Registered outcomes17

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

1 to 17 of 17

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	CGI-I	CGI-I was assessed at Weeks 6, 13/EOT & 15. Overall improvement scores relate to Week 13/EOT visit.	Phelan-McDermid Syndrome-specific Clinical Global Impression of Improvement Scale (CGI-I) - Overall Improvement Score on a 7 point Likert scale (1-7) where lower scores are better.
Secondary	CIC	CIC was assessed at Week13/EOT	Caregiver Impression of Improvement: Measured on a 7 point Likert scale (1-7) where lower scores are better.
Secondary	CGI-S	Change in score assessed from baseline (visit 3, week 0) to visit 16 (week 13/EOT).	Phelan-McDermid 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 a lower score is better.
Secondary	Top 3 Concerns	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall improvement score.	Caregiver Top 3 Concerns - Total Concerns Severity: Change from baseline. The range of scores was (0-30) for total concerns, with higher scores being worse
Secondary	MB-CDI	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16).	MacArthur-Bates Communicative Development Inventory (MB-CDI) - change from baseline. Range of scores was (0-792) with higher scores being better.
Secondary	ORCA	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in Total Score.	Observer-Reported Communication Ability (ORCA) - Change from baseline in Total Score. Range of Scores was (25.8-83.8) with higher scores being better
Secondary	ABC-2	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total score.	Aberrant Behavior Checklist-2 (ABC-2) - Total score: Change from baseline. Range of scores was (0-174) with higher scores being worse
Secondary	CSHQ	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total score.	Child Sleep Habits Questionnaire (CSHQ) - Change from baseline in Total Score. Range of scores was (33-99) with higher scores being worse.
Secondary	GIHQ	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total frequency score.	Gastrointestinal Health Questionnaire (GIHQ) - Total Frequency Score: Change from Baseline. Range of scores was (0-212) with higher scores being worse.
Secondary	VABS-3	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in composite standard score.	Vineland Adaptive Behavior Scales-3, Change from baseline in Composite Standard Score. Range of scores was (20-140) with higher scores being better.
Secondary	QL-Disability	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall score score.	Quality of Life Inventory-Disability (QL-Disability) Overall Score - change from baseline. Range of scores was (0-100) with higher scores being better.
Secondary	ICND	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall quality of life rating score.	Impact of Childhood Neurological Disability (ICND) - Change from baseline in overall quality of life rating. Range of (1- 6) for quality of life rating, with higher scores indicating greater impact.
Secondary	PMS-DSRS	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall severity score.	PMS Clinician Domain Specific Rating Scale - Change from baseline in overall severity score. Range of Scores Was (0-20) With Higher Scores Being Worse.
Secondary	Behavior Problems Inventory - Short Form	Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total frequency score.	Total Frequency Score - Change from Baseline. Range of scores was (0-4) for each of 30 behaviors, total range (0-120), with lower scores being better.

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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