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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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10 February 2025 |
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Main ID: |
NCT05011851 |
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Date of registration:
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05/08/2021 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
AS-001 |
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Scientific title:
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An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome |
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Date of first enrolment:
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July 12, 2022 |
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Target sample size:
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17 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT05011851 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Australia
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Contacts
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Contact type:
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Name:
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James Shaw |
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Address:
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Telephone:
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Email:
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Affiliation:
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Neuren Pharmaceuticals |
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Key inclusion & exclusion criteria
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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
Age minimum:
3 Years
Age maximum:
17 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Angelman Syndrome
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Intervention(s)
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Drug: NNZ-2591
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Primary Outcome(s)
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Pharmacokinetic - Typical t1/2 in 30 kg Child
[Time Frame: 13 weeks]
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Pharmacokinetic - Typical AUC24 of 30kg Child
[Time Frame: 13 weeks]
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Safety and Tolerability
[Time Frame: 13 weeks]
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Secondary Outcome(s)
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Vineland Adaptive Behavior Scales-3, Interview version
[Time Frame: 13 weeks]
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Angelman syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I)
[Time Frame: 13 weeks]
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Caregiver Top 3 Concerns
[Time Frame: 13 weeks]
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Angelman syndrome-specific Clinical Global Impression Scale - Severity (CGI-S): Overall Score
[Time Frame: 13 weeks]
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Angelman syndrome Clinician Domain Specific Rating Scale (AS-DSRS)
[Time Frame: 13 weeks]
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Exploratory efficacy measurement
[Time Frame: 13 weeks]
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Child Sleep Habits Questionnaire (CSHQ)
[Time Frame: 13 weeks]
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MacArthur-Bates Communicative Development Inventory (MB-CDI)
[Time Frame: 13 weeks]
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Observer-Reported Communication Ability (ORCA)
[Time Frame: 13 weeks]
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Quality of Life Inventory-Disability (QI-Disability)
[Time Frame: 13 weeks]
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Aberrant Behavior Checklist-2 (ABC-2)
[Time Frame: 13 weeks]
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Gastrointestinal Health Questionnaire (GIHQ)
[Time Frame: 13 weeks]
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Caregiver Impression of Improvement : Overall Score
[Time Frame: 13 weeks]
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Impact of Childhood Neurological Disability (ICND)
[Time Frame: 13 weeks]
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Secondary ID(s)
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NEU-2591-AS-001
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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| Results available: |
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