Trial · NCT05025332
An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
NCT05025332, Phase 2, Completed, Neuren Pharmaceuticals Limited; results first posted 2025-05-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001). Trials, record NCT05025332. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05025332
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
- NCT number
- NCT05025332
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 28 participants, actual
- Lead sponsor
- Neuren Pharmaceuticals Limited
- Conditions
- Pitt Hopkins Syndrome
- Interventions
- NNZ-2591
- First posted
- 2021-08-27
- Last updated
- 2025-06-18
- Results first posted
- 2025-05-18
Source ClinicalTrials.gov
Registered outcomes18
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 - 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. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety and Tolerability | 13 weeks | Safety population - all participants receiving at least one dose of NNZ-2591 | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Pharmacokinetic - Mean AUC24 | Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13. | All participants enrolled in this study who receive the study drug through to the morning dose of Week 2 (Visit 5) as a minimum, and who underwent PK sample collection at least one of the specified post-dose time point(s). | ug.h/mL | MEAN |
| PRIMARY | Pharmacokinetic - t1/2 | Pre-dose, 1-3 h post-dose and/or 4-7 h post-dose at Weeks 2, 6 and 13. | All participants enrolled in this study who receive the study drug through to the morning dose of Week 2 (Visit 5) as a minimum, and who undergo PK sample collection at least one of the specified post-dose time point(s). | hours | MEAN |
| 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. | Modified intention to treat (MITT) | score on a scale | MEAN |
| SECONDARY | Caregiver Impression of Improvement: Overall Score | CIC was assessed at Week13/EOT | score on a scale | MEAN | |
| 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). | Modified intention to treat (MITT) population | score on a scale | MEAN |
| SECONDARY | Caregiver Top 3 Concerns | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in average concern severity. | Modified intention to treat population | score on a scale | MEAN |
| SECONDARY | MacArthur-Bates Communicative Development Inventory (MB-CDI) | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13). | score on a scale | MEAN | |
| SECONDARY | Observer-Reported Communication Ability (ORCA) | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in Total Score. | T-score | MEAN | |
| SECONDARY | Aberrant Behavior Checklist-2 (ABC-2) | Change from baseline (visit 3, week 0) to visit 16/EOT (week 13) in total score. | score on a scale | MEAN |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group23
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety and Tolerability | NNZ-2591 | Participants with TEAE | 15 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with Serious TEAE | 0 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants who discontinued due to TEAE | 4 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with mild TEAE as highest intensity | 12 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with moderate TEAE as highest intensity | 3 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with severe TEAE as highest intensity | 0 | Participants | |
| Pharmacokinetic - Mean AUC24 | NNZ-2591 | 305 | Standard Deviation 56.1 | ug.h/mL | |
| Pharmacokinetic - t1/2 | NNZ-2591 | 7.17 | Standard Deviation 1.64 | hours | |
| Pitt Hopkins Syndrome-specific Clinical Global Impression Scale (CGI-I) - Overall Improvement | Modified Intention to Treat | 2.6 | Standard Deviation 0.92 | score on a scale | |
| Caregiver Impression of Improvement: Overall Score | Modified Intention to Treat | 3.0 | Standard Deviation 1.00 | score on a scale |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Screening | STARTED | NNZ-2591 | 28 | |
| Screening | COMPLETED | NNZ-2591 | 16 | |
| Screening | NOT COMPLETED | NNZ-2591 | 12 | |
| Treatment & Follow Up | STARTED | NNZ-2591 | 16 | |
| Treatment & Follow Up | COMPLETED | NNZ-2591 | 11 | |
| Treatment & Follow Up | NOT COMPLETED | NNZ-2591 | 5 |
Source ClinicalTrials.gov
Adverse events37
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Constipation | Gastrointestinal disorders | NNZ-2591 | 3 | 16 | 3 |
| other | Diarrhea | Gastrointestinal disorders | NNZ-2591 | 4 | 16 | 4 |
| other | Vomiting | Gastrointestinal disorders | NNZ-2591 | 2 | 16 | 2 |
| other | Fatigue | General disorders | NNZ-2591 | 4 | 16 | 4 |
| other | Somnolence | Nervous system disorders | NNZ-2591 | 2 | 16 | 2 |
| other | Irritability | Psychiatric disorders | NNZ-2591 | 2 | 16 | 2 |
| other | Contusion | Injury, poisoning and procedural complications | NNZ-2591 | 2 | 16 | 2 |
| other | Gastroenteritis - viral | Infections and infestations | NNZ-2591 | 2 | 16 | 2 |
| other | Nasopharyngitis | Infections and infestations | NNZ-2591 | 3 | 16 | 3 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | NNZ-2591 | 2 | 16 | 2 |
1 to 10 of 37Source ClinicalTrials.gov
See all 37 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | NNZ-2591 | 0 | 16 |
| Total, other adverse events | NNZ-2591 | 15 | 16 |
| Total, serious adverse events | NNZ-2591 | 0 | 16 |
Source ClinicalTrials.gov
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
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