Trial · NCT05025332
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 18 of 18
Download this page as JSON| 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 - Mean AUC24 | Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13. | Approximately nine sparse pharmacokinetic (PK) samples were collected from each participant under steady-state conditions. These samples were taken at pre-dose, 1-3 hours post-dose, and/or 4-7 hours post-dose during Weeks 2, 6, and 13. The individual pharmacokinetic parameters for NNZ-2591, including half-life (t1/2) and area under the curve over 24 hours (AUC24), were derived using subject-level concentration-time profiles from the study population model. |
| Primary | Pharmacokinetic - t1/2 | Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13. | Approximately nine sparse pharmacokinetic (PK) samples were collected from each participant under steady-state conditions. These samples were taken at pre-dose, 1-3 hours post-dose, and/or 4-7 hours post-dose during Weeks 2, 6, and 13. The individual pharmacokinetic parameters for NNZ-2591, including half-life (t1/2) and area under the curve over 24 hours (AUC24), were derived using subject-level concentration-time profiles from the study population model. |
| Secondary | Pitt Hopkins Syndrome-specific Clinical Global Impression Scale (CGI-I) - Overall Improvement | CGI-I was assessed at weeks 6, 13/EOT & 15. Overall improvement scores relate to week 13/EOT visit. | Pitt Hopkins Syndrome-specific Clinical Global Impression Scale (CGI-I) - Overall Improvement. Score on a Likert scale (1-7) where lower scores are better. |
| Secondary | Caregiver Impression of Improvement: Overall Score | CIC was assessed at Week13/EOT | Caregiver Impression of Improvement: Overall Score. Measured on a 7 point Likert scale (1-7) where lower scores are better. |
| Secondary | Pitt Hopkins Syndrome-specific Clinical Global Impression Scale - Severity (CGI-S) - Overall Score | Change in score assessed from baseline (visit 3, week 0) to visit 16 (week 13/EOT). | Pitt Hopkins syndrome-specific Clinical Global Impression Scale - Severity (CGI-S) - Change from baselines on overall Score based on a 7 point Likert scale (1-7) where lower scores are better. |
| Secondary | Caregiver Top 3 Concerns | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in average concern severity. | Caregiver Top 3 Concerns: Change from baseline in Average Concern Severity. The average concern severity defined as the average of the severity scores for the three concerns evaluated at a given visit, and was calculated as long as at least one concern was useable for analysis at the visit. Scores range from 0 - 10 with higher scores indicating greater concern severity. A negative change from baseline indicates improvement. |
| Secondary | MacArthur-Bates Communicative Development Inventory (MB-CDI) | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13). | MacArthur-Bates Communicative Development Inventory (MB-CDI): Words Understood Domain. Scores ranges from 0-396, with higher scores indicating greater language ability. A positive change from baseline indicates improvement. |
| Secondary | Observer-Reported Communication Ability (ORCA) | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in Total Score. | Observer-Reported Communication Ability (ORCA): Change from baseline in Modified t-score. The ORCA measures an individual's communication abilities based on observations made by caregivers, parents, or other relevant observers, and is based on 4 domains: Expressive Communication, Receptive Communication, Social Communication, and Pragmatic Language Skills. Scores range from 25.8 - 83.8, with higher scores indicating greater communication ability. A positive change from baseline indicates improvement. A T-score standardizes the individual's performance relative to a normative sample. It typically has a mean of 50 and a standard deviation of 10. T score = 50 + 10 × (X-µ)/σ Where: X is the individual's raw score μ is the population mean σ is the standard deviation |
| Secondary | Aberrant Behavior Checklist-2 (ABC-2) | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in total score. | Aberrant Behavior Checklist-2 (ABC-2): Change from baseline in Total Score. Range of scores is 0-174, with higher scores indicating more behavioral issues. A negative change from baseline indicates improvement. |
| Secondary | CSHQ | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in total score. | Child Sleep Habits Questionnaire (CSHQ). Total Score. Change from baseline. Range of scores was (33-99) with higher scores being worse. |
| Secondary | GIHQ | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in total frequency score. | Gastrointestinal Health Questionnaire (GIHQ). Change from baseline in total frequency score. Range of scores was (0-197) with higher scores being worse. |
| Secondary | Vineland Adaptive Behavior Scales-3, Interview Version | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in composite standard score. | Vineland Adaptive Behavior Scales-3 (VABS-3), Interview version, Change from baseline in Adaptive Behavior Composite Standard Score. Scores range from 20 - 140 with higher scores indicating greater functional abilities. A positive change from baseline indicates improvement. |
| Secondary | Modified Two-minute Walk Test | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in distance travelled on two minute walk test. | Modified two-minute walk test. Change from baseline in distance travelled (m) on 2 minute walk test. The test was only administered for participants who were ambulatory and able to complete the assessment. A positive score on change from baseline indicates improvement in distance walked. |
| Secondary | QI-Disability | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in overall score score. | Quality of Life Inventory-Disability (QI-Disability). Overall Score change from baseline. Scores range from 0 - 100 with higher scores indicating better quality of life. A positive change from baseline indicates improvement. |
| Secondary | ICND | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in overall quality of life rating score. | Impact of Childhood Neurological Disability (ICND)-Overall quality of life rating, change from baseline. Score ranges from 1 - 6, with a higher score indicating better quality of life. A positive change from baseline indicates improvement. |
| Secondary | Behavior Problems Inventory - Short Form | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in Total Frequency score. | Change from baseline in Behavior Problems Inventory - Short Form Total Frequency Score. Scores range from 0-120, with higher scores indicating greater frequency in behavior problems. A negative change from baseline indicates improvement. |
| Secondary | Bayley Scales of Infant Development (BSID-4): Non Verbal Development Quotient (NVDQ) | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in NVDQ score. | Change from baseline in Non Verbal Development Quotient (NVDQ). Scores range from 40 - 160, with higher scores indicating greater development. A positive change from baseline indicates improvement. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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.
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