Trial · NCT05025241
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
NCT05025241, Phase 2, Completed, Neuren Pharmaceuticals Limited; results first posted 2025-05-30
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001). Trials, record NCT05025241. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05025241
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
- NCT number
- NCT05025241
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 18 participants, actual
- Lead sponsor
- Neuren Pharmaceuticals Limited
- Conditions
- Phelan-McDermid Syndrome
- Interventions
- NNZ-2591
- First posted
- 2021-08-27
- Last updated
- 2025-06-03
- Results first posted
- 2025-05-30
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 - 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 |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
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 | Intention to treat population | 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. | PK Population | µg.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 | CGI-I | CGI-I was assessed at Weeks 6, 13/EOT & 15. Overall improvement scores relate to Week 13/EOT visit. | Intention to treat | score on a scale | MEAN |
| SECONDARY | CIC | CIC was assessed at Week13/EOT | Intention to treat population | score on a scale | MEAN |
| SECONDARY | CGI-S | Change in score assessed from baseline (visit 3, week 0) to visit 16 (week 13/EOT). | Intention to treat | score on a scale | MEAN |
| SECONDARY | Top 3 Concerns | Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in overall improvement score. | Intention to treat population | score on a scale | MEAN |
| SECONDARY | MB-CDI | Change from baseline (visit 3, week 0) to visit 13/EOT (week 16). | Intention to treat population | score on a scale | MEAN |
| SECONDARY | ORCA | Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in Total Score. | Intention to treat population | score on a scale | MEAN |
| SECONDARY | ABC-2 | Change from baseline (visit 3, week 0) to visit 13/EOT (week 16) in total score. | Intention to treat population | score on a scale | MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 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 any AE | 17 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with TEAE | 17 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with Serious TEAE | 1 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants who discontinued due to TEAE | 3 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with mild TEAE | 11 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with moderate TEAE | 5 | Participants | |
| Safety and Tolerability | NNZ-2591 | Participants with severe TEAE | 1 | Participants | |
| Pharmacokinetic - Mean AUC24 | PK Population | 411 | Standard Deviation 116 | µg.h/mL | |
| Pharmacokinetic - t1/2 | PK Population | 8.02 | Standard Deviation 2.33 | hours | |
| CGI-I | NNZ-2591 | 2.4 | Standard Deviation 0.86 | 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 | 23 | |
| Screening | COMPLETED | NNZ-2591 | 18 | |
| Screening | NOT COMPLETED | NNZ-2591 | 5 | |
| Treatment and Follow Up | STARTED | NNZ-2591 | 18 | |
| Treatment and Follow Up | COMPLETED | NNZ-2591 | 15 | |
| Treatment and Follow Up | NOT COMPLETED | NNZ-2591 | 3 |
Source ClinicalTrials.gov
Adverse events16
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 | 2 | 18 | 2 |
| other | Diarrhea | Gastrointestinal disorders | NNZ-2591 | 2 | 18 | 2 |
| other | Nausea | Gastrointestinal disorders | NNZ-2591 | 2 | 18 | 2 |
| other | Vomiting | Gastrointestinal disorders | NNZ-2591 | 2 | 18 | 2 |
| other | Fatigue | General disorders | NNZ-2591 | 2 | 18 | 2 |
| other | Pyrexia | General disorders | NNZ-2591 | 3 | 18 | 3 |
| other | Covid 19 | Infections and infestations | NNZ-2591 | 3 | 18 | 3 |
| other | Nasopharyngitis | Infections and infestations | NNZ-2591 | 2 | 18 | 2 |
| other | Otitis Media | Infections and infestations | NNZ-2591 | 2 | 18 | 2 |
| other | Decreased appetite | Metabolism and nutrition disorders | NNZ-2591 | 3 | 18 | 3 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | NNZ-2591 | 0 | 18 |
| Total, other adverse events | NNZ-2591 | 17 | 18 |
| Total, serious adverse events | NNZ-2591 | 1 | 18 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 41450730 | DERIVED |
Source PubMed
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/NCT05025241
GET https://conductscience.org/api/trials/v1/NCT05025241/results?page=2