Trial · NCT05011851
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
NCT05011851, Phase 2, Completed, Neuren Pharmaceuticals Limited
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome. Trials, record NCT05011851. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05011851
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
- NCT number
- NCT05011851
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- Neuren Pharmaceuticals Limited
- Conditions
- Angelman Syndrome
- Interventions
- NNZ-2591
- First posted
- 2021-08-18
- Last updated
- 2025-01-31
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety and Tolerability | 13 weeks | To examine the incidence, severity and frequency of adverse events (AEs), including serious adverse events (SAEs) during treatment with NNZ-2591. |
| Primary | Pharmacokinetic - Typical AUC24 of 30kg Child | 13 weeks | Area under the concentration-time curve of NNZ-2591 over 24 hours |
| Primary | Pharmacokinetic - Typical t1/2 in 30 kg Child | 13 weeks | Apparent terminal elimination half-life of NNZ-2591 |
| Secondary | Angelman syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I) | 13 weeks | Assessed by Angelman syndrome-specific Clinical Global Impression Scale-Overall Improvement (CGI-I). Score on a Likert scale (1-7) where lower scores are better |
| Secondary | Caregiver Impression of Improvement : Overall Score | 13 weeks | Caregiver Impression of Improvement: Overall Score. Measured on a 7-point Likert scale (1-7) where lower scores are better. |
| Secondary | Angelman syndrome-specific Clinical Global Impression Scale - Severity (CGI-S): Overall Score | 13 weeks | Angelman syndrome-specific Clinical Global Impression Scale-Severity (CGI-S): Change from baseline on overall score based on a 7-point Likert scale (1-7) where lower scores are better. |
| Secondary | Angelman syndrome Clinician Domain Specific Rating Scale (AS-DSRS) | 13 weeks | Angelman syndrome Clinician Domain Specific Rating Scale (AS-DSRS). Change from baseline in total score based on a 5-point Likert scale (0-4) where lower scores are better. |
| Secondary | Caregiver Top 3 Concerns | 13 weeks | Caregiver Top 3 Concerns: Change from baseline in Average Concerns Severity. |
| Secondary | MacArthur-Bates Communicative Development Inventory (MB-CDI) | 13 weeks | MacArthur-Bates Communicative Development Inventory (MB-CDI) |
| Secondary | Observer-Reported Communication Ability (ORCA) | 13 weeks | Observer-Reported Communication Ability (ORCA). Change from baseline in modified t-score. Scores range from 25.8 - 83.8 with higher scores indicating greater communication abililty. A positive change from baseline indicates improvement |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05011851
GET https://conductscience.org/api/trials/v1/NCT05011851/registered