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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05011851
Other study ID # NEU-2591-AS-001
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date July 12, 2022
Est. completion date June 30, 2024

Study information

Verified date August 2023
Source Neuren Pharmaceuticals Limited
Contact Fernanda Cecchin
Phone +61 2 9171 3274
Email Fernanda.Cecchin@novotech-cro.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


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


Recruitment information / eligibility

Status Recruiting
Enrollment 20
Est. completion date June 30, 2024
Est. primary completion date June 30, 2024
Accepts healthy volunteers No
Gender All
Age group 3 Years to 17 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
Australia Austin Health Heidelberg Victoria
Australia 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

Sponsors (1)

Lead Sponsor Collaborator
Neuren Pharmaceuticals Limited

Country where clinical trial is conducted

Australia, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety and Tolerability To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591. 13 weeks
Primary Pharmacokinetic - Measurement of Cmax Maximum observed concentration (Cmax) of NNZ-2591 13 weeks
Primary Pharmacokinetic - Measurement of AUC Area under the concentration-time curve of NNZ-2591 13 weeks
Primary Pharmacokinetic - Measurement of time to Cmax Time to Cmax of NNZ-2591 13 weeks
Primary Pharmacokinetic - Measurement of t1/2 Apparent terminal elimination half-life of NNZ-2591 13 weeks
Secondary Exploratory efficacy measurement Assessed by Angelman syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Caregiver Impression of Change 13 weeks
Secondary Exploratory efficacy measurement Assessed by Angelman syndrome-specific Clinical Global Impression Scales-Domain Improvement 13 weeks
Secondary Exploratory efficacy measurement Assessed by Angelman syndrome-specific Clinical Global Impression Scale-Severity (CGI-S)-Overall and Domain 13 weeks
Secondary Exploratory efficacy measurement Assessed by Angelman syndrome Clinician Domain Specific Rating Scale (AS-DSRS) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Caregiver Top 3 Concerns Likert Scale 13 weeks
Secondary Exploratory efficacy measurement Assessed by MacArthur-Bates Communicative Development Inventory (MB-CDI) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Observer-Reported Communication Ability (ORCA) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Aberrant Behavior Checklist-2 (ABC-2) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Child Sleep Habits Questionnaire (CSHQ) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Gastrointestinal Health Questionnaire (GIHQ) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Vineland Adaptive Behavior Scales-3, Interview version 13 weeks
Secondary Exploratory efficacy measurement Assessed by Bayley Scales of Infant Development-4, Vineland Motor subscales 13 weeks
Secondary Exploratory efficacy measurement Caregiver Diaries 13 weeks
Secondary Exploratory efficacy measurement Assessed by Quality of Life Inventory-Disability (QI-Disability) 13 weeks
Secondary Exploratory efficacy measurement Assessed by Impact of Childhood Neurological Disability (ICND)-Overall quality of life rating 13 weeks
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