interventions

NNZ-2591

NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.

Intervention Type: DRUG

Other Intervention Names

Cyclo-L-Glycyl-L-2-Allylproline

Eligibility Criteria

Inclusion Criteria

1. Clinical diagnosis of AS with a documented disease-causing genetic etiology known to impact maternally derived UBE3A expression in brain.

2. Males or females aged 3-17 years

3. Body Weight of >12Kg

4. Subjects with a Clinical Global Impression - Severity (CGI-S) score of 3 or greater

5. Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit

6. Each subject must be able to swallow the study medication provided as a liquid solution.

7. Caregiver(s) must have sufficient English language skills.

Exclusion Criteria

1. Mosaicism for disease-causing mutation.

2. Clinically Significant abnormalities in safety laboratory testing or vital signs at screening

3. Abnormal QTcF interval or prolongation at Screening.

4. Positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and previous COVID 19 infection with last 12 months that required hospitalization.

5. Unstable or changes to Psychotropic treatment 2 weeks prior to screening .

6. Excluded concomitant treatments

7. Actively undergoing regression or loss of skills.

8. Unstable seizure profile.

9. Current clinically significant renal conditions and abnormalities

10. Current clinically significant cardiovascular, hepatic, gastrointestinal, respiratory, endocrine disease, or clinically significant organ impairment.

11. Current clinically significant hypo or hyperthyroidism, Type 1 or Type 2 diabetes mellitus requiring insulin (whether well controlled or uncontrolled), or uncontrolled Type 1 or Type 2 diabetes.

12. Has planned surgery during the study.

13. History of, or current, cerebrovascular disease or brain trauma.

14. History of, or current catatonia or catatonia-like symptoms.

15. History of, or current, malignancy.

16. Current major or persistent depressive disorder (including bipolar depression).

17. Significant, uncorrected visual or uncorrected hearing impairment.

18. Allergy to strawberry.

19. Positive pregnancy test

20. Subject is judged by the Investigator or Medical Monitor to be inappropriate for the study

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Neuren Pharmaceuticals Limited

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

James Shaw

Role: STUDY_DIRECTOR

Neuren Pharmaceuticals

Locations

Sydney Children's Hospital

Randwick, New South Wales, Australia

Centre for Clinical Trials in Rare Neurodevelopmental Disorders at Children's Health Queensland Hospital and Health Service

South Brisbane, Queensland, Australia

Austin Health

Heidelberg, Victoria, Australia

Countries

Australia

Other Identifiers

NEU-2591-AS-001

Identifier Type: -

Identifier Source: org_study_id