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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05011851




Registration number
NCT05011851
Ethics application status
Date submitted
5/08/2021
Date registered
18/08/2021

Titles & IDs
Public title
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
Scientific title
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
Secondary ID [1] 0 0
NEU-2591-AS-001
Universal Trial Number (UTN)
Trial acronym
AS-001
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Angelman Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - NNZ-2591

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


Treatment: Drugs: NNZ-2591
NNZ-2591 oral solution (50mg/mL) to be administered twice daily for 13 weeks.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and Tolerability
Timepoint [1] 0 0
13 weeks
Primary outcome [2] 0 0
Pharmacokinetic - Typical AUC24 of 30kg Child
Timepoint [2] 0 0
13 weeks
Primary outcome [3] 0 0
Pharmacokinetic - Typical t1/2 in 30 kg Child
Timepoint [3] 0 0
13 weeks
Secondary outcome [1] 0 0
Angelman syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I)
Timepoint [1] 0 0
13 weeks
Secondary outcome [2] 0 0
Caregiver Impression of Improvement : Overall Score
Timepoint [2] 0 0
13 weeks
Secondary outcome [3] 0 0
Angelman syndrome-specific Clinical Global Impression Scale - Severity (CGI-S): Overall Score
Timepoint [3] 0 0
13 weeks
Secondary outcome [4] 0 0
Angelman syndrome Clinician Domain Specific Rating Scale (AS-DSRS)
Timepoint [4] 0 0
13 weeks
Secondary outcome [5] 0 0
Caregiver Top 3 Concerns
Timepoint [5] 0 0
13 weeks
Secondary outcome [6] 0 0
MacArthur-Bates Communicative Development Inventory (MB-CDI)
Timepoint [6] 0 0
13 weeks
Secondary outcome [7] 0 0
Observer-Reported Communication Ability (ORCA)
Timepoint [7] 0 0
13 weeks
Secondary outcome [8] 0 0
Aberrant Behavior Checklist-2 (ABC-2)
Timepoint [8] 0 0
13 weeks
Secondary outcome [9] 0 0
Child Sleep Habits Questionnaire (CSHQ)
Timepoint [9] 0 0
13 weeks
Secondary outcome [10] 0 0
Gastrointestinal Health Questionnaire (GIHQ)
Timepoint [10] 0 0
13 weeks
Secondary outcome [11] 0 0
Vineland Adaptive Behavior Scales-3, Interview version
Timepoint [11] 0 0
13 weeks
Secondary outcome [12] 0 0
Exploratory efficacy measurement
Timepoint [12] 0 0
13 weeks
Secondary outcome [13] 0 0
Quality of Life Inventory-Disability (QI-Disability)
Timepoint [13] 0 0
13 weeks
Secondary outcome [14] 0 0
Impact of Childhood Neurological Disability (ICND)
Timepoint [14] 0 0
13 weeks

Eligibility
Key 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.
Minimum age
3 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/07/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
16/07/2024
Sample size
Target
Accrual to date
Final
17
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
Centre for Clinical Trials in Rare Neurodevelopmental Disorders at Children's Health Queensland Hospital and Health Service - South Brisbane
Recruitment hospital [3] 0 0
Austin Health - Heidelberg
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3084 - Heidelberg

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Neuren Pharmaceuticals Limited
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
James Shaw
Address 0 0
Neuren Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Fernanda Cecchin
Address 0 0
Country 0 0
Phone 0 0
+61 2 9171 3274
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05011851